IP Library › Granted Patent US 9,963,715
Granted Patent B2
US 9,963,715 · App. 14/013,236 · Granted May 8, 2018

Methods and compositions for treatment of a genetic condition

Inventors: Gregory J. Cost (Richmond, CA); Philip D. Gregory (Richmond, CA); Dmitry Guschin (Richmond, CA); Michael C. Holmes (Richmond, CA); Jeffrey C. Miller (Richmond, CA); David Paschon (Richmond, CA); Edward J. Rebar (Richmond, CA); Andreas Reik (Richmond, CA); Fyodor Urnov (Richmond, CA); Lei Zhang (Richmond, CA)
Assignee: Sangamo Therapeutics, Inc.
C12N15/85C07K14/4702C07K14/805C12N9/22C12N15/52C07K2319/81
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Quick Facts
Patent No.
US 9,963,715
App. No.
14/013,236
Granted
May 8, 2018
Kind
B2
Abstract

Disclosed herein are nucleases and methods of using these nucleases for alteration of a globin gene and generation of cells.

Claims (93)

1. A zinc finger protein comprising five or six zinc finger domains designated and ordered F1 to F5 or F1 to F6 from N-terminus to C-terminus, wherein F1 to F5 or F1 to F6 comprises any one of the following recognition helix regions:

(i) F1: DRSNLSR (SEQ ID NO:9);

F2: LRQNLIM (SEQ ID NO:161);

F3: TSANLTV (SEQ ID NO:162);

F4: RSDHLSR (SEQ ID NO:94);

F5: QSGNLAR (SEQ ID NO:39); and

F6: QRNDRKS (SEQ ID NO:163), wherein the zinc finger protein binds to a target site as set forth in SEQ ID NO:160;

(ii) F1: DRSNLSR (SEQ ID NO:9);

F2: LRQNLIM (SEQ ID NO:161);

F3: LQSQLNR (SEQ ID NO:164);

F4: RSDHLSR (SEQ ID NO:94);

F5: QSGNLAR (SEQ ID NO:39); and

F6: QRNDRKS (SEQ ID NO:163), wherein the zinc finger protein binds to a target site as set forth in SEQ ID NO:160;

(iii) F1: DRANLSR (SEQ ID NO:165);

F2: LRQNLIM (SEQ ID NO:161);

F3: LQSQLNR (SEQ ID NO:164);

F4: RSDHLSR (SEQ ID NO:94);

F5: QSGNLAR (SEQ ID NO:39); and

F6: QRNDRKS (SEQ ID NO:163), wherein the zinc finger protein binds to a target site as set forth in SEQ ID NO:160;

(iv) F1: DRSNLSR (SEQ ID NO:9);

F2: TSSNRNH (SEQ ID NO:166);

F3: HSGNLTK (SEQ ID NO:167);

F4: RSDHLSR (SEQ ID NO:94);

F5: QSGNLAR (SEQ ID NO:39); and

F6: QKVDLSR (SEQ ID NO:168), wherein the zinc finger protein binds to a target site as set forth in SEQ ID NO:160;

(v) F1: DRSNLSR (SEQ ID NO:9);

F2: TSSNRNH (SEQ ID NO:166);

F3: QANNLKV (SEQ ID NO:169);

F4: RSDHLSR (SEQ ID NO:94);

F5: QSGNLAR (SEQ ID NO:39); and

F6: QKVDLSR (SEQ ID NO:168), wherein the zinc finger protein binds to a target site as set forth in SEQ ID NO:160;

(vi) F1: RSDHLSA (SEQ ID NO:59);

F2: DRSALAR (SEQ ID NO:30);

F3: RSDSLSR (SEQ ID NO:171);

F4: DRSVRTK (SEQ ID NO:172);

F5: RSDHLSA (SEQ ID NO:59); and

F6: QRSNLKV (SEQ ID NO:173), wherein the zinc finger protein binds to a target site as set forth in SEQ ID NO:170;

(vii) F1: RSDHLTQ (SEQ ID NO:35);

F2: DRSALAR (SEQ ID NO:30);

F3: RSDSLSR (SEQ ID NO:171);

F4: DRSVRTK (SEQ ID NO:172);

F5: RSDHLSA (SEQ ID NO:59); and

F6: QRSNLKV (SEQ ID NO:173), wherein the zinc finger protein binds to a target site as set forth in SEQ ID NO:170;

(viii) F1: RSDHLTT (SEQ ID NO:174);

