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Gene Therapy

5 GoKawiil briefs on this topic

Life Biosciences begins human trial of Yuancheng Lu's age-reversal gene therapy for glaucoma

Geneticist Yuancheng Lu, working in David Sinclair's Harvard lab, developed a reprogramming gene therapy in 2018 that regenerated crushed optic nerves in mice and restored their vision. That same therapy, now called ER-100, has been adapted for humans by the startup Life Biosciences, which in June injected it into the eye of a glaucoma patient as part of a clinical trial.

Deaths of two children in Chinese gene-therapy trials raise oversight concerns

Two children died in separate gene-editing clinical trials in China, prompting scrutiny from researchers, ethicists and legal scholars over how such studies are approved and monitored. Investigators say the trials involved ethical breaches that could carry lasting consequences for China's biomedical research sector.

Deaths of two children in Chinese gene-therapy trials spark scrutiny of oversight

Two children died in separate Chinese gene-editing trials — a boy in a 2025 trial by HuidaGene Therapeutics and a six-year-old girl in March 2024 after treatment developed by researchers at Shanghai Jiao Tong University and Xinhua Hospital. Shanghai Jiao Tong University has launched an investigation into the girl's case and a related study published in Nature, while HuidaGene disclosed the boy's death publicly in August.

CRISPR gene-editing therapy sustains 50% cholesterol drop for a full year, Cleveland Clinic finds

An updated study published in the New England Journal of Medicine shows that a one-time CRISPR-Cas9 gene-editing infusion kept LDL cholesterol and triglycerides roughly 50% lower a full year after treatment in patients who received the highest dose. The follow-up builds on a small November 2025 pilot trial of 15 patients with medication-resistant high cholesterol, testing five dosage levels for safety, with researchers now confirming the effect appears durable rather than temporary.

Chinese researchers create injectable muscle grafts that self-contract under the skin

Scientists in China developed lab-grown muscle tissue, called myografts, that can be injected subcutaneously and form vascularized structures that continuously contract on their own. In mouse studies published in Nature Aging, the grafts improved overall muscle mass, metabolic function and regenerative outcomes in aging and obese animals.