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    ASGCT 2025: CRISPR-Cas12 Editing Shows Early Clinical Benefit in Duchenne Muscular Dystrophy

    At the upcoming annual meeting of the American Society of Cell and Gene Therapy, scientists from Huidagene Therapeutics will share an update on the development of their CRISPR-high-fidelity(hf)Cas12Max-based gene editing therapy for Duchenne muscular dystrophy, one of the most severe forms of...
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    SARS-CoV-2 Likely Spread Through Wildlife Trade, Not Bat Migration

    Since the start of the COVID-19 pandemic, there has been controversy surrounding the origins of SARS-CoV-2. New research from the University of California, San Diego (UCSD), School of Medicine suggests that the virus likely reached the human epicenter in Wuhan, China, not via bat migration but...
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    Light Microscopy-Based Connectomics Reconstructs Brain Tissue Including Synaptic Connections

    Our brain is a complex organ. Billions of nerve cells are wired in an intricate network, constantly processing signals, enabling us to recall memories or to move our bodies. Making sense of this complicated network requires a precise look into how these nerve cells are arranged and connected. A...
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    Vision Loss in Diabetic Mice Promoted by Hypoglycemia, Treated with HIF Inhibitors

    Vision loss is a complication for many diabetic patients. In patients with diabetic retinopathy, the principal cause of vision loss is the breakdown of the inner blood-retinal barrier (iBRB)—an important boundary that regulates the flow of nutrients, waste, and water in and out of the retina—due...
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    Gut Microbiome Linked to Rheumatoid Arthritis Through Reprogrammed T Helper Cells

    After spending years tracing the origin and migration pattern of an unusual type of immune cell in mice, researchers headed by a team at The Ohio State University College of Medicine have shown how the activity of “good” microbes in the gut is linked to rheumatoid arthritis (RA) and...
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    Astraveus Enters CAR-T Therapy Manufacturing Partnership with NecstGen

    Paris-based Astraveus formed a strategic partnership with The Netherlands Center for the Clinical Advancement of Stem Cell and Gene Therapies (NecstGen) to evaluate The Lakhesys Benchtop Cell Factory for the manufacturing of CAR-T therapies. NecstGen will use Astraveus’ Lakhesys product at its...
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    BMS Commits $40B over Five Years to U.S. R&D, Manufacturing

    Bristol Myers Squibb (BMS) Board Chair and CEO Christopher Boerner, PhD Bristol Myers Squibb, Board Chair of BMS and CEO Christopher Boerner, PhD, have committed the pharma giant to investing $40 billion over the next five years toward U.S. research and development (R&D), technology, and...
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    AI Uncovers Gut Signature Tied to Complex Regional Pain Syndrome

    By training machine learning algorithms on data from gut bacteria, scientists from McGill University and their collaborators developed a computational tool to detect patterns in the microbiome that are connected to complex regional pain syndrome (CRPS), a relatively rare condition that affects...
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    Opposing Forces: How GSK3β and ERK1 Phosphorylation Shape Huntington’s Disease

    It is well understood that the neurodegenerative disorder Huntington’s disease is caused by an N-terminal polyQ-expansion (>35) in the Huntingtin (HTT) gene. This mutation leads to axonal degeneration and significant neuronal death. Now, new work builds off of previous findings that two...
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    Muscle’s Circadian Clock Regulates Protein Degradation in Zebrafish, May Lead to Treatments

    Muscle cells contain their own circadian clocks and disrupting them with shift work can have a profound impact on aging, according to the results of a preclinical study carried out by Jeffrey Kelu, PhD, and Simon Hughes, PhD, at the Randall Centre for Cell and Molecular Biophysics, King’s...
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    Regeneration Biomedical’s Cell Therapy Reduces Tau, Boosts Cognition in Alzheimer’s Cases

    Patients in an ongoing Phase I clinical trial of a new stem cell therapy for Alzheimer’s disease (AD) are showing a reduction in proteins linked to disease progression and improvements in cognitive scoring. This is according to preliminary data from Regeneration Biomedical, a clinical-stage...
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    This Week in Gaming News: April 28th – May 4th, 2025

    If the relentless anxiety of the real-world news cycle is getting you down, then why not grab a chair and join us for another rundown of what’s been happening in the gaming world this week? Of course, some of the news out of the industry hasn’t exactly been positive this week either, so it might...
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    Recursion Halts Four Pipeline Programs, Sharpening Cancer, Rare Disease Focus

    Recursion said it will end development for four of its 11 pipeline programs—one of which the company will consider outlicensing to a partner instead—and pause a fifth program, in a pruning designed to further focus the artificial intelligence (AI)-based drug developer on cancer and rare disease...
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    CRISPR-Edited TILs Fight Advanced Colorectal Cancer in Patients

    Immunotherapeutic strategies have made huge strides in cancer treatment for many solid tumors over the past decade. But the promising results have failed to make a large change in the treatment of gastrointestinal forms of cancer. Now, a first-in-human clinical trial is testing a CRISPR/Cas9...
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    StockWatch: Second Time’s the Charm for Abeona’s Gene Therapy

    “Third time’s the charm,” the old saying goes, but for Abeona Therapeutics (NASDAQ: ABEO), the second time around proved successful for the company’s first-ever pipeline candidate to make it through clinical trials. The FDA approved Abeona’s Zevaskyn (prademagene zamikeracel) as the first (and...
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    Mitosis Mechanism for Viral Genome Insertions Unlocks Cell Defense Applications

    Viruses insert “transposable elements” into the genetic material of host cells to replicate. While cells’ defense mechanisms have learned to silence most of these viral insertions, a few “jumping genes” retain the potential to disrupt essential genes and cause serious health threats, such as...
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    RoosterBio and Thermo Fisher Scientific Agree to Collaborate to Advance Cell and Exosome Therapy Manufacturing

    RoosterBio, a supplier of adult human mesenchymal stem/stromal cells (hMSCs), engineered media, and bioprocess development services, entered into a collaboration with Thermo Fisher Scientific which aims to accelerate the availability of new cell and exosome therapies that have the potential to...
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    Antivenom Cocktail Protects Against 19 of World’s Deadliest Snakes

    Scientists have used antibodies from a human donor with a self-induced hyper-immunity to snake venom to develop what they claim is the most broadly effective antivenom cocktail to date. Their preclinical in vivo tests demonstrate that the antivenom, which comprises two protective antibodies and...
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    Targeting GI Drug Delivery with GlycoCaging Could Improve IBD Treatments

    Scientists headed by a team at the University of British Columbia (UBC) have developed a new approach to drug design that can deliver therapeutics directly to a specific part of the gastrointestinal (GI) tract. The drug delivery system, GlycoCaging, is based on bespoke glycoconjugates of a...
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    The Oncomicrobe in the Room: Surprise Antimicrobial Agent Targets F. nucleatum

    Long overlooked as a benign resident of the oral microbiome, Fusobacterium nucleatum has been increasingly linked to cancer progression, metastasis, and chemotherapeutic resistance. Now, researchers have unexpectedly uncovered a compound that may selectively take it out without collateral damage...
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