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Deaf Toddler Hears For The First Time After Pioneering Gene Therapy [Forbes]
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A toddler has been able to hear for the first time after being treated with a pioneering gene therapy for deafness. The girl who lives in the U.K. according to this article from the , has a hereditary mutation in a gene called otoferlin, an important part of allowing people to sense sound in the cells of the ear. Deafness caused by otoferlin mutations is very rare, accounting for less than 1% of all people who are deaf and only 30-50 people a year in the United States are diagnosed with otoferlin-related deafness. "This is a relatively rare form of deafness," said Dr. Lawrence Lustig, M.D., Chair of Columbia University's Department of Otolaryngology, who was presenting the work at the in Baltimore. "But because we have good cellular preservation in the cochlea, it gives us a wonderful opportunity to intervene in early childhood to try to rescue hearing loss before degeneration occurs," added Lustig who is an investigator on the clinical trial. The is run by biotechnology compan
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