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US 11,603,529 B2Granted
Variant RNAi
AssigneeGenzyme Corporation
InventorsCatherine R. O'Riordan, Adam Palermo, Brenda Richards, Lisa M. Stanek
Life of this patent
Estimating the term…
Abstract
Provided herein are RNAi molecules for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.
Claims43
An RNAi comprising a first strand and a second strand, wherein
- a) the first strand and the second strand form a duplex;
- b) the first strand comprises a guide region, wherein the guide region comprises the nucleic acid sequence 5′-UGGCCGUCCAUCUUGGACCCG-3′ (SEQ ID NO:1) or a nucleic acid sequence having more than 90% identity to SEQ ID NO:
- 1;
- or 5′-AGUCGGUGUGGUUGACAAGCA-3′ (SEQ ID NO:7), or a nucleic acid sequence having more than 90% identity to SEQ ID NO:
- 7;
- and
- c) the second strand comprises a non-guide region.
4 more elements
Dependencies are inferred from the claim text.