Back to results
US 12,473,545 B2Granted
Regulation of RAN translation by PKR and eIF2α-P pathways
AssigneeUniversity of Florida Research Foundation, Inc.
InventorsLaura Ranum, Tao Zu
Life of this patent
Estimating the term…
Abstract
Methods and compositions for modulating repeat non-ATG protein (RAN protein) translation are provided. In some aspects, the disclosure provides methods of inhibiting RAN protein translation by contacting a cell with an effective amount of an inhibitor of eIF2 phosphorylation or an inhibitor of protein kinase R (PKR). In some embodiments, methods described by the disclosure are useful for treating diseases associated with RAN protein translation, such as certain neurodegenerative diseases.
Claims6
A recombinant adeno-associated virus (rAAV) comprising:
- (i) a capsid protein;
- and
- (ii) a nucleic acid comprising a transgene operably linked to a promoter, wherein the transgene encodes a dominant negative variant of protein kinase R (PKR) comprising a K296R mutation relative to amino acid position 296 in the wild-type PKR amino acid sequence set forth in SEQ ID NO:
- 15.
1 more element
Dependencies are inferred from the claim text.