Abstract
Mutations in the dystrophin gene result in Duchenne muscular dystrophy (DMD), a progressive muscle-wasting disease. Adeno-associated virus (AAV) mediated gene replacement, and CRISPR/Cas9-mediated genome editing hold the potential to treat DMD. Molecular and biochemical analyses are essential to determine gene transfer efficiency and therapeutic efficacy. In this chapter, we present a series of methods routinely used in our laboratory to extract and quantify DNA, RNA, and protein in gene therapy studies performed in the canine DMD model.
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Acknowledgments
The research on the canine DMD model in the Duan lab is currently supported by the National Institutes of Health (NS-90634 and AR-70517), Jesse Davidson Foundation-Defeat Duchenne Canada, Hope for Javier, Jackson Freel DMD Research Fund, Parent Project Muscular Dystrophy, Jett Foundation, Michael’s Cause, Ryan’s Quest, Solid Biosciences Inc., and the University of Missouri.
Disclosure
DD is a member of the scientific advisory board for Solid Biosciences and equity holders of Solid Biosciences. DD is a member of the scientific advisory board for Sardocor Corp. In the last 3 years, the Duan lab has received research supports unrelated to this project from Solid Biosciences and Edgewise.
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Hakim, C.H., Pérez-López, D., Burke, M.J., Teixeira, J., Duan, D. (2023). Molecular and Biochemical Assessment of Gene Therapy in the Canine Model of Duchenne Muscular Dystrophy. In: Maruyama, R., Yokota, T. (eds) Muscular Dystrophy Therapeutics. Methods in Molecular Biology, vol 2587. Humana, New York, NY. https://doi.org/10.1007/978-1-0716-2772-3_15
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DOI: https://doi.org/10.1007/978-1-0716-2772-3_15
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