Gene therapy cures deafness

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Gene therapy has recently been used to successfully treat certain types of congenital deafness in children. In a groundbreaking development, an 11-year-old boy named Aissam Dam, who was born deaf due to a rare genetic abnormality, was able to hear for the first time following gene therapy treatment at the Children's Hospital of Philadelphia (CHOP)16. This treatment targeted a mutation in the OTOF gene, which is responsible for a form of deafness known as DFNB97. Additionally, a team co-led by Zheng-Yi Chen at the Massachusetts Eye and Ear Infirmary and Harvard Medical School conducted a study in Fudan, China, where six children aged 1 to 7 with the same inherited mutation of the OTOF gene received gene therapy. This approach has shown promising results, with the children gaining the ability to hear2. The success of these treatments provides hope for developing therapies for other types of genetic deafness, as there are over 150 genes known to cause congenital hearing loss68. The gene therapy works by delivering a corrected copy of the gene responsible for the condition, allowing the sensory cells in the inner ear to transmit sound signals to the brain8. While these treatments currently target a specific and rare condition, they open the possibility of treating a wider range of genetic hearing impairments in the future10. It's important to note that while these developments are significant, gene therapy for deafness is still in the experimental stages and is not yet widely available. The treatments have been successful in a limited number of cases, and further research is needed to determine their long-term efficacy and potential application to other forms of deafness267.
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