Discovery Alert

It started out as "sort of a stupid thing to do", recalls Joe Bondy-Denomy, a microbiologist at the University of California, San Francisco. As a graduate student in the early 2010s, he tried to infect bacteria with viruses that, on paper, shouldn't have stood a chance. He knew that these viruses, or phages, were susceptible to CRISPR-Cas, the bacterial defense system that scientists have harnessed as a powerful tool for gene editing. And in most cases, he was right: the CRISPR machinery chopped the incoming phages into bits. But in a few instances, against the odds, the intruders survived. Learn More

A molecular switch has the ability to turn on a substance in animals that repairs neurological damage in disorders such as multiple sclerosis (MS), Mayo Clinic researchers discovered. The early research in animal models could advance an already approved Food and Drug Administration therapy and also could lead to new strategies for treating diseases of the central nervous system. Research by Isobel Scarisbrick, Ph.D., published in the Journal of Neuroscience finds that by genetically switching off a receptor activated by blood proteins, named Protease Activated Receptor 1 (PAR1), the body switches on regeneration of myelin, a fatty substance that coats and protects nerves. Learn More

A research team has reproduced and visualized the earliest developmental steps in human immune cell production in the laboratory. One day the advance could lead to a patient's own skin cells being used to produce new cells for cancer immunotherapy or to test autoimmune disease interventions. The group, led by Professors Ed Stanley and Andrew Elefanty, from the Murdoch Children's Research Institute in Melbourne, Australia, said the work has added definitive evidence about how the body's earliest immune cells are formed. The research combined genetic engineering and a novel way of growing stem cells, to make the breakthrough, which has been published in the journal Nature Cell Biology. Learn More

A novel nanoparticle vaccine that combines two major influenza proteins is effective in providing broad, long-lasting protection against influenza virus in mice, showing promise as a universal flu vaccine, according to a study by the Institute for Biomedical Sciences at Georgia State University. The double-layered nanoparticle vaccine contains the influenza virus proteins matrix protein 2 ectodomain (M2e) and neuraminidase (NA). Mice were immunized with the nanoparticle vaccine before being exposed to influenza virus, and they were protected against six different strains of the virus. The findings are published in the journal Advanced Healthcare Materials. Learn More

Increasing abundance of plastic waste has alarmed the society, the environmental fate of microplastics has been difficult to trace. A research group used carbon isotope labeling to follow the fate of polyethylene in the food chain. To the researchers' surprise, plastic carbon was transformed into beneficial fatty acids, omega-3 and omega-6, by the microbes originating from humic lakes. In the recently published study, Dr. Sami Taipale and his co-workers studied biodegradation of polyethylene, which is one of the most used plastics. Polyethylene was labelled with 13C-isotope, which enables the most sensitive technology for studying the fate of slowly degrading materials. Learn More

Mitochondria, tiny structures present in most cells, are known for their energy-generating machinery. Now, Salk researchers have discovered a new function of mitochondria: they set off molecular alarms when cells are exposed to stress or chemicals that can damage DNA, such as chemotherapy. The results, recently published online in Nature Metabolism, could lead to new cancer treatments that prevent tumors from becoming resistant to chemotherapy. "Mitochondria are acting as a first line of defense in sensing DNA stress. The mitochondria tell the rest of the cell, 'Hey, I'm under attack, you better protect yourself,'" says Gerald Shadel, a professor in Salk's Molecular and Cell Biology Laboratory. Learn More

Researchers at Rice University, Texas A&M University, Biola University and Durham (U.K.) University showed that motorized molecules developed in the Rice lab of chemist James Tour are effective at killing antibiotic-resistant microbes within minutes. The motors target the bacteria and, once activated with light, burrow through their exteriors. While bacteria can evolve to resist antibiotics by locking the antibiotics out, the bacteria have no defense against molecular drills. Antibiotics able to get through openings made by the drills are once again lethal to the bacteria. The researchers reported their results in the American Chemical Society journal ACS Nano. Learn More

Scientists at Sanford Burnham Prebys Medical Discovery Institute have shown that worms live longer lives if they produce excess levels of a protein, p62, which recognizes toxic cell proteins that are tagged for destruction. The discovery, published in Nature Communications, could help uncover treatments for age-related conditions, such as Alzheimer's disease, which are often caused by accumulation of misfolded proteins. While the scientists see much potential in their findings, they urge caution for direct translations to humans as high levels of p62 have been shown to be associated with cancer in humans. Learn More

In mouse models of Alzheimer's disease, the investigational drug candidates known as CMS121 and J147 improve memory and slow the degeneration of brain cells. Now, Salk researchers have shown how these compounds can also slow aging in healthy older mice, blocking the damage to brain cells that normally occurs during aging and restoring the levels of specific molecules to those seen in younger brains. The research, published last month in the journal eLife, suggests that the drug candidates may be useful for treating a broader array of conditions and points out a new pathway that links normal aging to Alzheimer's disease. Learn More

Seqirus has announced the results from a trial led by a team of researchers from Brown University that showed FLUAD®, an adjuvanted trivalent influenza vaccine (aTIV), was more effective than standard non-adjuvanted trivalent influenza vaccine (TIV) in reducing the risk of all-cause hospitalizations, as well as hospitalization for influenza and pneumonia, in adults 65 years and older living in United States(U.S.) nursing homes during the 2016/17 influenza season. These findings were presented at the recent National Foundation for Infectious Diseases (NFID) 2019 Clinical Vaccinology Course (CVC), which took place in Washington, D.C. Learn More

In November 2018, He Jiankui, a researcher in Shenzhen, China, claimed he utilized CRISPR-Cas9 gene editing to alter the DNA of embryos for seven couples. However, aside from the public presentations and the resulting controversy and government crackdown on He Jiankui in China, the actual technical results have not been published. This week, the MIT Technology Review announced they had received a copy of He's unpublished manuscript titled "Birth of Twins After Genome Editing for HIV Resistance" and subjected it to a thorough review. Among their observations, that the claims that He and his team made are not supported by the data and that the supposed medical benefits are dubious at best. Learn More

Placebo groups have long been viewed as a necessary evil by the medical profession when no other option is available. Nowadays, wherever possible, clinical trials will involve an 'active' control, where the new therapy is measured against the current standard of care available rather than just a placebo. However, a clarification in 2002 by the World Medical Association stated placebo-controlled trials are ethically acceptable even when a proven therapy is available. Fortunately, a potential solution to the ethical quandary raised by placebo groups is on the horizon. The digitisation of health records means real world evidence could be used to create a synthetic control arm to replace clinical trials' existing control arms. Learn More

