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Assessment of Therapeutic Potential of a Dual AAV Approach for Duchenne Muscular Dystrophy by Sonia Albini; Laura Palmieri; Auriane Dubois; Nathalie Bourg; William Lostal; Isabelle Richard is a Biochemistry, Genetics and Molecular Biology article available to read on EtoBox.

What is Assessment of Therapeutic Potential of a Dual AAV Approach for Duchenne Muscular Dystrophy about?

Duchenne muscular dystrophy (DMD) is a yet incurable rare genetic disease that affects the skeletal and cardiac muscles, leading to progressive muscle wasting and premature death. DMD is caused by the lack of dystrophin, a muscle protein essential for the biochemical support and integrity of muscle fibers. Gene replacement strategies for Duchenne muscular dystrophy (DMD) employing the adeno-associated virus (AAV) face the challenge imposed by the limited packaging capacity of AAV, only allowing the accommodation of a short version of dystrophin (μDys) that is still far removed from correcting human disease. The need to develop strategies leading to the expression of a best performing dystrophin variant led to only few studies reporting on the use of dual vectors, but none reported on a method to assess in vivo transgene reconstitution efficiency, the degree of which directly affects the use of safe AAV dosing. We report here on the generation of a dual AAV vector approach for the expression of a larger dystrophin version (quasidystrophin) based on homologous recombination, and the development of a methodology employing a strategic droplet digital PCR design, to determine the recomb

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Author

Sonia Albini; Laura Palmieri; Auriane Dubois; Nathalie Bourg; William Lostal; Isabelle Richard

Publisher

MDPI AG

Published

2023

Language

EN

Field

Biochemistry, Genetics and Molecular Biology (Life Sciences)