RNA-Editing Compositions and Methods of Use
AssigneeShape Therapeutics Inc.
InventorsYiannis SAVVA, Richard SULLIVAN, Brian BOOTH, Adrian BRIGGS, Debojit BOSE, Susan BYRNE, Stephen BURLEIGH
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Abstract
Provided herein are engineered latent guide RNAs that bind target RNAs to form a guide-target RNA scaffold and are substrates for RNA editing entities, which chemically modify the base of a nucleotide of the target RNA. Also provided herein are compositions, vectors, and cells comprising the engineered latent guide RNAs disclosed herein and methods of use thereof.
Claims134
An engineered guide RNA that, upon hybridization to a target RNA implicated in a disease or condition, forms a guide-target RNA scaffold comprising a structural feature selected from the group consisting of a bulge, an internal loop, a hairpin, and any combination thereof, wherein the structural feature substantially forms upon hybridization to the target RNA.
The method of any one of claims 108-114, wherein the engineered guide RNA comprises a polynucleotide having at least 80%, at least 85%, at least 90%, at least 95%, at least 97%, at least 99%, or 100% sequence identity to any one of SEQ ID NO:
- 35-42, 46-52, 111-207, or 344-345.
The engineered guide RNA, polynucleotide, delivery vector, or pharmaceutical composition for the use of claim 127, wherein the neurological disease is Parkinson's disease, Alzheimer's disease, a Tauopathy, or dementia.
Dependencies are inferred from the claim text.