Over the course of several months, researchers in Israel at the Weizmann Institute of Science created Escherichia coli strains that consume CO2 for energy instead of organic compounds. This achievement in synthetic biology highlights the incredible plasticity of bacterial metabolism and could provide the framework for future carbon-neutral bioproduction. The work appears November 27th in the journal Cell. Learn More

Genentech presented positive results from the Phase III Iclinical trial of Tecentriq in combination with Avastin in unresectable hepatocellular carcinoma (HCC) in people have haven't received previous systemic therapy. Tecentriq is the company's PD-L1 checkpoint inhibitor. Avastin is an antibody that binds to VEGF and interferes with tumor blood supply. In the trial, the drug combination showed statistically significant and clinically meaningful improvements in overall survival (OS) and progression-free survival (PFS) compared to the cohort receiving the tyrosine kinase inhibitor sorafenib. Learn More

With a shortage of new tuberculosis drugs in the pipeline, a software tool from the University of Michigan can predict how current drugs -- including unlikely candidates -- can be combined in new ways to create more effective treatments. "This could replace our traditional trial-and-error system for drug development that is comparatively slow and expensive," said Sriram Chandrasekaran, U-M assistant professor of biomedical engineering, who leads the research. Dubbed INDIGO, short for INferring Drug Interactions using chemoGenomics and Orthology, the software tool has shown that the potency of tuberculosis drugs can be amplified when they are teamed with antipsychotics or antimalarials. Learn More

In a breakthrough with important implications for the future of immunotherapy for breast cancer, UC San Francisco scientists have found that blocking the activity of a single enzyme can prevent a common type of breast cancer from spreading to distant organs. While studying a mouse model that replicates key features of early-stage human breast cancer, the researchers discovered that a ubiquitous enzyme called MMP9 is an essential component of the cancer's metastasis-promoting machinery, helping to create a hospitable environment for itinerant cancer cells to form new metastatic tumors. The new study is published this week in the journal Life Science Alliance. Learn More

Researchers led by Simon Cook at the Babraham Institute have found that cellular recycling (autophagy) is repressed during the process of cell division, and how repression of autophagy during mitosis utilizes a different master regulator. The findings address a long-standing point of contention in biology. The research uncovered the molecular intricacies of the cell signaling cascades that function to maintain genome integrity by repressing autophagy during the dynamic process of cell division. Researchers utilized cell signaling, autophagy, mass spectrometry and imaging expertise to resolve the fundamental question. Their findings were published this week in the journal Molecular Cell. Learn More

Researchers at McGill University's Faculty of Medicine have made important strides in understanding the functioning of enzymes that play an integral role in the production of antibiotics and other therapeutics. "This includes compounds made in microbes by massive enzymes called nonribosomal peptide synthetases, or NRPSs. NRPSs synthesize all sorts of antibiotics, which can kill dangerous fungi and bacteria, as well as compounds to help us fight off viral infections and cancers. In order to synthesize these drugs, NRPSs operate similar to a factory assembly line, consisting of a series of workstations. Learn More

Through the use of cutting-edge gene sequencing, a team of researchers from Abbott Laboratories discovered a new strain of HIV, the first time a new subtype of HIV-1 has been identified in nearly 20 years. The Abbott research marks the first time a new subtype of "Group M" HIV virus has been identified since guidelines for classifying new strains of HIV were established in 2000. Group M viruses are responsible for the global pandemic, which can be traced back to the Democratic Republic of Congo in Sub-Saharan Africa, Abbott said. The findings from the Abbott research have been published in the Journal of Acquired Immune Deficiency Syndromes (JAIDS). Learn More

Cell culture media have been an essential tool of biology for more than 70 years. Remarkably, the composition of these potions hasn't fundamentally changed much over that time, primarily because they deliver what scientists need: Cells that stay viable and rapidly divide. But Jason Cantor is thinking about cell culture media from another angle: Can we make it more human? Cantor, a metabolism investigator at the Morgridge Institute for Research and assistant professor of biochemistry at the University of Wisconsin-Madison, is a pioneer in the new development of "physiologic media," which are intended to place laboratory cells into an environment that very closely mimics real biological conditions. Learn More

A new type of micromotor -- powered by ultrasound and steered by magnets -- can move around individual cells and microscopic particles in crowded environments without damaging them. In one demonstration, a micromotor pushed around silica particles to spell out letters. Researchers also controlled the micromotors to climb up microsized blocks and stairs, demonstrating their ability to move over three dimensional obstacles. The micromotors are hollow, half capsule-shaped polymer structures coated with gold. They contain a small piece of magnetic nickel in their bodies, which allows them to be steered with magnets. The inner surface is chemically treated to repel water so that when it is submerged in water, an air bubble spontaneously forms inside the micromotor. Learn More

A nationwide team of researchers has found an antibody that protects mice against a wide range of potentially lethal influenza viruses, advancing efforts to design of a universal vaccine that could either treat or protect people against all strains of the virus. The study, which Scripps Research conducted jointly with Washington University School of Medicine in St. Louis and Icahn School of Medicine at Mount Sinai in New York, points to a new approach to tackle severe cases of the flu, including pandemics. The research is published in the October 25th, 2019 issue of Science. Learn More

For all its well-earned fame, the gene-editing tool CRISPR is, in reality, pretty hard on the genome. It's a pair of DNA scissors that cuts the double helix, and what's called "editing" is actually a cell's hasty attempt to patch things back together. That introduces errors: critics have even called these unpredictable changes a form of "genome vandalism." This week, in the latest-and possibly most important-of recent improvements to CRISPR technology, researchers at Harvard have introduced "prime editing," a molecular gadget they say can rewrite any type of genetic error without actually severing the DNA strand, as CRISPR does. Learn More

Gene therapy has broadened the treatment possibilities for those with immune system deficiencies and blood-based conditions, such as sickle cell anemia and leukemia. But today's standard process for administering gene therapy is expensive and time-consuming -- a result of the many steps required to deliver the healthy genes into the patients' blood stem cells to correct a genetic problem. In a discovery that appears in the journal Blood, scientists at Scripps Research believe they have found a way to sidestep some of the current difficulties, resulting in a more efficient gene delivery method that would save money and improve treatment outcomes. Learn More

