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Tumour stress drives T cell exhaustion – antioxidants may reverse it
A new research study has discovered that targeted antioxidants could restore T cell function – offering a potential boost for cancer immunotherapies like CAR-T.
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Cell & gene therapy
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CAR T’s biggest hurdle: solving the toxicity problem
CAR T therapies are saving lives, but toxicities such as CRS and ICANS remain a major barrier. What will it take to overcome them?
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Scientists spotlight lung disease as fast-track model for ageing drugs
Researchers have identified idiopathic pulmonary fibrosis (IPF) as a powerful model for exploring treatments that target the biology of ageing.
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New 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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Key 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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Gene 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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New 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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Inside 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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Cholesterol 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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Kidney 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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New 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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Lupus 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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Turning 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.
