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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Detalles Bibliográficos
Autores principales: Vasireddy, Vidyullatha, Mills, Jason A., Gaddameedi, Rajashekhar, Basner-Tschakarjan, Etiena, Kohnke, Monika, Black, Aaron D., Alexandrov, Krill, Zhou, Shangzhen, Maguire, Albert M., Chung, Daniel C., Mac, Helen, Sullivan, Lisa, Gadue, Paul, Bennicelli, Jeannette L., French, Deborah L., Bennett, Jean
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2013
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3646845/
https://www.ncbi.nlm.nih.gov/pubmed/23667438
http://dx.doi.org/10.1371/journal.pone.0061396

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3646845/
https://www.ncbi.nlm.nih.gov/pubmed/23667438
http://dx.doi.org/10.1371/journal.pone.0061396

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