Cancer cells are masters at avoiding detection, but a new system developed by Yale scientists can make them stand out from the crowd and help the immune system spot and eliminate tumors that other forms of immunotherapies might miss, the researchers reported on October 14th, 2019 in the journal Nature Immunology. The new system reduced or eliminated melanoma and triple-negative breast and pancreatic tumors in mice, even those located far from the primary tumor source, the researchers report. "This is an entirely new form of immunotherapy," said Sidi Chen, assistant professor of genetics and senior author of the study. Learn More

A research group at Ontario's Institute for Cancer Research has discovered a novel cancer-driving mutation in the vast non-coding regions of the human cancer genome, also known as the "dark matter" of human cancer DNA. The mutation, as described in two related studies published in Nature on October 9, 2019, represents a new potential therapeutic target for several types of cancer including brain, liver and blood cancer. This target could be used to develop novel treatments for patients with these difficult-to-treat diseases. The research group discovered that the mutation, termed the U1-snRNA mutation, could disrupt normal RNA splicing and thereby alter the transcription of cancer-driving genes. Learn More

In a recently published study, the laboratory of Dario C. Altieri, M.D., Wistar president and CEO, director of the Institute's Cancer Center and the Robert & Penny Fox Distinguished Professor, showed that MFF is highly expressed in cancer and interacts with VDAC1, a key regulator of mitochondrial cell death, to keep tumor cells alive. Altieri and collaborators analyzed the structure of the MFF-VDAC1 interface to determine what portions of the MFF protein are required for its interaction with VDAC1. As a result, this induced acute mitochondrial dysfunction and consequently cell death. These results appeared online in the journal Cancer Research. Learn More

MIT chemical engineers have now developed a new series of lipid nanoparticles to deliver such vaccines. They showed that the particles trigger efficient production of the protein encoded by the RNA, and they also behave like an "adjuvant," further boosting the vaccine effectiveness. In a study of mice, they used this RNA vaccine to successfully inhibit the growth of melanoma tumors. The study appears in the September 30th issue of Nature Biotechnology. "One of the key discoveries of this paper is that you can build RNA delivery lipids that can also activate the immune system in important ways," says Daniel Anderson, an associate professor in MIT's Department of Chemical Engineering. Learn More

Within six months of the U.S. FDA's move to restrict the label of two immunotherapies, usage of those therapies among oncologists dropped by about 50 percent, according to a new study from researchers in the Abramson Cancer Center at the University of Pennsylvania. The findings come as the world of cancer medicine grapples with the rapid pace of approval for new therapies, particularly as it relates to the FDA's accelerated approval (AA) program. Researchers say these findings offer reassurance that oncologists can be nimble enough to quickly incorporate the latest guidelines into their practices when new safety data comes to light. JAMA published the results this week. Learn More

Bioinorganic chemist Osami Shoji of Nagoya University and collaborators have found a way to hijack this 'haem acquisition system' for drug delivery. They developed a powder formed of HasA and the pigment gallium phthalocyanine (GaPc), which, when applied to a culture of P. aeruginosa, was consumed by the bacteria. The technique killed 99.9% of Pseudomonas aeruginosa, a potentially deadly, antibiotic-resistant bacterium present in hospitals. The strategy should also work for other dangerous bacteria. The strategy also worked on other bacteria with the HasR receptor on their membranes, but not on ones without it. Learn More

The complexity of molecular structures in the cell is amazing. Having achieved great success in elucidating these structures in recent years, biologists are now taking on the next challenge: to find out more about how they are constructed. A new research project now provides insight into a very unusual construction process in the unicellular parasite Trypanosoma brucei. The parasite Trypanosoma brucei was used as a model system since its mitoribosomes are particularly complex and, therefore, likely to require many assembly steps. The researchers could follow all these steps in detail. Learn More

Scientists at Cold Spring Harbor Laboratory (CSHL) have identified 10 cancer drugs currently in clinical trials that do not work how clinicians thought they would. In identifying what went wrong, experts can now work to improve drug discovery and personalized medicine. The discovery started out with an entirely different goal. Over the past few years, CSHL Fellow Jason Sheltzer's lab has been working to identify genes tied to low survival rates among cancer patients. During this work, the researchers discovered that MELK, a protein often found in high levels in tumors, has absolutely no influence on cancer growth. Learn More

A small clinical study in California has suggested for the first time that it might be possible to reverse the body's epigenetic clock, which measures a person's biological age. For one year, nine healthy volunteers took a cocktail of three common drugs - growth hormone and two diabetes medications - and on average shed 2.5 years of their biological ages, measured by analyzing marks on a person's genomes. The participants' immune systems also showed signs of rejuvenation. The results were a surprise even to the trial organizers - but researchers caution that the findings are preliminary because the trial was small and did not include a control arm. Learn More

Researchers at Oregon State University have made an important advance toward understanding why certain cells in the nervous system are prone to breaking down and dying, which is what happens in patients with ALS and other neurodegenerative disorders. The study into the role a protein known as heat shock protein 90 plays in intracellular signaling is a key step on the way to figuring out the reason some motor neurons in the spinal cord die and some do not. Findings, which could eventually lead to therapies to counter motor neuron death, were published in Experimental Biology and Medicine. Learn More

Genentech has announced that the Phase III BLOCKSTONE study showed preventive treatment with Xofluza™ (baloxavir marboxil) after exposure to an infected household member significantly reduced the risk of people developing the flu by 86 percent versus placebo. The results show just 1.9 percent of Xofluza-treated household members had the flu compared with 13.6 percent in the placebo-treated group. This benefit with Xofluza remained statistically significant versus placebo regardless of influenza A subtype. No serious adverse events were reported for Xofluza. Learn More

The ability to edit genes in living organisms offers the opportunity to treat a plethora of inherited diseases. However, many types of gene-editing tools are unable to target critical areas of DNA. and creating such a technology has been difficult as living tissue contains diverse types of cells. Now, Salk Institute researchers have developed a new tool -- dubbed SATI -- to edit the mouse genome, enabling the team to target a broad range of mutations and cell types. The new genome-editing technology, described in Cell Research on August 23rd, 2019, could be expanded for use in a broad range of gene mutation conditions. Learn More

A cancer therapy invented at Rice University has crossed a milestone in clinical trials, a major development in a decades long quest to develop a treatment that destroys tumors without the debilitating side effects of chemotherapy, invasive surgery and radiation. Thirteen of the first 15 prostate cancer patients treated in a clinical trial of the nanoparticle-based, focal therapy showed no detectable signs of cancer a year after treatment, according to a study published this week in the Proceedings of the National Academy of Sciences (PNAS). Learn More

