The outcomes of an international Phase III clinical trial conducted by researchers from the University of Pennsylvania Perelman School of Medicine and a multicenter group of investigators, published in the New England Journal of Medicine, show that adults with haemophilia B experienced an average 71% reduction in the number of […]
Gene Therapy
A Phase 1/2 trial included 15 individuals in total, among them three pediatric patients. The patients all suffered from the disease Leber congenital amaurosis caused by a gene that is essential in making proteins crucial to vision.
The protein dystrophin, absent in people with muscular dystrophy, will subsequently begin to be produced by the additional genetic code.
Researchers at the University of Iowa, the Broad Institute of MIT, Harvard, and Harvard have now created a gene-editing method that effectively fixes the most prevalent mutation affecting 85% of patients
Delivering a healthy copy of a gene to a person whose gene(s) are malfunctioning is known as gene therapy.
