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Systemic and Persistent Muscle Gene Expression in Rhesus Monkeys with a Liver De-Targeted Adeno-Associated Virus Vector

The liver is a major off-target organ in gene therapy approaches for cardiac and musculoskeletal disorders. Intravenous administration of most of the naturally occurring adeno-associated virus (AAV) strains invariably results in vector genome sequestration within the liver. In the current study, we...

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Dades bibliogràfiques
Publicat a:Hum Gene Ther
Autors principals: Tarantal, Alice F., Lee, C. Chang I., Martinez, Michele L., Asokan, Aravind, Samulski, R.Jude
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc. 2017
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5444483/
https://ncbi.nlm.nih.gov/pubmed/28125921
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2016.130
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