Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy-Reference-Cited by-同舟云学术

Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy

Published:2016 Issue: Volume:3 Page:16060
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Armbruster Nicole,Lattanzi Annalisa,Jeavons Matthieu,Van Wittenberghe Laetitia,Gjata Bernard,Marais Thibaut,Martin Samia,Vignaud Alban,Voit Thomas,Mavilio Fulvio,Barkats Martine,Buj-Bello Ana

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference61 articles.

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5. Identification and characterization of a spinal muscular atrophy-determining gene;Lefebvre;Cell,1995

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