WASHINGTON DC: A new study in mice suggests that replacing a dysfunctional gene may improve survival in some people with arrhythmogenic right ventricular cardiomyopathy (ARVC), a rare inherited disorder in which the heart’s muscular walls gradually weaken, putting patients at risk of dangerous irregular heartbeats.The investigational treatment targets the loss of function of a gene … Read more
Gene therapy has allowed several children born with inherited deafness to hear. A small study published Wednesday documents significantly restored hearing in five of six kids treated in China. On Tuesday, the Children’s Hospital of Philadelphia announced similar improvements in an 11-year-old boy treated there. And earlier this month, Chinese researchers published a study showing … Read more
Gautam Dongre’s two children in India and Pascazia Mazeze’s son in Tanzania live with an inherited blood disorder that turns blood cells into instruments of pain. Now that new gene therapies promise a cure for their sickle cell disease, Dongre said he’s “praying the treatment should come to us.” But experts say the one-time treatment … Read more
