Dr. Howard Smith Oncall

Gene Therapy For Deaf Children

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Sinopsis

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 hea