Methods of targeting repetitive rna in huntington's disease
AssigneeThe Regents of the University of California
InventorsEugene Yeo, Kathryn H. Morelli
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Abstract
Provided herein are methods of treating Huntington's disease in a subject, the method comprising administering to the subject a therapeutically effective amount of a pharmaceutical composition comprising: (a) a guide RNA (gRNA), wherein the guide RNA comprises a repeat sequence complementary to a target CAG-expansion RNA, and wherein the gRNA allele-selectively targets the target CAG-expansion RNA; and (b) a CRISPR-associated protein or a nucleic acid sequence encoding the CRISPR-associated protein, wherein the CRISPR-associated protein comprises a Cas13d polypeptide.
Claims61
A method of treating Huntington's disease in a subject, the method comprising administering to the subject a therapeutically effective amount of a pharmaceutical composition comprising:
- (a) a guide RNA (gRNA), wherein the guide RNA comprises a repeat sequence complementary to a target CAG-expansion RNA, and wherein the gRNA allele-selectively targets the target CAG-expansion RNA;
- and
- (b) a CRISPR-associated protein or a nucleic acid sequence encoding the CRISPR-associated protein, wherein the CRISPR-associated protein comprises a Cas13d polypeptide.
A recombinant allele-selective expression system for CRISPR/Cas-directed RNA targeting of a target CAG-expansion RNA comprising:
- (a) a guide RNA, wherein the guide RNA comprises a repeat sequence complementary to the target CAG-expansion RNA;
- and
- (b) a CRISPR-associated protein or a nucleic acid sequence encoding the CRISPR-associated protein, wherein the CRISPR-associated protein comprises a Cas13d polypeptide.
A vector comprising a nucleic acid encoding (i) a guide RNA, wherein the guide RNA comprises a repeat sequence complementary to a target CAG-expansion RNA, and wherein the gRNA allele-selectively targets the target CAG-expansion RNA;
- and (ii) a CRISPR-associated protein, wherein the CRISPR-associated protein comprises a Cas13d polypeptide.
Dependencies are inferred from the claim text.