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Potential CRISPR Base Editing Therapeutic Options in a Sorsby Fundus Dystrophy Patient

TIMP3 mutations are associated with early-onset macular choroidal neovascularisation for which no treatment currently exists. CRISPR base editing, with its ability to irreversibly correct point mutations by chemical modification of nucleobases at DNA level, may be a therapeutic option. We report a b...

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Detalles Bibliográficos
Autores principales: Elsayed, Maram E. A. Abdalla, Kaukonen, Maria, Kiraly, Peter, Kapetanovic, Jasmina Cehajic, MacLaren, Robert E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9690532/
https://www.ncbi.nlm.nih.gov/pubmed/36421778
http://dx.doi.org/10.3390/genes13112103