Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice-Reference-Cited by-同舟云学术

Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

Published:2010-11-30 Issue:4 Volume:20 Page:681-693
ISSN:1460-2083
Container-title:Human Molecular Genetics
language:en
Short-container-title:

Author:

Dominguez Elisa,Marais Thibaut,Chatauret Nicolas,Benkhelifa-Ziyyat Sofia,Duque Sandra,Ravassard Philippe,Carcenac Romain,Astord Stéphanie,de Moura Aurélie Pereira,Voit Thomas,Barkats Martine

Publisher

Oxford University Press (OUP)

Subject

Genetics(clinical),Genetics,Molecular Biology,General Medicine

Link

http://academic.oup.com/hmg/article-pdf/20/4/681/17254332/ddq514.pdf

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4. Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2;Mailman;Genet. Med.,2002

5. Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy;Azzouz;J. Clin. Invest.,2004

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