Patent Application Titled "Differential Knockout Of An Allele Of A Heterozygous Bestrophin 1 Gene" Published Online (USPTO 20230174974).
In: Genomics & Genetics Weekly, 2023-06-30, S. 2225-2225
serialPeriodical
Zugriff:
The method of claim 41, wherein the cell comprises a mutant BEST1 allele and a functional BEST1 allele. The method of claim 57, wherein the sequence of the rs1800009 SNP position in the mutant BEST1 allele differs from the sequence of the rs1800009 SNP position in the functional BEST1 allele. "According to some embodiments of the present invention, there is provided a method for treating Best Vitelliform Macular Dystrophy, the method comprising delivering to a subject having Best Vitelliform Macular Dystrophy, a composition comprising an RNA molecule comprising a guide sequence portion having 17-20 nucleotides in the sequence of 17-20 contiguous nucleotides set forth in any one of SEQ ID NOs: 1-3010 and a CRISPR nuclease. [Extracted from the article]
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Titel: |
Patent Application Titled "Differential Knockout Of An Allele Of A Heterozygous Bestrophin 1 Gene" Published Online (USPTO 20230174974).
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Zeitschrift: | Genomics & Genetics Weekly, 2023-06-30, S. 2225-2225 |
Veröffentlichung: | 2023 |
Medientyp: | serialPeriodical |
ISSN: | 1531-6467 (print) |
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