Germline CRISPR/Cas9-Mediated Gene Editing Prevents Vision Loss in a Novel Mouse Model of Aniridia

Aniridia is a rare eye disorder, which is caused by mutations in the paired box 6 (PAX6) gene and results in vision loss due to the lack of a long-term vision-saving therapy. One potential approach to treating aniridia is targeted CRISPR-based genome editing. To enable the Pax6 small eye (Sey) mouse...

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Detalles Bibliográficos
Autores principales: Mirjalili Mohanna, Seyedeh Zeinab, Hickmott, Jack W., Lam, Siu Ling, Chiu, Nina Y., Lengyell, Tess C., Tam, Beatrice M., Moritz, Orson L., Simpson, Elizabeth M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7114625/
https://www.ncbi.nlm.nih.gov/pubmed/32258211
http://dx.doi.org/10.1016/j.omtm.2020.03.002

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7114625/
https://www.ncbi.nlm.nih.gov/pubmed/32258211
http://dx.doi.org/10.1016/j.omtm.2020.03.002

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