Methods and compositions for treatment of a genetic condition
First Claim
Patent Images
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 Assignments
0 Petitions
Accused Products
Abstract
Disclosed herein are nucleases and methods of using these nucleases for alteration of a globin gene and generation of cells.
-
Citations
11 Claims
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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); andF6;
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); andF6;
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); andF6;
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); andF6;
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); andF6;
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); andF6;
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); andF6;
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); andF6;
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); andF6;
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); andF6;
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); andF5;
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); andF5;
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); andF6;
QSGDLTR (SEQ ID NO;
13), wherein the zinc finger protein binds to a target site as set forth in SEQ ID NO;
71. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11)
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Specification