دانلود کتاب Muscle Gene Therapy

فهرست مطالب :

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