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Methods and compositions for treatment of a genetic condition

  • US 9,963,715 B2
  • Filed: 08/29/2013
  • Issued: 05/08/2018
  • Est. Priority Date: 08/29/2012
  • Status: Active Grant
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.

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