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Gene therapy found safe in trial of Pompe disease treatment

Posted on: 22 February 2018, source: UPI news
The first human trial of gene therapy to treat respiratory problems in early-onset Pompe disease of children was found safe, according to health researchers in Florida. University of Florida Health scientists conducted a successful trial of nine participants who had the incurable disease, which damages muscle and nerve cells throughout the body. Results of the treatment, which utilizes an adeno-associated virus, were published in the journal Human Gene Therapy Clinical Development.