Repair of Retinal Degeneration following Ex Vivo Minicircle DNA Gene Therapy and Transplantation of Corrected Photoreceptor Progenitors

The authors describe retinal reconstruction and restoration of visual function in heritably blind mice missing the rhodopsin gene using a novel method of ex vivo gene therapy and cell transplantation. Photoreceptor precursors with the same chromosomal genetic mutation were treated ex vivo using mini...

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Detalles Bibliográficos
Publicado en:Mol Ther
Main Authors: Barnea-Cramer, Alona O., Singh, Mandeep, Fischer, Dominik, De Silva, Samantha, McClements, Michelle E., Barnard, Alun R., MacLaren, Robert E.
Formato: Artigo
Idioma:Inglês
Publicado: American Society of Gene & Cell Therapy 2020
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Original Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC7054814/
https://ncbi.nlm.nih.gov/pubmed/32027843
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.01.023
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