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.
Claims23
A method for treating Huntington's disease in a mammal comprising administering to the mammal an RNAi comprising a first strand comprising a first nucleic acid comprising the sequence 5′-UGGCCGUCCAUCUUGGACCCG-3′ (SEQ ID NO:1) and a second strand comprising a second nucleic acid comprising the sequence 5′-CGGGUCCAAGAUGGACGGCCA-3′ (SEQ ID NO:2) or a first strand comprising a first nucleic acid comprising the sequence 5′-AGUCGGUGUGGUUGACAAGCA-3′ (SEQ ID NO:
- 7) and a second strand comprising a second nucleic acid comprising the sequence 5′-UGCUUGUCAACCACACCGACU-3′ (SEQ ID NO:8).
A method for inhibiting the expression of htt in a mammal with Huntington's disease comprising administering to the mammal an RNAi comprising a first strand comprising a first nucleic acid comprising the sequence 5′-UGGCCGUCCAUCUUGGACCCG -3′ (SEQ ID NO:1) and a second strand comprising a second nucleic acid comprising the sequence 5′-CGGGUCCAAGAUGGACGGCCA-3′ (SEQ ID NO:2) or a first strand comprising a first nucleic acid comprising the sequence 5′-AGUCGGUGUGGUUGACAAGCA -3′ (SEQ ID NO:7) and a second strand comprising a second nucleic acid comprising the sequence 5′-UGCUUGUCAACCACACCGACU-3′ (SEQ ID NO:8).
A method for inhibiting the accumulation of htt in a cell of a mammal with Huntington's disease comprising administering to the mammal an RNAi comprising a first strand comprising a first nucleic acid comprising the sequence 5′-UGGCCGUCCAUCUUGGACCCG -3′ (SEQ ID NO:1) and a second strand comprising a second nucleic acid comprising the sequence 5′-CGGGUCCAAGAUGGACGGCCA-3′ (SEQ ID NO:2) or a first strand comprising a first nucleic acid comprising the sequence 5′-AGUCGGUGUGGUUGACAAGCA -3′ (SEQ ID NO:7) and a second strand comprising a second nucleic acid comprising the sequence 5′-UGCUUGUCAACCACACCGACU-3′ (SEQ ID NO:8).
Dependencies are inferred from the claim text.