Muscle Gene Therapy

Front Matter ....Pages i-xvi
Front Matter ....Pages 1-1
 An Overview of Muscle Biology and Physiology for Muscle Gene Therapy  (Paul M. L. Janssen, Jonathan P. Davis)....Pages 3-12
 Molecular Basis of Muscle Disease  (Ning Liu, Rhonda Bassel-Duby)....Pages 13-39
 Animal Models for Muscle Disease and Muscle Gene Therapy  (Stephanie M. Shrader, Roberta Wrighten, Bruce F. Smith)....Pages 41-63
 Muscle Stem Cell Biology and Implications in Gene Therapy  (Terence Partridge)....Pages 65-80
 Pluripotent Stem Cells for Gene Therapy of Hereditary Muscle Disorders  (Thierry VandenDriessche, Yoke Chin Chai, Dimitri Boon, Marinee K. Chuah)....Pages 81-97
 MicroRNAs (miRs) in Muscle Gene Therapy  (Alessio Rotini, Giorgia Giacomazzi, Ester Sara Di Filippo, Maurilio Sampaolesi)....Pages 99-119
 Immune System Regulation of Muscle Injury and Disease  (Jenna M. Kastenschmidt, Ali H. Mannaa, Karissa J. Muñoz, S. Armando Villalta)....Pages 121-139
 Design of Muscle Gene Therapy Expression Cassette  (Yi Lai, Dongsheng Duan)....Pages 141-156
 Non-viral Vector for Muscle-Mediated Gene Therapy  (Serge Braun)....Pages 157-178
 Viral Vectors for Muscle Gene Therapy  (Dan Wang, Alexander Brown, Guangping Gao)....Pages 179-192
 Development of Next-Generation Muscle Gene Therapy AAV Vectors  (Michael E. Nance, Dongsheng Duan)....Pages 193-206
 Histological and Biochemical Evaluation of Muscle Gene Therapy  (Michael W. Lawlor, Joel S. Schneider, Martin K. Childers, Kristy J. Brown)....Pages 207-226
 Optical Polarization Tractography Imaging of Structural Changes in the Skeletal and Cardiac Muscles of the mdx4cv Mice  (Gang Yao)....Pages 227-237
 Biomarkers for Muscle Disease Gene Therapy  (Yetrib Hathout, Kristy J. Brown, Kanneboyina Nagaraju, Eric P. Hoffman)....Pages 239-252
 Large-Scale Clinical Manufacturing of AAV Vectors for Systemic Muscle Gene Therapy  (Nathalie Clément)....Pages 253-273
 Genome Editing for Muscle Gene Therapy  (Alan O’Brien, Ronald D. Cohn)....Pages 275-287
Front Matter ....Pages 289-289
 Considerations on Preclinical Neuromuscular Disease Gene Therapy Studies  (Dongsheng Duan)....Pages 291-326
 Gene Replacement Therapy for Duchenne Muscular Dystrophy  (Katrin Hollinger, Julie M. Crudele, Jeffrey S. Chamberlain)....Pages 327-337
 Recent Advances in AON-Mediated Exon-Skipping Therapy for Duchenne Muscular Dystrophy  (Xianjun Gao, Gang Han, HaiFang Yin)....Pages 339-354
 AAV-Mediated Exon Skipping for Duchenne Muscular Dystrophy  (Rachid Benchaouir, Aurelie Goyenvalle)....Pages 355-370
 Alternate Translational Initiation of Dystrophin: A Novel Therapeutic Approach  (Nicolas Wein, Kevin M. Flanigan)....Pages 371-382
 Genome Editing for Duchenne Muscular Dystrophy  (Christopher E. Nelson, Charles A. Gersbach)....Pages 383-403
 Sarcolipin Knockdown Therapy for Duchenne Muscular Dystrophy  (Satvik Mareedu, Shalini Dwivedi, Nandita Niranjan, Gopal J. Babu)....Pages 405-415
