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Gene Therapy Sounds and Looks Promising for Inherited Deafness and Blindness
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作者 Jennifer Welsh 《Engineering》 2025年第7期9-12,共4页
At a February 2025 meeting,researchers presented their latest findings from a phase 1/2 clinical trial of a gene therapy,DB-OTO,developed by Regeneron Pharmaceuticals(Tarrytown,NY,USA)for a particular type of profound... At a February 2025 meeting,researchers presented their latest findings from a phase 1/2 clinical trial of a gene therapy,DB-OTO,developed by Regeneron Pharmaceuticals(Tarrytown,NY,USA)for a particular type of profound hearing loss in children.Nearly all the 12 participants,with no serious adverse events,experienced clinically meaningful and durable hearing improve-ments[1].A toddler born deaf,for example,could hear well enough after treatment to quack when asked,“What sound does a duck make?”[2]. 展开更多
关键词 gene therapydb otodeveloped profound hearing loss gene therapy hearing loss Regeneron Pharmaceuticals BLINDNESS phase clinical trial db oto
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