Reproductive biologists at Weill Cornell Medicine in New York City are attempting to use the powerful gene-editing technique called CRISPR to alter genes in human sperm. The research is aimed at finding new ways to prevent disorders caused by genetic mutations that are passed down from men - including some forms of male infertility. The team is starting with a gene that can increase the risk for breast, ovarian, prostate and other cancers. The experiments are just starting and have not yet been successful. But the research raises many of the same hopes - and fears - as editing the DNA of human embryos. Nevertheless, the researchers defend the work. Learn More

The lab of Cheryl Kerfeld at Michigan State University has created a synthetic nano-sized factory, based on natural ones found in bacteria. To accomplish this researchers shuffled segments of the amino acid sequence and glued the original ends together. They then introduced new termini on the inner face of the protein. The result is a new, synthetic shell protein that looks almost identical to its natural counterpart. The team tested the concept by fusing a fluorescent cargo protein to the new BMC-H protein. Microscopy and biochemical testing show the cargo on the inside of the shell. The new study is published in Metabolic Engineering. Learn More

Scientists have discovered that the building blocks of proteins can stabilize cell membranes. This finding may explain how the first cells emerged from the primordial soup billions of years ago: The protein building blocks could have stabilized cell membranes against salt and ions that were present in ancient oceans. In addition, membranes may have been a site for these precursor molecules to co-localize, a potential mechanism to explain what brought together the ingredients for life. The findings, published this week in the Proceedings of the National Academy of Sciences, go beyond explaining how amino acids could have stabilized membranes in unfavorable environments. Learn More

A team of biologists has discovered how cells become different from each other during embryogenesis, a finding that offers new insights into genetic activity and has implications for better understanding the onset of disease and birth defects. Specifically, previous research had identified promoters -- or "on/off" switches" -- for thousands of genes. However, these studies had not delineated how these promoters are activated, leaving unclear fundamental aspects of how cells become different from each other during embryogenesis. The Molecular Cell study focused on a gene called hunchback (hb), which makes cells in the head region of the fly embryo that are different from cells in the abdomen. Learn More

Researchers for the first time have combined a powerful microscopy technique with automated image analysis algorithms to distinguish between healthy and metastatic cancerous tissue without relying on invasive biopsies or the use of a contrast dye. This new approach could one day help doctors detect cancer metastasis that is otherwise difficult to see via standard imaging technologies during operations. "The method utilized in this work identifies in a completely label-free manner cellular and tissue features at the microscopic level, essentially acting like a biopsy without a knife," added Dimitra Pouli from Tufts University, Medford, Mass., U.S.A., lead author of the study. Learn More

In this week's issue of Science Translational Medicine, researchers at Fred Hutchinson Cancer Research Center used CRISPR-Cas9 to edit long-lived blood stem cells to reverse the clinical symptoms observed with several blood disorders, including sickle cell disease and beta-thalassemia. It's the first time that scientists have specifically edited the genetic makeup of a specialized subset of adult blood stem cells that are the source of all cells in the blood and immune system. The proof-of-principle study suggests that efficient modification of targeted stem cells could reduce the costs of gene-editing treatments for blood disorders and other diseases while decreasing the risks of unwanted effects that can occur with a less discriminating approach. Learn More

New research from the USC Viterbi School of Engineering could be key to our understanding of how the aging process works. The findings potentially pave the way for better cancer treatments and revolutionary new drugs that could vastly improve human health in the twilight years. The work, from Assistant Professor of Chemical Engineering and Materials Science Nick Graham and his team in collaboration with Scott Fraser, Provost Professor of Biological Sciences and Biomedical Engineering, and Pin Wang, Zohrab A. Kaprielian Fellow in Engineering, was recently published in the Journal of Biological Chemistry. Learn More

A new molecular mechanism discovered by UT Southwestern researchers indicates that drugs currently used to treat less than 10 percent of breast cancer patients could have broader effectiveness in treating all cancers where the drugs are used, including ovarian and prostate cancers. The new study also revealed a potential biomarker indicating when these drugs, called PARP inhibitors, can be unleashed in the fight against cancer. This research helps explain why breast cancer patients can be responsive to PARP inhibitors even if they don't have BRCA gene mutations. The research team's findings were published in the journal Molecular Cell this week. Learn More

University of Calgary researchers are the first to discover a previously unidentified cell population in the pericardial fluid found inside the sac around the heart. The discovery could lead to new treatments for patients with injured hearts. The study led by Drs. Paul Kubes, PhD, Justin Deniset, PhD and Paul Fedak, MD, PhD is published in the journal Immunity. The Kubes lab, in collaboration with the Fedak lab, found that a specific cell, a Gata6+ pericardial cavity macrophage, helps heal an injured heart in mice. The cell was discovered in the pericardial fluid of a mouse with a heart injury. Learn More

Peptides, one of the fundamental building blocks of life, can be formed from the primitive precursors of amino acids under conditions similar to those expected on the primordial Earth, finds a new UCL study. The findings, published in Nature, could be a missing piece of the puzzle of how life first formed. "This is the first time that peptides have been convincingly shown to form without using amino acids in water, using relatively gentle conditions likely to be available on the primitive Earth," said co-author Dr Saidul Islam (UCL Chemistry). Learn More

A scientific team led by Case Western Reserve University School of Medicine and Louis Stokes Cleveland VA Medical Center have shown that using two antibiotic drugs to fight P. aeruginosa in mouse models is significantly more effective than either antibiotic alone. The antibiotics, referred to as a "double whammy" were ceftazidime-avibactam, a combination drug used to treat a wide variety of serious bacterial infections, and fosfomycin, used to primarily treat infections of the urinary tract.  The study appears in the recent Journal of Infectious Diseases. Learn More

In a major collaborative effort, researchers at the Lewis Katz School of Medicine at Temple University and the University of Nebraska Medical Center (UNMC) have for the first time eliminated replication-competent HIV-1 DNA -- the virus responsible for AIDS -- from the genomes of living animals. The study, reported online July 2 in the journal Nature Communications, marks a critical step toward the development of a possible cure for human HIV infection. For the new study, Dr. Khalili and colleagues combined their CRISPR-CAS 9gene editing system with a recently developed therapeutic strategy known as long-acting slow-effective release (LASER) ART. Learn More

