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Analysis of Pathogenic Variants Correctable With CRISPR Base Editing Among Patients With Recessive Inherited Retinal Degeneration
IMPORTANCE: Many common inherited retinal diseases are not easily treated with gene therapy. Gene editing with base editors may allow the targeted repair of single-nucleotide transition variants in DNA and RNA. It is unknown how many patients have pathogenic variants that are correctable with a base...
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| Gepubliceerd in: | JAMA Ophthalmol |
|---|---|
| Hoofdauteurs: | , , |
| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
American Medical Association
2021
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| Onderwerpen: | |
| Online toegang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7844696/ https://ncbi.nlm.nih.gov/pubmed/33507217 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1001/jamaophthalmol.2020.6418 |
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