AAV-mediated gene therapy for choroideremia: preclinical studies in personalized models.

AAV-mediated gene therapy for choroideremia: preclinical studies in personalized models.

Choroideremia (CHM) is an X- linked retinal degeneration that is symptomatic in the 1(st) or 2(nd) decade of life causing nyctalopia and loss of peripheral vision. The disease progresses through mid-life, when most patients become blind. CHM is a favorable target for gene augmentation therapy, as th...

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Bibliographic Details
Main Authors: Vidyullatha Vasireddy, Jason A Mills, Rajashekhar Gaddameedi, Etiena Basner-Tschakarjan, Monika Kohnke, Aaron D Black, Krill Alexandrov, Shangzhen Zhou, Albert M Maguire, Daniel C Chung, Helen Mac, Lisa Sullivan, Paul Gadue, Jeannette L Bennicelli, Deborah L French, Jean Bennett
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2013-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC3646845?pdf=render

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http://europepmc.org/articles/PMC3646845?pdf=render

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