Duchenne muscular dystrophy and dystrophin: pathogenesis and opportunities for treatment
Author:
Affiliation:
1. MRC Functional Genetics Unit, University of Oxford South Parks Road Oxford OX1 3QX UK
Publisher
EMBO
Subject
Genetics,Molecular Biology,Biochemistry
Link
https://onlinelibrary.wiley.com/doi/pdf/10.1038/sj.embor.7400221
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1. Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients
2. Antisense-Induced Multiexon Skipping for Duchenne Muscular Dystrophy Makes More Sense
3. Negamycin Restores Dystrophin Expression in Skeletal and Cardiac Muscles of mdx Mice
4. Systemic delivery of human microdystrophin to regenerating mouse dystrophic muscle by muscle progenitor cells
5. LARGE can functionally bypass α-dystroglycan glycosylation defects in distinct congenital muscular dystrophies
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