Gene Therapy For Deaf Children

Vidcast:  https://www.instagram.com/p/DQdnYr9jZwV/An exciting, ground-breaking clinical study now shows that children born with a defective gene coding for a vital inner ear protein can have that gene  repaired and hearing restored. This phenomenally successful preliminary clinical trial was recently published in the New England Journal of Medicine.Genetic bioengineers at New York’s Regeneron Pharmaceuticals loaded a normal copy of the otoferlin gene into a dual adeno-associated virus acting as a Trojan horse.  Twelve children, born without the ability to synthesize otoferlin protein, received the gene injection, dubbed DB-OTO therapy, into their inner ears at 3 clinical centers: Harvard’s Mass. Eye and Ear Infirmary, UC San Diego’s Children’s Hospital, and University College London.  Otoferlin is necessary for the inner ear’s ability to convert sound vibrations into electrical impulses.  At 24 weeks post-injection, 9 of the 12 children, 75%, regained measurable hearing.  Three, 25%, developed near normal hearing.  The gene therapy was well-tolerated without any significant side effects.This gene therapy, with further refinement and after larger clinical trials, may be a one-and-done treatment for one common form of congenital deafness. Cochlear implants will continue to be essential therapy for other types of genetic and acquired severe hearing losses pending development of other genetic and/or chemical cochlear modifications.https://www.nejm.org/doi/full/10.1056/NEJMoa2400521#deafness #children #congenital #otoferlin #dboto