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US 2023/0407279 A1Published application
Crispr/cas9 targeted excision of the intronic ctg18.1 trinucleotide repeat expansion of tcf4 as a therapy in fuchs' endothelial corneal dystrophy
AssigneeAvellino Lab USA, Inc.
InventorTara MOORE
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Abstract
The present disclosure relates to Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR associated protein 9 (Cas9) systems, and methods of use thereof for gene editing.
Claims20
A method of altering a gene product, the method comprising:
- administering into a cell an engineered CRISPR/Cas9 system comprising at least one vector comprising:
- (i) a nucleotide molecule encoding Cas9 nuclease;
- (ii) a first sgRNA comprising a first CRISPR targeting RNA (crRNA) sequence that hybridizes to a nucleotide sequence complementary to a first target sequence in a first intron, the first target sequence being positioned 5′ of a disease-causing repeat expansion that is present in the first intron;
- and
- (iii) a second sgRNA comprising a second crRNA sequence that hybridizes to a nucleotide sequence complementary to a second target sequence in the first intron, the second target sequence being positioned 3′ of the disease-causing repeat expansion,
- wherein the at least one vector does not have a nucleotide molecule encoding Cas9 nuclease and a crRNA sequence that naturally occur together.
3 more elements
A single guide RNA (sgRNA) comprising a nucleotide sequence having at least 85% sequence identify to a sequence selected from the group consisting of guide sequences shown in Table 1A or Table 1B.
Dependencies are inferred from the claim text.