In a surprising marriage of science and art, researchers at MIT have developed a system for converting the molecular structures of proteins, the basic building blocks of all living beings, into audible sound that resembles musical passages. Then, reversing the process, they can introduce some variations into the music and convert it back into new proteins never before seen in nature. Although it's not quite as simple as humming a new protein into existence, the new system comes close. It provides a systematic way of translating a protein's sequence of amino acids into a musical sequence, using the physical properties of the molecules to determine the sounds. Learn More

St. Jude Children's Research Hospital scientists have discovered a pathway that functions like a car wash to prevent the buildup of a toxic protein associated with Alzheimer's disease. The findings in a mouse model of Alzheimer's offer a possible new approach to treatment of the chronic neurodegenerative disorder, which is the sixth leading cause of death in the U.S. The newly identified pathway also helps regulate inflammation, so the discovery could yield strategies for unleashing the immune response against malignant brain tumors. The report appears online in the journal Cell. Learn More

Traditionally, scientists have used light, x-rays, and electrons to peer inside tissues and cells. Today, scientists can trace thread-like fibers of nerves throughout the brain and even watch living mouse embryos conjure the beating cells of a rudimentary heart. But there's one thing these microscopes can't see: what's happening in cells at the genomic level. Now, biophysicist Joshua Weinstein and colleagues have invented an unorthodox type of imaging dubbed "DNA microscopy" that can do just that. Instead of relying on light (or any kind of optics at all), the team uses DNA "bar codes" to help pinpoint molecules' relative positions within a sample. Their work appears in this week's journal Cell. Learn More

Researchers at the University of California San Francisco have discovered one of the first concrete examples of how the microbiome can interfere with a drug's intended path through the body. Focusing on levodopa (L-dopa), the primary treatment for Parkinson's disease, they identified which bacteria out of the trillions of species is responsible for degrading the drug and how to stop this microbial interference. But this microbial interference may not be limited to L-dopa and Parkinson's disease. Their study could shepherd additional work to discover exactly who is in our gut, what they can do, and how they can impact our health, for better or worse.  Learn More

Transgenerational bisphenol A (BPA) exposure may contribute to autism, according to a mouse study published in the Endocrine Society's journal Endocrinology. Endocrine disrupting chemicals (EDCs) are chemicals or mixtures of chemicals that interfere with the way the body's hormones work. BPA is a common EDC used in plastics and food storage material, and it is already present in most humans' urine or blood. Animal studies have linked BPA to anxiety, aggression, and poor learning and social interactions. Studies of human populations report associations between BPA and neurobehavioral issues like attention deficit hyperactivity disorder and autism. Learn More

Discovery of a new feature of a large class of pathogenic viruses, referred to by researchers as a 'pocket', may allow development of new antiviral medications for the common cold, polio, and other illnesses, according to a new study published this week in the open-access journal PLOS Biology. Researchers Rana Abdelnabi and Johan Neyts of the University of Leuven, Belgium, and James Geraets and Sarah Butcher of the University of Helsinki and their colleagues note that while it is possible that the newly-discovered pocket may also mutate to make picornaviruses resistant to therapies developed against them, the authors suggest the pocket may be crucial enough for viral replication that viruses containing mutant versions may be less viable. Learn More

An international team of researcher's searched more than 8,000 virus genomes and found that many newly-discovered giant viruses contain multiple genes for a type of enzyme called cytochrome P450. P450 enzymes are common in animals, plants and bacteria, but finding them in new viruses is unexpected. Prior to the giant viruses, it was never considered that viruses would have these genes. The study, led by researchers at Woods Hole Oceanographic Institution (WHOI) and Swansea University Medical School, is published this week in Proceedings of the National Academy of Sciences. Learn More

Six months ago, a Chinese scientist announced that he had edited the genomes of two babies born last year. The germline edits with CRISPR-Cas9 supposedly changed the CCR5 gene to prevent HIV from invading immune cells. An analysis of records in the U.K. Biobank shows that having two copies of this mutation is associated with a 21 percent increase in mortality. The researchers scanned more than 400,000 genomes and associated health records and found that people who had two mutated copies of the gene had a significantly higher death rate between ages 41 and 78 than those with one or no copies. The research appears in this week's journal Nature Medicine. Learn More

Many people don't believe in evolution. Only one-third of Americans say it occurs through processes like natural selection and no involvement by God. But besides religion, there are several reasons why some reject the fact life evolves, such as not seeing adaptation happening with your own eyes. Scientists have now provided further proof of evolution in action with a video of bacteria evolving resistance to an antibiotic drug. The short film shows the results of a study by microbiologists at the Université Claude Bernard Lyon 1 in France, which reveals how rapidly Escherichia coli can acquire genes that confer the ability to resist the effects of tetracycline. Learn More

The rose may be one of the most iconic symbols of the fragility of love in popular culture, but now the flower could hold more than just symbolic value. A new device for collecting and purifying water, developed at The University of Texas at Austin, was inspired by a rose and, while more engineered than enchanted, is a dramatic improvement on current methods. Each flower-like structure costs less than 2 cents and can produce more than half a gallon of water per hour per square meter. In a paper published in the most recent issue of the journal Advanced Materials, the authors outline how an origami rose provided the inspiration for developing a new kind of solar-steaming system. Learn More

Researchers from the Hubrecht Institute and Radboud University have developed a human model in which they use organoids, or mini organs, to study the function of specific genes that are mutated in liver cancer. Using this method, they have found that mutations in BAP1, a gene commonly mutated in liver cancer, changes the behavior of the cells, which may make them more likely to be invasive. Since the model makes it relatively straightforward to manipulate healthy human organoids with CRISPR/Cas9, it may be used to study the function of the many genes with unknown function in liver cancer. Their results were published this week in the scientific journal Cell Stem Cell. Learn More

Researchers at the Icahn School of Medicine at Mount Sinai have demonstrated that stem cells derived from the placenta known as Cdx2 cells can regenerate healthy heart cells after heart attacks in animal models. The findings, published in this week's issue of Proceedings of the National Academy of Sciences, may represent a novel treatment for regenerating the heart and other organs. Researchers noted two other properties of the Cdx2 cells: they have all the proteins of embryonic stem cells, which are known to generate all organs of the body, but also additional proteins, giving them the ability to travel directly to the injury site, which is something embryonic stem cells cannot do, and they appear to avoid the host immune response. Learn More

