Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping
| Autor: | Michelle Eggers, Nicolas Wein, Hemantkumar D Chavan, Kevin M. Flanigan, Adrienne J Bradley, Liubov V Gushchina, Emma C Frair, Hsin-Jung Chou, Megan A. Waldrop, Tabatha R. Simmons, Natalie Rohan |
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| Rok vydání: | 2021 |
| Předmět: |
musculoskeletal diseases
congenital hereditary and neonatal diseases and abnormalities 0303 health sciences business.industry Duchenne muscular dystrophy medicine.disease medicine.disease_cause Exon skipping 03 medical and health sciences Exon 0302 clinical medicine 030220 oncology & carcinogenesis Toxicity Genetics medicine Cancer research Molecular Medicine Vector (molecular biology) business Molecular Biology Adeno-associated virus 030304 developmental biology |
| Zdroj: | Human Gene Therapy. 32:882-894 |
| ISSN: | 1557-7422 1043-0342 |
| Popis: | 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 o... |
| Databáze: | OpenAIRE |
| Externí odkaz: |
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