Design and testing of regulatory cassettes for optimal activity in skeletal and cardiac muscles
CL Himeda, X Chen, SD Hauschka - Muscle gene therapy: methods and …, 2010 - Springer
CL Himeda, X Chen, SD Hauschka
Muscle gene therapy: methods and protocols, 2010•SpringerGene therapy for muscular dystrophies requires efficient gene delivery to the striated
musculature and specific, high-level expression of the therapeutic gene in a physiologically
diverse array of muscles. This can be achieved by the use of recombinant adeno-associated
virus vectors in conjunction with muscle-specific regulatory cassettes. We have constructed
several generations of regulatory cassettes based on the enhancer and promoter of the
muscle creatine kinase gene, some of which include heterologous enhancers and individual …
musculature and specific, high-level expression of the therapeutic gene in a physiologically
diverse array of muscles. This can be achieved by the use of recombinant adeno-associated
virus vectors in conjunction with muscle-specific regulatory cassettes. We have constructed
several generations of regulatory cassettes based on the enhancer and promoter of the
muscle creatine kinase gene, some of which include heterologous enhancers and individual …
Abstract
Gene therapy for muscular dystrophies requires efficient gene delivery to the striated musculature and specific, high-level expression of the therapeutic gene in a physiologically diverse array of muscles. This can be achieved by the use of recombinant adeno-associated virus vectors in conjunction with muscle-specific regulatory cassettes. We have constructed several generations of regulatory cassettes based on the enhancer and promoter of the muscle creatine kinase gene, some of which include heterologous enhancers and individual elements from other muscle genes. Since the relative importance of many control elements varies among different anatomical muscles, we are aiming to tailor these cassettes for high-level expression in cardiac muscle, and in fast and slow skeletal muscles. With the achievement of efficient intravascular gene delivery to isolated limbs, selected muscle groups, and heart in large animal models, the design of cassettes optimized for activity in different muscle types is now a practical goal. In this protocol, we outline the key steps involved in the design of regulatory cassettes for optimal activity in skeletal and cardiac muscle, and testing in mature muscle fiber cultures. The basic principles described here can also be applied to engineering tissue-specific regulatory cassettes for other cell types.
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