Summary and Future Direction

In summary, AAV-mediated gene therapy for muscular dystrophies on small animal models by systemic therapeutic gene delivery has been very successful. It can fundamentally restore the defective gene leading to functional recovery of the disease phenotype. In this chapter, we described how gene therapy was applied to multiple forms of muscular dystrophies, particularly, DMD, congenital muscular dystrophy, as well as limb-girdle muscular dystrophy. These studies, along with the work of many other groups, have laid a solid foundation for the development of gene therapy regime for muscular dystrophy patients. In recent years, tremendous efforts have been made to translate the encouraging results from small animal models to large animal models, and eventually to muscular dystrophy patients. One of the examples is the translation from mdx mice to the golden retriever muscular dystrophy (GRMD) dogs and to DMD patients. A number of new challenges absent in the mouse studies are met in the studies involving the GRMD dogs, which are a few hundred times larger than mice, manifest more severe and diverse disease pheno-types, and more importantly, come with more complex immune systems. Cellular immune responses against AAV vectors that were not seen in mice have been observed in the GRMD dogs (Wang et al. 2007a, b). These new challenges call for efforts on improvement of targeted gene delivery, tissue-specific therapeutic gene expression, immune modulation and new vector development and large-scale production technologies. Given the concerted efforts and exponential advances in muscular dystrophy gene therapy research and development over the past 10 years, we are confident that the challenges will be conquered one by one, and gene therapy for muscular dystrophies will eventually succeed.

Acknowledgement Most of the work was funded by NIH.

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