Adeno-Associated Virus 8 and 9 Myofibre Type/Size Tropism Profiling Reveals Therapeutic Effect of Microdystrophin in Canines

Adeno-associated virus (AAV) 8 and 9 are in clinical trials for treating neuromuscular diseases such as Duchenne muscular dystrophy (DMD). Muscle consists of myofibres of different types and sizes. However, little is known about the fibre type and fibre size tropism of AAV in large mammals. We evalu...

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Veröffentlicht in:	Journal of cachexia, sarcopenia and muscle sarcopenia and muscle, 2025-02, Vol.16 (1), p.e13681
Hauptverfasser:	Burke, Matthew J, Blatt, Braiden M, Teixeira, James A, Pérez-López, Dennis O, Yue, Yongping, Pan, Xiufang, Hakim, Chady H, Yao, Gang, Herzog, Roland W, Duan, Dongsheng
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Dependovirus - genetics Disease Models, Animal Dogs Dystrophin - genetics Dystrophin - metabolism Genetic Therapy - methods Genetic Vectors Muscle, Skeletal - metabolism Muscular Dystrophy, Duchenne - genetics Muscular Dystrophy, Duchenne - therapy Transduction, Genetic Transgenes
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