All Gene Therapy articles – Page 2
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ArticleResetting retinal networks with a gene-agnostic approach
What if treating blindness did not mean fixing a single faulty gene? Find out how modifier gene therapy is designed to protect vision by targeting broader retinal pathways.
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ArticleCircular RNA technology: the future of gene therapy
Pioneering circular RNA could redefine what the future of gene therapy looks like. Erik Digman Wiklund, CEO of Circio, shares how his company’s platform is enhancing gene expression and tackling toxicity challenges through smarter design and scientific collaboration.
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NewsNew mRNA therapy could inform future male infertility treatments
Scientists have restored sperm production in mice with a genetic form of male infertility using mRNA delivered via lipid nanoparticles, with the hope of informing future treatments for non-obstructive azoospermia.
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ArticleSPNS1 mutations reveal new lysosomal lipid recycling pathway
Scientists have linked rare mutations in SPNS1 to a previously unknown lipid recycling pathway in lysosomes, revealing how faulty fat processing can trigger muscle and liver disease.
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ArticleStreamlining Biomanufacturing of Personalised Cancer Immunotherapies with Synthetic DNA
A Synthetic DNA Approach for Speed, Scale & Flexibility
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NewsNew gene therapy restores brain function in SYNGAP1 disorder
Scientists have developed a new gene therapy that reversed symptoms of SYNGAP1-related brain disorders in mice, which could lead to new treatments for this group of neurological conditions.
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ArticleAdvancing gene editing: the role of lipid nanoparticles in CRISPR delivery
CRISPR therapies depend on delivery and lipid nanoparticles are emerging as a more flexible and scalable option than viral vectors.
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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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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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News
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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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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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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ArticleThe future of CNS drug development: signs of real progress
New therapeutic approaches are emerging for CNS disorders – but can they overcome the toughest barriers in drug development? Find out what is driving progress and what still stands in the way.
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Gene therapy boosts CAR-T power against glioblastoma
Researchers at SR-TIGET in Milan have developed a novel gene therapy approach that supercharges CAR-T cell therapy against glioblastoma – strengthening treatment against one of the world’s deadliest brain cancers.
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WhitepaperTransform your drug discovery pipeline with 3D cellular models
Expert insights reveal how 3D cellular models are revolutionising preclinical testing and clinical translation.