Oligonucleotides for htt-1a modulation
OwnerAnastasia Khvorova, Julia Alterman +3inventors
InventorsAnastasia Khvorova, Julia Alterman, Socheata Ly, Faith Conroy, Daniel O'Reilly
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Abstract
This disclosure relates to novel HTT-1A targeting sequences. Novel HTT-1A targeting oligonucleotides for the treatment of neurodegenerative diseases are also provided.
Claims47
A double stranded RNA (dsRNA) molecule comprising a sense strand and an antisense strand, wherein the antisense strand comprises a sequence substantially complementary to a HTT nucleic acid sequence of SEQ ID NO:
- 1
A double stranded RNA (dsRNA) molecule comprising a sense strand and an antisense strand, wherein the antisense strand comprises a sequence substantially complementary to a HTT-1A nucleic acid sequence of one of SEQ ID NOs:
- 2-19.
6-8. (canceled)
10-21. (canceled)
23-33. (canceled)
35-37. (canceled)
(canceled)
41-46. (canceled)
49-53. (canceled)
55-68. (canceled)
71-72. (canceled)
75-79. (canceled)
A vector comprising a regulatory sequence operably linked to a nucleotide sequence that encodes a dsRNA molecule substantially complementary to a HTT-1A nucleic acid sequence of SEQ ID NO:
- 2-19, optionally wherein:
- said dsRNA molecule inhibits the expression of said HTT-1A gene by at least 30%, 50%, or 80%;
- and/or
- the dsRNA comprises a sense strand and an antisense strand, wherein the antisense strand comprises a sequence substantially complementary to a HTT-1A nucleic acid sequence of SEQ ID NO:
- 2-19.
81-84. (canceled)
(canceled)
A branched RNA compound comprising:
- two or more RNA molecules comprising 15 to 35 nucleotides in length, and
- a sequence substantially complementary to a HTT-1A mRNA,
- wherein the two RNA molecules are connected to one another by one or more moieties independently selected from a linker, a spacer and a branching point.
91-94. (canceled)
96-122. (canceled)
124-157. (canceled)
159-177. (canceled)
179-180. (canceled)
183-187. (canceled)
Dependencies are inferred from the claim text.