Contents

Current Gene Therapy, Volume 15 - Number 4

Editorial (Thematic Issue):Stem Cell-Based and Gene Therapy for Hereditary Muscle Disorders

, 15(4): 327 - 328

Marinee K. Chuah


DOI: 10.2174/156652321504150722121542




Therapeutic Approaches for Dominant Muscle Diseases: Highlight on Myotonic Dystrophy

, 15(4): 329 - 337

A. F. Klein, S. Dastidar, D. Furling and M. K. Chuah


DOI: 10.2174/1566523215666150630120537




Preclinical Development of New Therapy for Glycogen Storage Diseases

, 15(4): 338 - 347

Baodong Sun, Elizabeth D. Brooks and Dwight D. Koeberl


DOI: 10.2174/1566523215666150630132253




Adult Stem Cells and Skeletal Muscle Regeneration

, 15(4): 348 - 363

Domiziana Costamagna, Emanuele Berardi, Gabriele Ceccarelli and Maurilio Sampaolesi


DOI: 10.2174/1566523215666150630121024




Pluripotent Stem Cells for Gene Therapy of Degenerative Muscle Diseases

, 15(4): 364 - 380

Mariana Loperfido, Heather B. Steele-Stallard, Francesco Saverio Tedesco and Thierry VandenDriessche


DOI: 10.2174/1566523215666150630121207




The Skeletal Muscle Environment and Its Role in Immunity and Tolerance to AAV Vector-Mediated Gene Transfer

, 15(4): 381 - 394

Florence Boisgerault and Federico Mingozzi


DOI: 10.2174/1566523215666150630121750




Adeno-Associated Virus (AAV) Mediated Dystrophin Gene Transfer Studies and Exon Skipping Strategies for Duchenne Muscular Dystrophy (DMD)

, 15(4): 395 - 415

Klaudia Kawecka, Michael Theodoulides, Yalin Hasoglu, Susan Jarmin, Hanna Kymalainen, Anita Le-Heron, Linda Popplewell, Alberto Malerba, George Dickson and Takis Athanasopoulos


DOI: 10.2174/1566523215666150710123830




Successful Combination of Sequential Gene Therapy and Rescue Allo-HSCT in Two Children with X-CGD - Importance of Timing

, 15(4): 416 - 427


DOI: 10.2174/1566523215666150515145255




Gene Manipulation of Human Embryonic Stem Cells by In Vitro-Synthesized mRNA for Gene Therapy

, 15(4): 428 - 435

Xiao Li Wang, Li Yu, Yan Ding, Xing Rong Guo, Ya Hong Yuan and Dong Sheng Li


DOI: 10.2174/1566523215666150515144533




A Simple High Efficiency Intra-Islet Transduction Protocol Using Lentiviral Vectors

, 15(4): 436 - 446

Carmen Maria Jimenez-Moreno, Irene de Gracia Herrera-Gomez, Livia Lopez-Noriega, Petra Isabel Lorenzo, Nadia Cobo-Vuilleumier, Esther Fuente-Martin, Jose Manuel Mellado-Gil, Geraldine Parnaud, Domenico Bosco, Benoit Raymond Gauthier and Alejandro Martin-Montalvo


DOI: 10.2174/1566523215666150630121557




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