Methods and compositions for treating huntington's disease
AssigneeSangamo Therapeutics, Inc.
InventorsJeffrey C. Miller, Edward J. Rebar, H. Steve Zhang
Life of this patent
Estimating the term…
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.
Claims9
An mRNA encoding a fusion protein comprising a transcriptional repression domain and a non-naturally occurring zinc finger protein comprising 5 or 6 zinc finger domains ordered F1 to F5 or F1 to F6, respectively, wherein the zinc finger protein is designated 33074, 30080, 30643, 30648, 30645, 30640, 30642, or 30646 and specifically binds to mutant Huntington's gene allele (mHtt) gene and comprises the recognition helix regions as follows:
- ZFP
- F1
- F2
- F3
- F4
- F5
- F6
- 33074
- RSDNLSE
- KRCNLRC
- QSGDLTR
- QSGDLTR
- RSDNLSE
- KRCNLRC
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- NO:
- 60)
- NO:
- 61)
- NO:
- 18)
- NO:
- 18)
- NO:
- 60)
- NO:
- 61)
- 33080
- QSGDLTR
- QSGDLTR
- RSDNLSE
- KRCNLRC
- QSGDLTR
- QSGDLTR
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- NO:
- 18)
- NO:
- 18)
- NO:
- 60)
- NO:
- 61)
- NO:
- 18)
- NO:
- 18)
- 30643
- RSDNLSE
- KRCNLRC
- QSSDLSR
- QWSTRKR
- QSSDLSR
- QWSTRKR
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- NO:
- 60)
- NO:
- 61)
- NO:
- 31)
- NO:
- 63)
- NO:
- 31)
- NO:
- 63)
- 30648
- RSDNLSE
- KRCNLRC
- RSDNLSE
- KRCNLRC
- RSDNLSE
- KRCNLRC
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- NO:
- 60)
- NO:
- 61)
- NO:
- 60)
- NO:
- 61)
- NO:
- 60)
- NO:
- 61)
- 30645
- RSDNLSE
- KRCNLRC
- QSSDLSR
- QWSTRKR
- QSGDLTR
- NA
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- NO:
- 60)
- NO:
- 61)
- NO:
- 31)
- NO:
- 63)
- NO:
- 18)
- 30640
- QSSDLSR
- QWSTRKR
- QSSDLSR
- QWSTRKR
- QSGDLTR
- NA
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- NO:
- 31)
- NO:
- 63)
- NO:
- 31)
- NO:
- 63)
- NO:
- 18)
- 30642
- QSGDLTR
- QSSDLSR
- QWSTRKR
- QSSDLSR
- QWSTRKR
- NA
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- NO:
- 18)
- NO:
- 31)
- NO:
- 63)
- NO:
- 31)
- NO:
- 63)
- 30646
- RSDNLSE
- KRCNLRC
- QSGDLTR
- QSSDLSR
- QWSTRKR
- NA
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- (SEQ ID
- NO:
- 60)
- NO:
- 61)
- NO:
- 18)
- NO:
- 31)
- NO:
- 63)
Dependencies are inferred from the claim text.