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...
Main Authors: | Maram E. A. Abdalla Elsayed, Maria Kaukonen, Peter Kiraly, Jasmina Cehajic Kapetanovic, Robert E. MacLaren |
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Format: | Article |
Language: | English |
Published: |
MDPI AG
2022-11-01
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Series: | Genes |
Subjects: | |
Online Access: | https://www.mdpi.com/2073-4425/13/11/2103 |
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