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Retro-Orbital Venous Sinus Delivery of rAAV9 Mediates High-Level Transduction of Brain and Retina Compared with Temporal Vein Delivery in Neonatal Mouse Pups

In order to pursue a clinical gene therapy for a human neurologic disease, it is often necessary to perform proof-of-concept trials in mouse models of that disease. In order to demonstrate a potential clinical efficacy, one must be able to select an appropriate vector and route of delivery for the a...

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Detalles Bibliográficos
Publicado en:Hum Gene Ther
Main Authors: Gruntman, Alisha M., Su, Lin, Flotte, Terence R.
Formato: Artigo
Idioma:Inglês
Publicado: Mary Ann Liebert, Inc. 2017
Assuntos:
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC5824662/
https://ncbi.nlm.nih.gov/pubmed/28319444
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2017.037
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