A new study conducted at the University of Copenhagen challenges traditional knowledge of stem cell development. The study reveals that the destiny of intestinal cells is not predetermined, but instead determined by the cells' surroundings. The findings may make it easier to manipulate stem cells for stem cell therapy. Results have just been published in Nature. The surprising findings are the result of a search for understanding of what controls the destiny of intestinal stem cells. By introducing luminescent proteins into the cells researchers could, using advanced microscopy, monitor the development of the individual cells. Learn More

Research uncovers key mechanism that allows some of the deadliest human RNA viruses to orchestrate the precise copying of the individual pieces of their viral genome and replicate. The findings identify new targets to inhibit viral replication and may inform the development of a novel class of antiviral drugs. The findings, published last week in PNAS, identify new potential targets to inhibit the replication of segmented viruses. This group includes several emerging and highly fatal viruses such as Lassa fever virus, bunyaviruses like La Crosse and Rift Valley fever, as well as the better known and more common influenza viruses. Learn More

Researchers from King's College London have found that therapy that can induce heart cells to regenerate after a heart attack. At present, when a patient survives a heart attack, they are left with permanent structural damage to their heart through the formation of a scar, which can lead to heart failure in the future. In contrast to fish and salamander, which can regenerate the heart throughout life. In this study, published this week in Nature, the team of investigators delivered a small piece of genetic material, called microRNA-199, to the heart of pigs, after a myocardial infarction which resulted in the almost complete recovery of cardiac function at one month later. Learn More

Synthetic proteins engineered to recognize overly active biological pathways can kill cancer cells while sparing their healthy peers, according to a study by researchers at the Stanford University School of Medicine. The customizable approach, which the researchers call RASER, relies on just two proteins: The first is activated in the presence of an "always on" growth signal often found in cancer cells, and the second carries out a researcher-programmed response, such as triggering the expression of genes involved in cell death. Although the experiments were confined to cells grown in the laboratory, the researchers believe the results could lead to a new type of cancer therapy. Learn More

Polymers are usually the go-to material for thermal insulation. Think of a silicone oven mitt, or a Styrofoam coffee cup, both manufactured from polymer materials that are excellent at trapping heat. Now MIT engineers have flipped the picture of the standard polymer insulator, by fabricating thin polymer films that conduct heat -- an ability normally associated with metals. In experiments, they found the films, which are thinner than plastic wrap, conduct heat better than many metals, including steel and ceramic. The team's results are published in the journal Nature Communications. Learn More

Researchers at DOE's Lawrence Berkeley National Laboratory (Berkeley Lab) have 3D-printed an all-liquid device that, with the click of a button, can be repeatedly reconfigured on demand to serve a wide range of applications -- from making battery materials to screening drug candidates. To make the 3D-printable fluidic device, Berkeley Lab researchers designed a specially patterned glass substrate. When two liquids - one containing nanoscale clay particles, another containing polymer particles - are printed onto the substrate, they come together at the interface of the two liquids and within milliseconds form a very thin channel or tube about 1 millimeter in diameter. The study's findings appear in the journal Nature Communications. Learn More

Although they are considered the simplest of all life forms, even microorganisms sense their environment and are able to actively move within it. The ability of cells to target or avoid particular substances is called chemotaxis. Until now, scientists have generally considered the chemotactic properties of bacteria to be a common feature of a species or population -- as if all cells behaved more or less the same. Now, researchers at ETH Zurich have observed the chemotaxis of bacteria in a behavioral experiment. "If you look with the appropriate technology, you'll find astonishing behavioral differences even within a population of genetically identical cells," report Mehdi Salek and Francesco Carrara, the lead authors of a study recently published in Nature Communications. Learn More

MD Anderson Cancer Center is ousting three scientists in connection with concerns China is trying to steal U.S. scientific research, the first such publicly disclosed punishments since federal officials directed some institutions to investigate specific professors in violation of granting agency policies. This comes after the center received e-mails last year from the NIH describing conflicts of interest or unreported foreign income by five faculty members. MD Anderson's action comes amid heightened concern that China and others are exploiting U.S.-funded research for their own benefit, enlisting students and visiting scholars to pilfer intellectual property, luring scientists to run "shadow laboratories" in their countries. Learn More

Citing lackluster financial performance, IBM is halting development and sales of a product that uses its Watson artificial intelligence software to help biopharma companies discover new drugs. The decision to shut down sales of Watson for Drug Discovery marks the highest-profile retreat in the company's effort to apply artificial intelligence to various areas of health care. Last year, the company scaled back on the hospital side of its business, and it's struggled to develop a reliable tool to assist doctors in treating cancer patients. Learn More

An experimental gene therapy has cured eight infants with the so-called Bubble Boy Disease, an immune-system deficiency so severe that children with it were at one time kept in total isolation for fear that even a simple common cold could be deadly. In a major scientific breakthrough, researchers at St. Jude Children's Research Hospital in Memphis have developed a one-time, personalized treatment used to correct the genetic defect and build fully functioning immune systems in infants with the condition, formally known as X-linked severe combined immunodeficiency, or SCID.  The boys, all toddlers now, have been released from the protective isolation units that dominated their early lives. Learn More

Stanford University has cleared Dr. Stephen Quake, a bioengineering professor, and two other professors, of any wrongdoing in their interactions with a Chinese researcher who roiled the scientific world by creating the first gene-edited babies. In a letter to Dr. Quake the university, referring to the Chinese scientist, He Jiankui, by his nickname, JK, says that Stanford's investigators concluded that Dr. Quake did not "directly participate in any way in JK's research, including in the conception or performance of the work." In fact, the letter said, Dr. Quake discouraged Dr. He from pursuing the project. Learn More

Bacterial cells that normally colonize our guts can distinguish themselves from other bacterial species using what's traditionally considered their enemy -- a virus. Researchers report that some bacteria use viruses that have infected them (i.e., phages) for self-recognition and thereby show greater fitness, repelling competitors that lack this adaptation.  "This is the first evidence that cells can distinguish themselves from related competitors through the use of a virus," says Thomas Wood of Pennsylvania State University, one of the co-senior authors on the study which appear in this weeks journal Cell Reports. Learn More

In a major medical breakthrough, Tel Aviv University researchers Prof. Dvir, Dr. Assaf Shapira of TAU's Faculty of Life Sciences and Nadav Moor, a doctoral student in Prof. Dvir's lab, have "printed" the world's first 3D vascularized engineered heart using a patient's own cells and biological materials. Until now, scientists in regenerative medicine, a field positioned at the crossroads of biology and technology, have been successful in printing only simple tissues without blood vessels. Their findings were published on April 15 in a study in Advanced Science. Learn More

