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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Publicat a:Mol Ther
Autors principals: Barnea-Cramer, Alona O., Singh, Mandeep, Fischer, Dominik, De Silva, Samantha, McClements, Michelle E., Barnard, Alun R., MacLaren, Robert E.
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2020
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Original Article
Accés en línia: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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