Doctors in the UK are claiming to have performed a monumental medical breakthrough. Using a form of gene therapy, they successfully treated—for the first time—the neurodegenerative disorder Huntington’s disease. On Wednesday, the company uniQure announced the results of its phase I/II trial testing the therapy, code-named AMT-130. The trial, conducted with the help of researchers […]
Deaf Teenager and 24-Year-Old Gain Ability to Hear After Experimental Gene Therapy
In recent years, gene therapy has emerged as a promising treatment for genetic forms of congenital deafness—hearing loss that’s present at birth—in children. A new study shows that gene therapy can bestow hearing in teenagers and adults with this condition, too. The study, published Wednesday in the journal Nature Medicine, recruited patients with deafness or […]