Dangerous airborne viruses are rendered harmless on-the-fly when exposed to energetic, charged fragments of air molecules, University of Michigan researchers have shown. They hope to one day harness this capability to replace a century-old device: the surgical mask. The U-M engineers have measured the virus-killing speed and effectiveness of nonthermal plasmas -- the ionized, or charged, particles that form around electrical discharges such as sparks. A nonthermal plasma reactor was able to inactivate or remove from the airstream 99.9% of a test virus, with the vast majority due to inactivation. Achieving these results in a fraction of a second within a stream of air holds promise for many applications where sterile air supplies are needed. Learn More

Scientists at the University of California San Diego have developed a new version of a gene drive that allows the spread of specific, favorable genetic variants, also known as 'alleles,' throughout a population. The new 'allelic drive' is equipped with a guide RNA that directs CRISPR to cut undesired variants of a gene and replace it with a preferred version. Using a word processing analogy, CRISPR-based gene drives allow scientists to edit sentences of genetic information, while the new allelic drive offers letter-by-letter editing. The research appears in this weeks Nature Communications. Learn More

An international team has unveiled a new CRISPR-based tool that acts more like a shredder than the usual scissor-like action of CRISPR-Cas9. The new approach, based on Type I CRISPR-Cas3, is able to wipe out long stretches of DNA in human cells with programmable targeting, and has been shown to work in human cells for the first time. Writing in Molecular Cell, they describe how they succeeded in getting a different kind of CRISPR-Cas system called Type I CRISPR-Cas3 to work as long-range DNA editing tool in human cells for the first time. Learn More

The first clinical trial of an innovative universal influenza vaccine candidate is examining the vaccine's safety and tolerability as well as its ability to induce an immune response in healthy volunteers. Scientists at the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, developed the experimental vaccine, known as H1ssF_3928. The vaccine is designed to teach the body to make protective immune responses against diverse influenza subtypes by focusing the immune system on a portion of the virus that varies relatively little from strain to strain. The vaccine candidate was developed as part of a broader research agenda to create a so-called "universal" influenza vaccine. Learn More

The plant that adds flavor, color and bitterness to beer also produces a primary compound that thwarts cancer cells, and two important derivatives of the compound do as well, new research at Oregon State University shows. Unlike the primary compound, xanthohumol, known as XN, the derivatives don't metabolize into phytoestrogens. The research showed, for the first time, that the derivatives have cancer-fighting effectiveness similar to that of XN in liver and colon carcinomas. That means the two non-estrogenic derivatives are attractive alternatives for testing, along with XN, in future preclinical studies. The study was published in the International Journal of Molecular Sciences. Learn More

New research from the University of Pittsburgh School of Medicine and the UPMC Hillman Cancer Center sheds light on how tumors use the body's regulators of immunity for their own benefit. The findings, Published this week in Nature Immunology, could be used to develop the next generation of immune therapies to fight various cancers. "Our findings uncovered a previously unknown biological mechanism that reveals new therapeutic approaches to promote anti-tumor immunity" said senior author Dario Vignali, Ph.D., who holds the Frank Dixon Chair in Cancer Immunology, and is professor and vice chair of immunology at Pitt's School of Medicine. Learn More

Since its invention, CRISPR has let scientists introduce DNA changes at specific locations in a genome. Often these precise changes are made one at a time. Perhaps not for much longer. A team at Harvard University says it has used the technique to make 13,200 genetic alterations to a single cell, a record for the gene-editing technology. The group, led by gene technologist George Church, wants to rewrite genomes at a far larger scale than has currently been possible, something it says could ultimately lead to the "radical redesign" of species-even humans. Learn More

A new combination treatment aimed at resistant and recurrent low-grade gliomas slowed tumor growth and killed tumor cells in laboratory and mouse models. Researchers at the Johns Hopkins Kimmel Cancer Center and the Johns Hopkins University School of Medicine combined carboplatin, a standard chemotherapy drug that works well against these brain tumors, and everolimus, which blocks an enzyme called mTOR that was shown in earlier research to fuel the growth of these tumors. The combination increased DNA damage and cell death in laboratory models. Their findings were published in the February 14th, 2019, issue of Neuro-Oncology. Learn More

Scientists from the German Cancer Research Center (DKFZ) have succeeded in reconstructing biomolecules in their mirror-image form. The researchers' goal is to create a mirror-image artificial protein synthesis system. Their aim is to produce mirror-image therapeutic proteins, such as antibodies, which would be protected from biological breakdown in the body and do not provoke any immune response. In future, they intend to build more than single molecules: "Our long-term goal is to create simple, artificial biological systems in mirror-image form that corresponds to those in nature but do not interact with the environment," says project leader Jörg Hoheisel. Learn More

Janssen and Scripps have generated an oral compound for treating flu that inhibits the HA protein on influenza H1 and H5 strains. The Janssen Pharmaceuticals and Scripps Research Institute team sought to identify small molecules that mimic a previously identified bNAb's interaction with HA and inhibit flu virus fusion. As reported in Science, the team screened a library of about 500,000 compounds for small molecules that blocked the bNAb from binding HA, to indicate they bind the same epitope as the bNAb. The researchers optimized the top hit for potency and aqueous solubility to enable oral administration. Learn More

Researchers have created tiny functional, remote-powered, walking robots, developing a multistep nanofabrication technique that turns a 4-inch specialized silicon wafer into a million microscopic robots in just weeks. Each one of a robot's four legs is just under 100-atoms-thick, but powered by laser light hitting the robots' solar panels, they propel the tiny robots. The researchers are now working on smart versions of the robots that could potentially make incredible journeys in the human body. Researchers presented their microscopic robot research this week at the American Physical Society March Meeting in Boston. Learn More

CRISPR, the gene editing technique that promises to revolutionize healthcare and medicine, is not perfect. Right from the beginning, there have been concerns that this technique, which makes it easy to select specific areas of the genome and quickly and easily snip out and replace pieces of DNA, may also make unintended cuts in other areas of the genome at the same time. And two new studies, one in mice embryos and the other in rice plants, seem to confirm this. Learn More

