Muscle and Heart Function Restoration in a Limb Girdle Muscular Dystrophy 2I (LGMD2I) Mouse Model by Systemic FKRP Gene Delivery-Reference-Cited by-同舟云学术

Muscle and Heart Function Restoration in a Limb Girdle Muscular Dystrophy 2I (LGMD2I) Mouse Model by Systemic FKRP Gene Delivery

Published:2014-11 Issue:11 Volume:22 Page:1890-1899
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Qiao Chunping,Wang Chi-Hsien,Zhao Chunxia,Lu Peijuan,Awano Hiroyuki,Xiao Bin,Li Jianbin,Yuan Zhenhua,Dai Yi,Martin Carrie Bette,Li Juan,Lu Qilong,Xiao Xiao

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference49 articles.

1. The muscular dystrophies;Emery;Lancet,2002

2. Post-translational disruption of dystroglycan-ligand interactions in congenital muscular dystrophies;Michele;Nature,2002

3. Refining genotype phenotype correlations in muscular dystrophies with defective glycosylation of dystroglycan;Godfrey;Brain,2007

4. Phenotypic spectrum associated with mutations in the fukutin-related protein gene;Mercuri;Ann Neurol,2003

5. Absence of post-phosphoryl modification in dystroglycanopathy mouse models and wild-type tissues expressing non-laminin binding form of α-dystroglycan;Kuga;J Biol Chem,2012

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