All Cell & Gene Therapy articles – Page 4
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NewsNew nanostructure makes CRISPR edits safer and three times faster
Northwestern University researchers have created DNA-coated nanoparticles that deliver CRISPR into cells three times more effectively while reducing toxicity. The advance could overcome one of the biggest barriers to gene-editing therapies.
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NewsKey newborn liver cells could boost paediatric gene therapy
A small subset of newborn liver cells – known as clonogenic hepatocytes – drives over 90 percent of adult liver growth. New research shows how targeting these cells early could improve the effectiveness and durability of paediatric gene therapies.
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Scientists chart ovarian reserve to help advance new infertility treatments
UCLA scientists have created the first detailed map of how the ovarian reserve forms in primates, offering new insights – and potential new treatments – for infertility, polycystic ovary syndrome (PCOS) and hormone-related conditions.
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ArticleGene therapies that listen and respond: the power of RNA regulation
Most gene therapies rely on static DNA promoters to control gene activity, but nature uses far more sophisticated tools. Dr Matthew Dale explores how harnessing RNA-level control could enable treatments that sense and respond in real time, offering unprecedented precision and safety.
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NewsNew M13 virus method could change future genetic treatments
Researchers at the University of Waterloo have developed a modified bacterial virus that can deliver gene therapies cheaper and more efficiently – moving us closer toward personalised, affordable genetic medicine.
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ArticleInside the Alzheimer’s study backing stem cells, not drugs
In this first-in-human Alzheimer’s study, Wnt-activated autologous stem cells are delivered intracerebroventricularly (directly into the brain) to address neuronal loss, while also reducing amyloid and tau biomarkers and improving cognition. Early data from this regenerative approach could help early drug discovery teams shape target selection, biomarker development and trial design.
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NewsCholesterol drug restores brain barrier function in 22q11.2 syndrome models
Researchers have found that mitochondrial dysfunction in the blood-brain barrier may drive neuropsychiatric disease in 22q11.2 deletion syndrome – and that a cholesterol drug could restore barrier function and ease symptoms.
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NewsKidney organoids reveal hidden toxicities in AAV gene therapy
Stem cell-derived kidney organoids have revealed hidden toxicities in adeno-associated virus (AAV) gene therapy delivery – offering a powerful new way to improve the safety of future treatments.
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NewsNew CRISPR breakthrough could transform sickle cell treatment
Researchers at UNSW Sydney have unveiled a next-generation CRISPR tool that could lead to safer treatments for genetic disorders, including Sickle Cell.
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ArticleLupus reimagined: targeting the cause, not just the symptoms
Engineered cell therapies are offering a potential new way to treat lupus – not by suppressing symptoms, but by reprogramming the immune system itself. For the first time, lasting remission looks like a real possibility.
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Targeted microglia transplant reverses signs of rare brain disorder
Stanford Medicine researchers have developed a targeted brain cell transplant that replaced most diseased microglia in mice with Sandhoff disease – extending their lifespan and reversing symptoms
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WebinarOvercoming Affinity and Expression Bottlenecks in TCR Discovery
This expert-led webinar discusses how to break through common bottlenecks in TCR discovery with practical strategies that help teams move faster and smarter.
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NewsTurning up the heat: simple temperature change makes versatile vaccine nanoparticles
University of Chicago researchers have developed a scalable nanoparticle platform that self-assembles with just a temperature change – enabling safe, solvent-free delivery of proteins and RNA for vaccines and therapies.
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NewsFuelling immunity: glucose helps T cells fight cancer better
A new study from the Van Andel Institute shows that glucose not only fuels T cells but also strengthens their internal signalling and cancer-fighting capabilities –offering a potential route to improved immunotherapies.
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NewsNew research shows squeezing cancer cells helps them survive
Scientists have discovered that cancer cells generate an instant burst of energy when physically squeezed, helping them repair DNA damage and survive harsh environments. This discovery means that therapies targeting this new survival mechanism could be developed.
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ArticleManaging CGT trials: the role of IRT from discovery to clinical development
Discover how interactive response technology (IRT) is revolutionising the management of cell and gene therapy (CGT) trials by streamlining complex workflows, ensuring regulatory compliance and enhancing patient outcomes.
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WebinarInnovating Obesity Drug Discovery: Trends, Challenges, and Translational Strategies
Facing roadblocks in obesity drug discovery? Discover how integrated, validated strategies are helping teams accelerate development and reduce risk.
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ArticlePlasmids: Tackling Supply Chain and Manufacturing Challenges
Gene therapy’s progress depends on reliable supply chains and efficient manufacturing. In this episode, we explore the challenges and strategies involved in plasmid production - an essential component in advancing life-changing therapies.
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NewsSmart nanogels help CAR-T cells penetrate tumours
Researchers in China have developed a next-generation CAR-T cell therapy armed with nanogels that can penetrate the dense barriers of solid tumours. This addresses a major limitation in the treatment of solid cancers with immunotherapy.
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NewsAutoimmune breakthrough: Egr-1 regulates treg cells in diseases like MS
Scientists have found a key mechanism driving immune regulation in autoimmune diseases like MS and IBD – which could lead to new targeted treatments.