Granted Patent
B2
US 12,747,273 · App. 17/423,063 · Granted Sep 29, 2026
HTT repressors comprising a zinc-finger protein transcription factor
Inventors:
Galen Carey (Lexington, MA); Matthew Chiocco (Southborough, MA); Vivian Choi (Lexington, MA); Brian Felice (Lexington, MA); Steven Froelich (Brisbane, CA); Debra Klatte (Lexington, MA); Jeffrey Miller (Brisbane, CA); David Paschon (Brisbane, CA); Edward Rebar (Brisbane, CA); Bryan Zeitler (Brisbane, CA); Lei Zhang (Brisbane, CA); H. Steve Zhang (Brisbane, CA)
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Abstract
Disclosed herein are HTT repressors and methods and compositions for use of these HTT repressors. Disclosed herein are methods and compositions for diagnosing, preventing and/or treating Huntington's Disease. In particular, provided herein are methods and compositions for modifying (e.g., modulating expression of) an HD HTT allele so as to prevent or treat Huntington Disease, including mHTT repressors (that repress mHTT transcripts and thus also repress mHTT protein expression).
Claims (10)
1 . A zinc finger protein transcription factor (ZFP-TF) comprising a zinc finger protein (ZFP) comprising the recognition helix region sequences in the following order from F1 to F4: F1 comprising SEQ ID NO: 7, F2 comprising SEQ ID NO: 8, F3 comprising SEQ ID NO: 9, and F4 comprising SEQ ID NO: 3.
2 . The ZFP-TF of claim 1 , comprising the amino acid sequence of SEQ ID NO: 21.
3 . A polynucleotide encoding the ZFP-TF of claim 1 .
4 . The polynucleotide encoding the ZFP-TF of claim 3 , comprising the polynucleotide sequence of SEQ ID NO: 16.
5 . An rAAV vector comprising one or more polynucleotides of claim 3 .
6 . The rAAV vector of claim 5 , further comprising a sequence encoding a nuclear localization signal (NLS).
7 . The rAAV vector of claim 6 , further comprising a sequence encoding a promoter driving expression of the ZFP-TF.
8 . An rAAV vector comprising one or more polynucleotides of claim 4 .
9 . A pharmaceutical composition comprising one or more polynucleotides according to claim 3 .
10 . A pharmaceutical composition comprising one or more polynucleotides according to claim 4 .
Continuity (2)
Provisional Application
62792701
· Jan 15, 2019
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