Synthetic Adeno-Associated Viral Vector Efficiently Targets Mouse and Nonhuman Primate Retina In Vivo

Gene therapy is a promising approach in the treatment of inherited and common complex disorders of the retina. Preclinical and clinical studies have validated the use of adeno-associated viral vectors (AAV) as a safe and efficient delivery vehicle for gene transfer. Retinal pigment epithelium and ro...

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Veröffentlicht in:Hum Gene Ther
Hauptverfasser: Carvalho, Livia S., Xiao, Ru, Wassmer, Sarah J., Langsdorf, Aliete, Zinn, Eric, Pacouret, Simon, Shah, Samiksha, Comander, Jason I., Kim, Leo A., Lim, Laurence, Vandenberghe, Luk H.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Mary Ann Liebert, Inc. 2018
Schlagworte:
Research Articles
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6066192/
https://ncbi.nlm.nih.gov/pubmed/29325457
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2017.154
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