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Gene therapy has allowed several children born with inherited deafness to hear. Globally, 34 million children have deafness or hearing loss, and genes are responsible for up to 60% of cases. Hereditary deafness is the latest condition scientists are targeting with gene therapy, which is already approved to treat illnesses such as sickle cell disease and severe hemophilia. Also, some people consider gene therapy for deafness ethically problematic. "This is real proof showing gene therapy is working,” Chen said.
Persons: , Zheng, Yi Chen, Dr, Yilai Shu, Chen, Eli Lilly, Akouos, he's, John Germiller, ” Germiller, , Lawrence Lustig, Teresa Blankmeyer Burke, ” Chen Organizations: Children's Hospital of Philadelphia, Fudan University, Shanghai Refreshgene Therapeutics, Regeneron Pharmaceuticals, Decibel Therapeutics, Columbia, Gallaudet University, Associated Press Health, Science Department, Howard Hughes Medical Institute’s Science, Educational Media Group, AP Locations: China, Shanghai, Philadelphia, Spain
Last year, after moving to Spain, his family took him to a hearing specialist, who made a surprising suggestion: Aissam might be eligible for a clinical trial using gene therapy. On Oct. 4, Aissam was treated at the Children’s Hospital of Philadelphia, becoming the first person to get gene therapy in the United States for congenital deafness. His is an extremely rare form, caused by a mutation in a single gene, otoferlin. Otoferlin deafness affects about 200,000 people worldwide. The goal of the gene therapy is to replace the mutated otoferlin gene in patients’ ears with a functional gene.
Persons: Aissam, ” Aissam, Organizations: Children’s Hospital of Philadelphia Locations: Aissam, Morocco, Spain, United States
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