Gene therapy restores hearing in deaf mice… down to a whisper stat : Yahoo

"Outer hair cells amplify sound, allowing inner hair cells to send a stronger signal to the brain. BOSTON, Feb. 6, 2017 /PRNewswire-USNewswire/ -- In the summer of 2015, a team at Boston Children's Hospital and Harvard Medical School reported restoring rudimentary hearing in genetically deaf mice using gene Therapy. Now the Boston Children's research team reports restoring a much higher level of hearing — down to 25 decibels, the equivalent of a whisper — using an improved gene Therapy vector developed at Massachusetts Eye and Ear. Eye and Ear's Grousbeck Gene Therapy Center. Ushering in gene Therapy for deafnessGéléoc and colleagues at Boston Children's Hospital studied mice with a mutation in Ush1c, the same mutation that causes Usher type 1c in humans.




Caroline CaseyA novel gene therapy technique has partially restored hearing and balance in deaf mice. As with the standard gene therapy, the virus contains a copy of the missing or faulty gene responsible for the mice's deafness. The research team acknowledge that gene therapy for deafness in humans is many years away, but they suggest this study is 'an important proof of principle'. Nine out of 12 mice treated showed signs of improved hearing, and four mice could hear the equivalent of conversation in a loud restaurant, approximately 70 to 80 decibels. Mice that were completely deaf because they lacked a critical hair cell gene were able to hear the equivalent of a loud conversation after the treatment.


This orderly structure falls apart in the mutant mice (middle column), but is dramatically restored after gene therapy treatment. Mice suffering from a rare genetic hearing disorder, Usher syndrome, have had their hearing and balance restored after researchers used a virus to safely deliver a mutated gene to their inner ear. The virus was a synthetic variant of the adeno-associated virus, a small virus that infects humans without causing disease. Reintroduction of the normal copy of the gene in the ear of newborn mice lead to a recovery of balance and hearing function. This work is a proof of principal that such therapy could be used as a treatment for hearing loss.



collected by :Lucy William

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