1

Animal Models for Inherited Muscle Diseases   Bruce F. Smith and Roberta Wrighten

1

2

In Utero Muscle Gene Transfer   Bhanu Munil Koppanati and Paula R. Clemens

23

3

Gene Therapy for the Respiratory Muscles   Gawiyou Danialou and Basil J. Petrof

41

4

Muscular Dystrophy Gene Therapy in Small Animal Models   Chunping Qiao and Xiao Xiao

55

5

Antisense-Mediated Exon Skipping for Duchenne Muscular Dystrophy   Annemieke Aartsma-Rus and Gert-Jan B Van Ommen

69

6

Systemic Treatment of Duchenne Muscular Dystrophy by Antisense Oligomer-Induced Exon Skipping   Qi Long Lu and Bo Wu

85

7

Rnai Therapy for Dominant Muscular Dystrophies and Other Myopathies   Lindsay M. Wallace, Sara E. Garwick, and Scott Q. Harper

99

8

Combinatorial Gene Therapy Strategies for Treating Muscular Dystrophies   Catherine E. Winbanks and Paul Gregorevic

117

9

Duchenne Cardiomyopathy Gene Therapy   Jin-Hong Shin, Brian Bostick, Deborah M. Fine, Yongping Yue, and Dongsheng Duan

141

10

Systemic Gene Delivery for Muscle Gene Therapy   Dilip Garikipati and Jeffrey S. Chamberlain

163

11

Modulating Immune Responses in Muscle Gene Therapy   Valder R. Arruda

181

12

Delivering Large Therapeutic Genes for Muscle Gene Therapy   Yi Lai, Yongping Yue, Brian Bostick, and Dongsheng Duan

205

13

Muscle as a Metabolic Factory for Gene Therapy   Cathryn S. Mah, Christina A. Pacak, and Barry J. Byrne

219

14

Muscle as a Target for Genetic Vaccine   Yan Zhi and Guangping Gao

231

15

Combining Stem Cells and Exon Skipping Strategy to Treat Muscular Dystrophy   Mirella Meregalli, Andrea Farini, and Yvan Torrente

249

16

Gene Therapy Clinical Trials for Muscular Dystrophies   Dominic J. Wells

257

Index

269