The first map of great white sharks' DNA has revealed "mutations" that protect the animals against cancer and other illnesses. Scientists hope more research could help apply the findings to treating age-related illnesses in humans. The great white's ability to repair its own DNA has evolved in ways ours hasn't. The research was carried out by a team of scientists at the Save Our Seas Foundation Shark Research Centre at Nova Southeastern University in Florida. Learn More

UC San Francisco scientists have used the CRISPR-Cas9 gene-editing system to create the first pluripotent stem cells that are functionally "invisible" to the immune system, a feat of biological engineering that, in laboratory studies, prevented rejection of stem cell transplants. Because these "universal" stem cells can be manufactured more efficiently than stem cells tailor-made for each patient - the individualized approach that dominated earlier efforts - they bring the promise of regenerative medicine a step closer to reality. Learn More

At EPFL, Selman Sakar's research team has developed micromachines able to mechanically stimulate cells and microtissue. These tools, which are powered by cell-sized artificial muscles, can carry out complicated manipulation tasks under physiological conditions on a microscopic scale. The tools consist of microactuators and soft robotic devices that are wirelessly activated by laser beams. They can also incorporate microfluidic chips, which means they can be used to perform combinatorial tests that involve high-throughput chemical and mechanical stimulation of a variety of biological samples. This research has been published in Lab on a Chip. Learn More

Moderna has announced the dosing of the first monoclonal antibody encoded by mRNA to be dosed in a human and the first development candidate from the Company's systemic therapeutics modalities to start clinical testing. We believe this trial will give us important information about how mRNA may be used to make systemically-available complex therapeutic proteins in a consistent, dose-dependent fashion," said Tal Zaks, M.D., Ph.D., chief medical officer at Moderna. Learn More

Researchers studying p53, the heralded cancer-fighting 'guardian of the genome,' found that the human protein also plays a role in promoting tumors, in addition to suppressing them. They found that the PUMA protein works inside the cell's mitochondria to switch energy production processes and stimulate cancer growth. In the January 31st issue of Cancer Cell, scientists at UC San Diego describe the culmination of more than four years of research on liver cancer that shows that WTp53 stimulates tumor growth by enhancing cancer metabolism. Learn More

Researchers from Charité -- Universitätsmedizin Berlin have discovered a protective mechanism which is used by the body to protect intestinal stem cells from turning cancerous. The body's innate immune system was found to play a pivotal role in this regard. The researchers were able to demonstrate that, rather than having a purely defensive role, the immune system is crucial in maintaining a healthy body. The study has been published in the scientific journal Nature. Learn More

To perform properly the task for which they have been synthesized, proteins must first assemble to form effective cellular "machines." But how do they recognize their partners at the right time? Researchers at the University of Geneva (UNIGE) have deciphered the fundamental role of the Not1 protein, conserved in all eukaryotic organisms: by regulating the activity of ribosomes Not1 allows proteins that must work together to be synthesized in the same place and at the same time. The identification of this previously unknown mechanism helps to better understand one of the most fundamental elements of cellular machinery. Results can be read in Nature Structural & Molecular Biology. Learn More

Salk Institute scientists studying the relationship of telomeres to cancer made a surprising discovery: a cellular recycling process called autophagy -- generally thought of as a survival mechanism -- actually promotes the death of cells, thereby preventing cancer initiation. The work, which appeared in the journal Nature on January 23rd, 2019, reveals autophagy to be a completely novel tumor-suppressing pathway and suggests that treatments to block the process in an effort to curb cancer may unintentionally promote it very early on. Learn More

Scientists have traditionally thought that DNA binding proteins use patterns in the genome's code of As, Cs, Ts, and Gs to guide them to the right location, with a given protein only binding to a specific sequence of letters. In the new study, published in Cell Systems, the Gladstone scientists discovered that proteins must rely on another clue to know where to bind: the DNA's three-dimensional shape. Learn More

Researchers from the Catalonian Institute of Bioengineering and the Seville Chemical Research Institute have described a new method for the transmission of electrons between proteins that refutes the evidence from experiments until now. This process, involved in the generation of energy in both animal and plant cells, will permit better understanding of the behaviour of proteins in the cells, as well as giving a deeper understanding of the energy dysfunctions that cause diseases. The findings were published in the review Nature Communications. Learn More

In an advance that could lead to new treatments for lung disease, MIT researchers have now designed an inhalable form of mRNA. This aerosol could be administered directly to the lungs to help treat diseases such as cystic fibrosis, the researchers say. The researchers showed that they could induce lung cells in mice to produce a target protein -- in this case, a bioluminescent protein. If the same success rate can be achieved with therapeutic proteins, that could be high enough to treat many lung diseases, the researchers say. Asha Patel, a former MIT postdoc who is now an assistant professor at Imperial College London, is the lead author of the paper, which appears in the January 4th, 2019 issue of the journal Advanced Materials. Learn More

Proteins have now been designed in the lab to zip together in much the same way that DNA molecules zip up to form a double helix. The technique, whose development was led by University of Washington School of Medicine scientists, could enable the design of protein nanomachines that can potentially help diagnose and treat disease, allow for the more exact engineering of cells and perform a wide variety of other tasks. The research was performed at UW Medicine's Institute of Protein Design and the Howard Hughes Medical Institute. The researchers report their findings in the December 19th, 2018 issue of the journal Nature. Learn More

Princeton molecular biologist Bonnie Bassler and graduate student Justin Silpe have identified a virus, VP882, that can listen in on bacterial conversations -- and then, in a twist, they found a way to use that to make it attack bacterial diseases like E. coli and cholera. "The idea that a virus is detecting a molecule that bacteria use for communication -- that is brand-new," said Bassler, the Squibb Professor of Molecular Biology. "Justin found this first naturally occurring case, and then he re-engineered that virus so that he can provide any sensory input he chooses, rather than the communication molecule, and then the virus kills on demand." Their paper will appear in the January 10th, 2019 issue of the journal Cell. Learn More

After over a decade of preclinical research and development, a new gene therapy treatment for sickle cell anemia (SCA) is reversing disease symptoms in two adults and showing early potential for transportability to resource-challenged parts of the world where SCA is most common. Preliminary data from a pilot Phase 1-2 clinical trial testing the gene-addition therapy were presented Dec. 3 at the American Society of Hematology's (ASH) annual meeting in San Diego by principal investigator Punam Malik, MD. She is a physician-scientist at the Cincinnati Children's Cancer and Blood Diseases Institute and director of its Comprehensive Sickle Cell Center. Malik called the data promising. Learn More