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Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.

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Description scientific article published in November 2017
Author/s

author: Nicole K. Paulk  Romain Hardet  Amine Meliani  Francesco Puzzo  Giuseppe Ronzitti  Pasqualina Colella  Dwight D Koeberl  Maria G Biferi  Deeksha Bali  Federico Mingozzi  Marcelo Simon-Sola  Pascal Laforet 

Publication date November 1, 2017
Language English
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