Summary and Future Directions

Preclinical and clinical studies have established the feasibility of the muscle as a factory for the treatment of metabolic diseases; however it is evident that improvements in transduction levels may be necessary for this approach to be successful clinically, especially for those diseases that require circulating protein for correction. Refinements such as improved vector packaging and purification systems as well as modifications within the vectors themselves to increase relative bioactivity are the focus of current and future studies.

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