Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes
Pasqualina Colella(Stanford Medicine), Federico Mingozzi(Spark Therapeutics (United States)), Laetitia van Wittenberghe(Genethon (France)), Mark A. Kay(Io Therapeutics (United States)), Pascal Laforêt(Assistance Publique – Hôpitaux de Paris), Romain Hardet(Inserm), Fanny Collaud(Inserm), Dwight D. Koeberl(Duke Medical Center), Giuseppe Ronzitti(Inserm), Pierre Vidal(University of Reunion Island), Christian Leborgne(Inserm), Maria Grazia Biferi(Inserm), Pauline Sellier(Inserm), Deeksha Bali(MRC Epidemiology Unit), Marcelo Simon‐Sola(Inserm), Francesco Puzzo(Inserm), M. Barkats(Inserm), Séverine Charles(Inserm), Florence Boisgérault(Inserm), Nicole K. Paulk(University of California, San Francisco)
Cited by 1
Related Papers
TFEB Links Autophagy to Lysosomal Biogenesis
|Science|2011|3.3k
Mutations in dynamin 2 cause dominant centronuclear myopathy
|Nature Genetics|2005|462
Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys
|Science Translational Medicine|2013|346