US Patent No. 9,963,715

METHODS AND COMPOSITIONS FOR TREATMENT OF A GENETIC CONDITION


Patent No. 9,963,715
Issue Date May 08, 2018
Title Methods And Compositions For Treatment Of A Genetic Condition
Inventorship Gregory J. Cost, Richmond, CA (US)
Philip D. Gregory, Richmond, CA (US)
Dmitry Guschin, Richmond, CA (US)
Michael C. Holmes, Richmond, CA (US)
Jeffrey C. Miller, Richmond, CA (US)
David Paschon, Richmond, CA (US)
Edward J. Rebar, Richmond, CA (US)
Andreas Reik, Richmond, CA (US)
Fyodor Urnov, Richmond, CA (US)
Lei Zhang, Richmond, CA (US)
Assignee Sangamo Therapeutics, Inc., Richmond, CA (US)

Claim of US Patent No. 9,963,715

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.