 Gene Therapy for Central Nervous System in Duchenne Muscular Dystrophy  (Cyrille Vaillend, Faouzi Zarrouki, Ophélie Vacca)....Pages 417-438
 Therapeutic Approaches for Dysferlinopathy in Animal Models  (William Lostal, Isabelle Richard)....Pages 439-451
 Muscle Cell Membrane Repair and Therapeutic Implications  (Renzhi Han)....Pages 453-467
 Dystroglycanopathy Gene Therapy: Unlocking the Potential of Genetic Engineering  (Charles H. Vannoy, Anthony Blaeser, Qi L. Lu)....Pages 469-490
 RNAi Therapy for Dominant Muscular Dystrophies and Other Myopathies  (Scott Q. Harper)....Pages 491-507
 Gene Therapy for Facioscapulohumeral Muscular Dystrophy (FSHD)  (Daniel G. Miller)....Pages 509-524
 Gene Therapy and Gene Editing for Myotonic Dystrophy  (Marinee Chuah, Yoke Chin Chai, Sumitava Dastidar, Thierry VandenDriessche)....Pages 525-548
 Gene Therapy for Oculopharyngeal Muscular Dystrophy  (Alberto Malerba, Fanny Roth, Vanessa Strings, Pradeep Harish, David Suhy, Capucine Trollet et al.)....Pages 549-564
 Gene Therapy for X-Linked Myotubular Myopathy  (Jean-Baptiste Dupont, Michael W. Lawlor, Martin K. Childers)....Pages 565-577
 Preclinical Gene Therapy Studies for Metabolic Myopathy  (Stephanie Salabarria, Barry J. Byrne, Cristina Liberati, Manuela Corti)....Pages 579-596
 Elimination of Mutant Mitochondrial DNA in Mitochondrial Myopathies Using Gene-Editing Enzymes  (Sandra R. Bacman, Carlos T. Moraes)....Pages 597-620
 Gene Therapy for CMT Inherited Neuropathy  (Kleopas A. Kleopa, Alexia Kagiava, Irene Sargiannidou)....Pages 621-644
 Muscle as a Potent Target in Vaccination  (Axel Rossi, Hildegard Büning)....Pages 645-659
Front Matter ....Pages 661-661
 Patient and Family Perspective on Muscle Gene Therapy  (Pat Furlong)....Pages 663-666
 Design of Clinical Trials for Gene Therapy in Muscular Dystrophy  (Jorge Quiroz, Kathryn Wagner)....Pages 667-680
 Path to Clinical Trials: Trial Design, Development of the Clinical Product, and Safety Concerns in the Implementation of Clinical Trials  (Jerry R. Mendell, Louise R. Rodino-Klapac, Christopher J. Shilling)....Pages 681-698
 Muscle MRI as an Endpoint in Clinical Trials  (Dirk Fischer, Ulrike Bonati, Mike P. Wattjes)....Pages 699-708
 Gene Therapy Clinical Trials for Duchenne and Limb Girdle Muscular Dystrophies: Lessons Learned  (Jerry R. Mendell, Louise R. Rodino-Klapac, Christopher Walker)....Pages 709-725
 Duchenne Muscular Dystrophy Exon-Skipping Trials  (Jerry R. Mendell, Zarife Sahenk, Louise R. Rodino-Klapac)....Pages 727-743
 What We Have Learned from 10 Years of DMD Exon-Skipping Trials  (Svitlana Pasteuning-Vuhman, Annemieke Aartsma-Rus)....Pages 745-758
 Clinical Gene Therapy Trials for Pompe Disease  (Cristina Liberati, Stephanie Salabarria, Manuela Corti, Barry J. Byrne)....Pages 759-774
 Muscle-Directed Gene Therapy for Alpha-1 Antitrypsin Deficiency  (Alisha M. Gruntman, Terence R. Flotte)....Pages 775-786
Back Matter ....Pages 787-811