Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping

Therapeutic exon skipping as a treatment for Duchenne muscular dystrophy (DMD) has largely concentrated on the delivery of antisense oligomers to treat out-of-frame exon deletions. Here we report on the preclinical development of an adeno-associated virus (AAV)-encapsidated viral vector containing f...

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Detalles Bibliográficos
Autores principales: Gushchina, Liubov V., Frair, Emma C., Rohan, Natalie, Bradley, Adrienne J., Simmons, Tabatha R., Chavan, Hemantkumar D., Chou, Hsin-Jung, Eggers, Michelle, Waldrop, Megan A., Wein, Nicolas, Flanigan, Kevin M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2021
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10112461/
https://www.ncbi.nlm.nih.gov/pubmed/33406986
http://dx.doi.org/10.1089/hum.2020.286

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