Methods and compositions for treating Huntington's disease
AssigneeSangamo Therapeutics, Inc.
InventorsJeffrey C. Miller, Edward J. Rebar, H. Steve Zhang
Life of this patent
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Abstract
Disclosed herein are compositions comprising non-naturally occurring zinc finger domains, fusion proteins comprising these zinc finger domains, polynucleotides encoding these proteins, cells expressing these proteins and pharmaceutical compositions comprising these proteins or polynucleotides as well as methods of modifying an Htt gene using these compositions for treating or preventing Huntington's Disease.
Claims13
A non-naturally occurring zinc finger protein comprising 6 zinc finger domains ordered F1 to F6, respectively, wherein the zinc finger protein specifically binds to mutant Huntington's gene allele (mHtt) gene and comprises the recognition helix regions as follows:
- (SEQ ID NO:
- 60)
- F1:
- RSDNLSE;
- (SEQ ID NO:
- 61)
- F2:
- KRCNLRC;
- (SEQ ID NO:
- 18)
- F3:
- QSGDLTR;
- (SEQ ID NO:
- 18)
- F4:
- QSGDLTR;
- (SEQ ID NO:
- 60)
- F5:
- RSDNLSE;
- and
- (SEQ ID NO:
- 61)
- F6:
- KRCNLRC.
Dependencies are inferred from the claim text.