A Phase 2/3 trial assessing a gene therapy for X-linked retinitis pigmentosa (XLRP) shows significant improvements in low-light vision...
Dr Tom Turner explores Professor Jennifer Doudna's reflections on the discovery of CRISPR and the future of genome editing in this conversation, which examines one of the most transformative advances in modern biology…
The US Food and Drug Administration (FDA) has put a clinical hold on an experimental gene therapy for Hunter syndrome due to nodules being discovered in the spine of five patients who previously received treatment...
Gene therapy designed to deliver a healthy copy of the ALPK3 gene can restore heart function, raising the possibility of a future treatment for some forms of inherited cardiomyopathy...
A six-year-old girl has died following an experimental genome editing treatment, prompting an investigation into the clinical trial by the university involved...
A new personalised genetic therapy has shown early success in treating two children with a rare genetic epilepsy syndrome...
An 11-year-old girl has become the first UK patient to receive an experimental gene therapy for an inherited eye disorder...
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