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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...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2022
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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 |