Children’s Hospital of Philadelphia (CHOP) Performs First in U.S. Gene Therapy Procedure to Treat Genetic Hearing Loss

Children’s Hospital of Philadelphia (CHOP) is proud to announce the initial results of an experimental gene therapy treatment of a patient with hereditary hearing loss in the United States. Findings to date indicate that the treatment was successful. While the gene involved is quite rare, the milestone represents a breakthrough in the treatment of patients around the world with hearing loss caused by dozens of different genetic mutations and marks another innovative move for gene and cell therapy in a new area of medicine."Gene therapy for hearing loss is something that we physicians and scientists in the world of hearing loss have been working toward for over 20 years, and it is finally here," said John A. Germiller, MD, PhD, an attending surgeon and Director of Clinical Research in the Division of Otolaryngology at CHOP, and Associate Professor at Perelman School of Medicine at the University of Pennsylvania. "While the gene therapy we performed in our patient was to correct an abnormality in one, very rare gene, these studies may open the door for future use for some of the over 150 other genes that cause childhood hearing loss."

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