Contents

Current Gene Therapy, Volume 12 - Number 3

Editorial [Hot Topic: Therapeutic Approaches to Muscular Dystrophies]

, 12(3): 137 - 138

Aurelie Goyenvalle


DOI: 10.2174/156652312800840568




Gene Replacement Therapies for Duchenne Muscular Dystrophy Using Adeno-Associated Viral Vectors

, 12(3): 139 - 151

Jane T. Seto, Julian N. Ramos, Lindsey Muir, Jeffrey S. Chamberlain and Guy L. Odom


DOI: 10.2174/156652312800840603




Antisense Oligonucleotide-Mediated Exon Skipping for Duchenne Muscular Dystrophy: Progress and Challenges

, 12(3): 152 - 160

Virginia Arechavala-Gomeza, Karen Anthony, Jennifer Morgan and Francesco Muntoni


DOI: 10.2174/156652312800840621




Use of Cell-Penetrating-Peptides in Oligonucleotide Splice Switching Therapy

, 12(3): 161 - 178

Samir A. El Andaloussi, Suzan M. Hammond, Imre Mager and Matthew J.A. Wood


DOI: 10.2174/156652312800840612




Splicing Modulation Mediated by Small Nuclear RNAs as Therapeutic Approaches for Muscular Dystrophies

, 12(3): 179 - 191

Rachid Benchaouir and Aurelie Goyenvalle


DOI: 10.2174/156652312800840586




The Role of Stem Cells in Muscular Dystrophies

, 12(3): 192 - 205

Mirella Meregalli, Andrea Farini, Federica Colleoni, Letizia Cassinelli and Yvan Torrente


DOI: 10.2174/156652312800840559




Pharmacologically Targeting the Primary Defect and Downstream Pathology in Duchenne Muscular Dystrophy

, 12(3): 206 - 244

Rebecca J. Fairclough, Kelly J. Perkins and Kay E. Davies


DOI: 10.2174/156652312800840595




Interference with Myostatin/ActRIIB Signaling as a Therapeutic Strategy for Duchenne Muscular Dystrophy

, 12(3): 245 - 259

Helge Amthor and Willem M.H. Hoogaars


DOI: 10.2174/156652312800840577




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