	US 11,723,952 B2
	Methods and compositions for treating Huntington's Disease
Jeffrey C. Miller, Brisbane, CA (US); Edward J. Rebar, Brisbane, CA (US); and H. Steve Zhang, Brisbane, CA (US)
Assigned to Sangamo Therapeutics, Inc., Brisbane, CA (US)
Filed by Sangamo Therapeutics, Inc., Brisbane, CA (US)
Filed on Nov. 16, 2020, as Appl. No. 17/98,970.
Application 17/098,970 is a continuation of application No. 16/587,428, filed on Sep. 30, 2019, granted, now 10,857,203.
Application 16/587,428 is a continuation of application No. 16/291,708, filed on Mar. 4, 2019, granted, now 10,471,123, issued on Nov. 12, 2019.
Application 16/291,708 is a continuation of application No. 15/294,062, filed on Oct. 14, 2016, granted, now 10,265,377, issued on Apr. 23, 2019.
Application 15/294,062 is a continuation of application No. 14/446,189, filed on Jul. 29, 2014, granted, now 9,499,597, issued on Nov. 22, 2016.
Application 14/446,189 is a continuation of application No. 13/781,176, filed on Feb. 28, 2013, granted, now 8,841,260, issued on Sep. 23, 2014.
Claims priority of provisional application 61/605,028, filed on Feb. 29, 2012.
Prior Publication US 2021/0060124 A1, Mar. 4, 2021
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 38/17 (2006.01); C07K 14/47 (2006.01); C12N 15/90 (2006.01)

CPC A61K 38/1709 (2013.01) [C07K 14/4703 (2013.01); C12N 15/907 (2013.01); C07K 2319/00 (2013.01); C07K 2319/70 (2013.01); C07K 2319/71 (2013.01); C07K 2319/81 (2013.01)]

9 Claims

1. 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

33074	RSDNLSE	KRCNLRC	QSGDLTR
	(SEQ ID	(SEQ ID	(SEQ ID
	NO: 60)	NO: 61)	NO: 18)

33080	QSGDLTR	QSGDLTR	RSDNLSE
	(SEQ ID	(SEQ ID	(SEQ ID
	NO: 18)	NO: 18)	NO: 60)

30643	RSDNLSE	KRCNLRC	QSSDLSR
	(SEQ ID	(SEQ ID	(SEQ ID
	NO: 60)	NO: 61)	NO: 31)

30648	RSDNLSE	KRCNLRC	RSDNLSE
	(SEQ ID	(SEQ ID	(SEQ ID
	NO: 60)	NO: 61)	NO: 60)

30645	RSDNLSE	KRCNLRC	QSSDLSR
	(SEQ ID	(SEQ ID	(SEQ ID
	NO: 60)	NO: 61)	NO: 31)

30640	QSSDLSR	QWSTRKR	QSSDLSR
	(SEQ ID	(SEQ ID	(SEQ ID
	NO: 31)	NO: 63)	NO: 31)

30642	QSGDLTR	QSSDLSR	QWSTRKR
	(SEQ ID	(SEQ ID	(SEQ ID
	NO: 18)	NO: 31)	NO: 63)

30646	RSDNLSE	KRCNLRC	QSGDLTR
	(SEQ ID	(SEQ ID	(SEQ ID
	NO: 60)	NO: 61)	NO: 18)

ZFP	F4	F5	F6

33074	QSGDLTR	RSDNLSE	KRCNLRC
	(SEQ ID	(SEQ ID	(SEQ ID
	NO: 18)	NO: 60)	NO: 61)

33080	KRCNLRC	QSGDLTR	QSGDLTR
	(SEQ ID	(SEQ ID	(SEQ ID
	NO: 61)	NO: 18)	NO: 18)

30643	QWSTRKR	QSSDLSR	QWSTRKR
	(SEQ ID	(SEQ ID	(SEQ ID
	NO: 63)	NO: 31)	NO: 63)

30648	KRCNLRC	RSDNLSE	KRCNLRC
	(SEQ ID	(SEQ ID	(SEQ ID
	NO: 61)	NO: 60)	NO: 61)

30645	QWSTRKR	QSGDLTR	NA
	(SEQ ID	(SEQ ID
	NO: 63)	NO: 18)

30640	QWSTRKR	QSGDLTR	NA
	(SEQ ID	(SEQ ID
	NO: 63)	NO: 18)

30642	QSSDLSR	QWSTRKR	NA
	(SEQ ID	(SEQ ID
	NO: 31)	NO: 63)

30646	QSSDLSR	QWSTRKR	NA
	(SEQ ID	(SEQ ID
	NO: 31)	NO: 63)