All Cell & Gene Therapy articles – Page 4
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ArticleAn ethical shift in NHP research: iPSC-derived cardiomyocytes for safer pharmacology
As regulators move to phase out primate testing, NHP-derived iPSC cardiomyocytes are emerging as a scalable, ethical and scientifically robust alternative - offering drug developers a clear path to faster, more predictive, and more responsible innovation.
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NewsKidney organoids show APOL1 gene’s role in chronic kidney disease
New research using stem cell-derived kidney organoids reveals how APOL1 gene mutations disrupt mitochondrial function in kidney cells – potentially leading to new targeted treatments.
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NewsNew CAR T therapy targets solid tumours safely and effectively
USC researchers have engineered CAR T cells to deliver a dual protein therapy that targets solid tumours – offering hope for cancers previously resistant to treatment.
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ArticleFast-tracking advanced therapies without compromising regulatory success
Early planning for potency CQAs, comparability and evolving global regulations can set advanced therapies on the fastest path to approval. Dr Christian Schneider shares how to prepare from the start to accelerate development without compromising the evidence regulators expect.
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NewsLab-grown kidneys show real function in new stem cell study
USC Stem Cell scientists have created the most advanced lab-grown kidney structures to date, combining key components into ‘assembloids’ that mature and function like real kidneys, which could open up new possibilities for modelling complex kidney diseases.
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NewsAnti-linker antibodies: a universal key for CAR-T detection
Every new CAR-T needs a new detection tool – until now. Anti-linker antibodies could change how researchers develop and track these therapies.
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NewsNew stem cell approach could repair stroke-damaged brains
A new experimental stem cell therapy shows promise in repairing brain damage after ischemic strokes – potentially allowing for the development of future treatments that extend the recovery window.
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ArticleBetter assays: the key step in moving drugs from lab to clinic
From gene therapy to Long Covid, better assays are helping researchers move promising drug candidates from early studies into clinical trials. Dr Alexandre Lucas explains the technologies, challenges and innovations driving this progress.
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NewsTumour 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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ArticleCAR 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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NewsScientists 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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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.