Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa

Our collaborative successful gene replacement therapy using AAV vectors expressing a variant of human RPGR-ORF15 in two canine models provided therapeutic proof of concept for translation into human treatment. The ORF15 sequence contained within this AAV vector, however, has ORF15 DNA sequence varia...

Fuld beskrivelse

Na minha lista:
Bibliografiske detaljer
Udgivet i:Hum Gene Ther
Main Authors: Deng, Wen-Tao, Dyka, Frank M., Dinculescu, Astra, Li, Jie, Zhu, Ping, Chiodo, Vince A., Boye, Sanford L., Conlon, Thomas J., Erger, Kirsten, Cossette, Travis, Hauswirth, William W.
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc. 2015
Fag:
Research Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4575541/
https://ncbi.nlm.nih.gov/pubmed/26076799
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2015.035
Tags: Tilføj Tag
Ingen Tags, Vær først til at tagge denne postø!

Lignende værker