F2: DRSALAR (SEQ ID NO:30);

F3: RSDSLSR (SEQ ID NO:171);

F4: DRSVRTK (SEQ ID NO:172);

F5: RSDHLSA (SEQ ID NO:59); and

F6: QRSNLKV (SEQ ID NO:173), wherein the zinc finger protein binds to a target site as set forth in SEQ ID NO:170;

(ix) F1: RSDHLSA (SEQ ID NO:59);

F2: WATARDR (SEQ ID NO:175);

F3: RSDSLSR (SEQ ID NO:171);

F4: HTKSLSR (SEQ ID NO:176);

F5: RSDHLSA (SEQ ID NO:59);

F6: QRSNLKV (SEQ ID NO:173); wherein the zinc finger protein binds to a target site as set forth in SEQ ID NO:170;

(x) F1: RSAHLTQ (SEQ ID NO:177);

F2: DRSVLRR (SEQ ID NO:178)

F3: RSDSLSR (SEQ ID NO:171);

F4: DRSVRTK (SEQ ID NO:172);

F5: RSDHLSA (SEQ ID NO:59); and

F6: QRSNLKV (SEQ ID NO:173), wherein the zinc finger protein binds to a target site as set forth in SEQ ID NO:170;

(xi) F1: RSDVLSE (SEQ ID NO:57);

F2: RNQHRKT (SEQ ID NO:58);

F3: RSDHLSA (SEQ ID NO:59);

F4: RSANLTR (SEQ ID NO:60;

F5: RSDVLSN (SEQ ID NO:61); and

F6: DRSTRIT (SEQ ID NO:62), wherein the zinc finger protein binds to a target site as set forth in SEQ ID NO:56;

(xii) F1: DRSNLSR (SEQ ID NO:9);

F2: HRQHLVT (SEQ ID NO:64);

F3: DRSNLTR (SEQ ID NO:65);

F4: QSGDLTR (SEQ ID NO:13); and

F5: HRSSLLN (SEQ ID NO:66), wherein the zinc finger protein binds to a target site as set forth in SEQ ID NO:63;

(xiii) F1: QSGHLSR (SEQ ID NO:32);

F2: RSDHLST (SEQ ID NO:67);

F3: RSADLSR (SEQ ID NO:68);

F4: RSDNLSQ (SEQ ID NO:69); and

F5: ASNDRKK (SEQ ID NO:70), wherein the zinc finger protein binds to a target site as shown in SEQ ID NO:66; or

(xiv) F1: RSDNLSA (SEQ ID NO:72);

F2: RNNDRKT (SEQ ID NO:73);

F3: DRSDLSR (SEQ ID NO:29);

F4: TSSNRTK (SEQ ID NO:74);

F5: QSGNLAR (SEQ ID NO:39); and

F6: QSGDLTR (SEQ ID NO:13), wherein the zinc finger protein binds to a target site as set forth in SEQ ID NO:71.

2. A fusion protein comprising the zinc finger protein of claim 1 and a cleavage domain or cleavage half-domain.

3. An isolated polynucleotide encoding the zinc finger proteins of claim 1 .

4. An isolated cell comprising the zinc finger proteins of claim 1 .

5. An isolated cell comprising the polynucleotides of claim 3 .

6. The cell of claim 5 , wherein the cell is a red blood cell precursor cell.

7. The cell of claim 5 , wherein the cell is a red blood cell (RBC).

8. The cell of claim 6 , wherein the precursor cell is a CD34+ hematopoietic stem cell.

9. A kit comprising the protein of claim 1 .

10. A kit comprising the polynucleotide of claim 3 .

11. An isolated cell comprising the fusion protein of claim 2 .

Assignments (2)
CHANGE OF NAME Recorded Feb 1, 2018
From: SANGAMO BIOSCIENCES, INC.
To: SANGAMO THERAPEUTICS, INC.
Reel/Frame 045228/0884 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jan 16, 2014
From: COST, GREGORY J.; GREGORY, PHILIP D.; GUSCHIN, DMITRY; HOLMES, MICHAEL C.; MILLER, JEFFREY C.; PASCHON, DAVID; REBAR, EDWARD J.; REIK, ANDREAS; URNOV, FYODOR; ZHANG, LEI
To: SANGAMO BIOSCIENCES, INC.
Reel/Frame 031987/0520 →
Continuity (2)
Provisional Application 61694693 · Aug 29, 2012
Related Publication 20140093913A1 · Apr 3, 2014