RNA TARGETING OF MUTATIONS VIA SUPPESSOR tRNAs AND DEAMINASES
AssigneeThe Regents of the University of California
InventorsPrashant Mali, Dhruva Katrekar
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Abstract
Aspects of the disclosure relate to a gene therapy approach for diseases, disorders, or conditions caused by mutation in the stop codon utilizing modified tRNA. At least 10-15% of all genetic diseases, including muscular dystrophy (e.g. Duchene muscular dystrophy), some cancers, beta thalassemia, Hurler syndrome, and cystic fibrosis, fall into this category. Not to be bound by theory, it is believed that this approach is safer than CRISPR approaches due to minimal off-target effects and the lack of genome level changes.
Claims22
1-92. (canceled)
A pharmaceutical composition in unit dose form comprising:
- (a) a DNA encoding a polynucleotide that comprises:
- (i) a recruiting domain that is recognized by an enzyme, and
- (ii) a targeting domain that hybridizes to a target RNA, and
- (b) a pharmaceutically acceptable excipient, diluent, or carrier,
- wherein the enzyme, when contacted with the polynucleotide and the target RNA, performs a chemical modification on a ribonucleotide base comprised in a nucleotide of the target RNA.
Dependencies are inferred from the claim text.