Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression
Patrice Vidal(Inserm), Federico Mingozzi(Spark Therapeutics (United States)), Séverine Charles(Inserm), Louisa Jauze(Inserm), Serena Pagliarani(University of Milan), Sabrina Lucchiari(University of Milan), Laetitia van Wittenberghe(Genethon (France)), Alban Vignaud(Genethon (France)), Pascal Laforêt(Assistance Publique – Hôpitaux de Paris), Giacomo P. Comi(University of Milan), Helena Costa Verdera(Spark Therapeutics (United States)), Edoardo Malfatti(Hôpitaux Universitaires Henri-Mondor), Fabienne Rajas(Université Claude Bernard Lyon 1), Fanny Collaud(Inserm), Giuseppe Ronzitti(Inserm), Solenne Marmier(Inserm), Bernard Gjata(Inserm), Pasqualina Colella(Stanford Medicine), Marcelo Simon Sola(Inserm), Francesco Puzzo(Inserm), Gilles Mithieux(Université Claude Bernard Lyon 1), Monika Gjorgjieva(Lyon 1 Université)
Cited by 37
Related Papers
TFEB Links Autophagy to Lysosomal Biogenesis
|Science|2011|3.3k
Brain energy rescue: an emerging therapeutic concept for neurodegenerative disorders of ageing
|Nature Reviews Drug Discovery|2020|982
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