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Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
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.
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.
13 October 2025 | By
A Synthetic DNA Approach for Speed, Scale & Flexibility
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.
CRISPR therapies depend on delivery and lipid nanoparticles are emerging as a more flexible and scalable option than viral vectors.
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.
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.
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.
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.
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.
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.
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.
Researchers at UNSW Sydney have unveiled a next-generation CRISPR tool that could lead to safer treatments for genetic disorders, including Sickle Cell.
29 July 2025 | By Eurofins Discovery
Facing roadblocks in obesity drug discovery? Discover how integrated, validated strategies are helping teams accelerate development and reduce risk.
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.
