Bioethics News

Gene Therapy for Rare Sanfilippo Disease to Be Tested in Humans

May 12, 2016

(UPI) – Researchers in Britain are preparing to launch a clinical trial for treatment of the rare Sanfilippo disease, a genetic disorder that manifests in early childhood and often results in death before adulthood. Manchester University researchers are working with the company Orchard Therapeutics to develop a gene therapy treatment involving a patients’ own stem cells to correct the single gene mutation, with plans to start a trial with humans soon.

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