A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy

Micro-dystrophins are highly promising candidates for treating Duchenne muscular dystrophy, a lethal muscle disease caused by dystrophin deficiency. Here, we report robust disease rescue in the severe DBA/2J-mdx model with a neuronal nitric oxide synthase (nNOS)-binding micro-dystrophin vector. 2 ×...

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Үндсэн зохиолчид:	Chady H. Hakim, Nalinda B. Wasala, Xiufang Pan, Kasun Kodippili, Yongping Yue, Keqing Zhang, Gang Yao, Brittney Haffner, Sean X. Duan, Julian Ramos, Joel S. Schneider, N. Nora Yang, Jeffrey S. Chamberlain, Dongsheng Duan
Формат:	Artigo
Хэл сонгох:	Inglês
Хэвлэсэн:	Elsevier 2017-09-01
Цуврал:	Molecular Therapy: Methods & Clinical Development
Нөхцлүүд:	AAV DMD micro-dystrophin mdx DBA/2J nNOS cardiomyopathy systemic gene therapy Duchenne muscular dystrophy adeno-associated virus
Онлайн хандалт:	http://www.sciencedirect.com/science/article/pii/S2329050117300815
Шошгууд:	Шошго нэмэх Шошго байхгүй, Энэхүү баримтыг шошголох эхний хүн болох!