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Stem cell gene therapy for Sanfilippo disease ready for human trial

University of Manchester and Central Manchester University Hospital NHS Foundation Trust (CMFT) researchers are to work with Orchard Therapeutics to test a stem cell gene therapy for Sanfilippo disease in a human trial.

Click on this link for more information.

source: http://www.europeanpharmaceuticalreview.com/41054/news/industry-news/stem-cell-therapy-sanfilippo-disease/

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