作者：
Francesco,Puzzo [1]
;
Pasqualina,Colella [2]
;
Maria G,Biferi [1]
;
Deeksha,Bali [3]
;
Nicole K,Paulk [4]
;
Patrice,Vidal [5]
;
Fanny,Collaud [1]
;
Marcelo,Simon-Sola [3]
;
Severine,Charles [1]
;
Romain,Hardet [1]
;
Christian,Leborgne [3]
;
Amine,Meliani [1]
;
Mathilde,Cohen-Tannoudji [3]
;
Stephanie,Astord [1]
;
Bernard,Gjata [1]
;
Pauline,Sellier [3]
;
Laetitia,van Wittenberghe [3]
;
Alban,Vignaud [3]
;
Florence,Boisgerault [1]
;
Martine,Barkats [1]
;
Pascal,Laforet [3]
;
Mark A,Kay [1]
;
Dwight D,Koeberl [1]
;
Giuseppe,Ronzitti [1]
;
Federico,Mingozzi [3]
作者单位：
INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.
[1]
Department of Life Sciences and Biotechnology, University of Ferrara, Ferrara, Italy.
[2]
University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.
[3]
Biochemical Genetics Laboratory, Duke University Health System, Durham, NC 27710, USA.
[4]
Departments of Pediatrics and Genetics, Stanford University, Stanford, CA 94305, USA.
[5]
Paris-Est Neuromuscular Center, Pitié-Salpêtrière Hospital and Raymond Poincaré Teaching Hospital, Garches, APHP, Paris, France.
[6]
Division of Medical Genetics, Department of Pediatrics and Department of Molecular Genetics and Microbiology, Duke University School of Medicine, Durham, NC 27710, USA.
[7]
INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France. gronzitti@genethon.fr fmingozzi@genethon.fr.
[8]
DOI
10.1126/scitranslmed.aam6375
PMID
29187643
发布时间
2018-11-13
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