Therapeutic nucleic acids, peptides and uses i
OwnerMark ISALAN, Michal MIELCAREKinventors
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Abstract
Disclosed herein are polypeptides for use in treating diseases associated with pathogenic genomic repeat sequences, such as neurological disorders. Also disclosed are nucleic acid molecules and vectors that encode such polypeptides. Therapeutic uses and methods for treating such diseases are also disclosed; in particular, therapeutic uses and methods comprising complementary pairs and combinations of therapeutic polypeptides, nucleic acids or vectors. Also disclosed is a method and associated peptides and nucleic acids for active, long-term delivery of therapeutic molecules to target cells in vivo or in vitro.
Claims22
A polypeptide comprising a zinc finger peptide having from 8 to 32 zinc finger domains (F1 to F32) according to Formula 2:
- X0-2 C X1-5 C X2-7 X−1 X+1 X+2 X+3 X+4 X+5 X+6 H X3-6 H/c where X is any amino acid, the numbers in subscript indicate the number of residues represented by X, and the numbers in superscript indicate a position of the amino acid in the α-helix;
- wherein the polypeptide binds to a 5′-GGGGCC-3′ nucleic acid repeat sequence;
- and at least 8 adjacent zinc finger domains, F1 to F8, have a recognition sequence X−1 X+1 X+2 X+3 X+4 X+5 X+6 according to the following pattern:
- F2, F4, F6, F8,
- F3, F5, F7, F9,
- F1
- F10, . . . F32
- F11, . . . F31
- ZFP I:
- SEQ ID NO:
- 8
- SEQ ID NO:
- 3
- SEQ ID NO:
- 8
- ZFP J:
- SEQ ID NO:
- 8
- SEQ ID NO:
- 3
- SEQ ID NO:
- 10
- ZFP K:
- SEQ ID NO:
- 10
- SEQ ID NO:
- 3
- SEQ ID NO:
- 10
- ZFP L:
- SEQ ID NO:
- 9
- SEQ ID NO:
- 3
- SEQ ID NO:
- 9
- ZFP M:
- SEQ ID NO:
- 9
- SEQ ID NO:
- 3
- SEQ ID NO:
- 11
- ZFP N:
- SEQ ID NO:
- 11
- SEQ ID NO:
- 3
- SEQ ID NO:
- 11
- ZFP W:
- SEQ ID NO:
- 8
- SEQ ID NO:
- 12
- SEQ ID NO:
- 8
- ZFP X:
- SEQ ID NO:
- 8
- SEQ ID NO:
- 12
- SEQ ID NO:
- 10
- ZFP Y:
- SEQ ID NO:
- 10
- SEQ ID NO:
- 12
- SEQ ID NO:
- 10
- ZFP Z:
- SEQ ID NO:
- 9
- SEQ ID NO:
- 12
- SEQ ID NO:
- 9
- ZFP AA:
- SEQ ID NO:
- 9
- SEQ ID NO:
- 12
- SEQ ID NO:
- 11
- ZFP AB:
- SEQ ID NO:
- 11
- SEQ ID NO:
- 12
- SEQ ID NO:
- 11
- ZFP AI:
- SEQ ID NO:
- 3
- SEQ ID NO:
- 8
- SEQ ID NO:
- 3
- ZFP AJ:
- SEQ ID NO:
- 3
- SEQ ID NO:
- 9
- SEQ ID NO:
- 3
- ZFP AK:
- SEQ ID NO:
- 3
- SEQ ID NO:
- 10
- SEQ ID NO:
- 3
- ZFP AL:
- SEQ ID NO:
- 3
- SEQ ID NO:
- 11
- SEQ ID NO:
- 3
- ZFP AS:
- SEQ ID NO:
- 12
- SEQ ID NO:
- 8
- SEQ ID NO:
- 12
- ZFP AT:
- SEQ ID NO:
- 12
- SEQ ID NO:
- 9
- SEQ ID NO:
- 12
- ZFP AU:
- SEQ ID NO:
- 12
- SEQ ID NO:
- 10
- SEQ ID NO:
- 12
- ZFP AV:
- SEQ ID NO:
- 12
- SEQ ID NO:
- 11
- SEQ ID NO:
- 12
- ZFP JX:
- SEQ ID NO:
- 181
- SEQ ID NO:
- 12
- SEQ ID NO:
- 181
- ZFP JY:
- SEQ ID NO:
- 182
- SEQ ID NO:
- 12
- SEQ ID NO:
- 183
- ZFP JZ:
- SEQ ID NO:
- 182
- SEQ ID NO:
- 12
- SEQ ID NO:
- 183
- ZFP KA:
- SEQ ID NO:
- 181
- SEQ ID NO:
- 133
- SEQ ID NO:
- 181
- ZFP KB:
- SEQ ID NO:
- 182
- SEQ ID NO:
- 133
- SEQ ID NO:
- 182
- ZFP KC:
- SEQ ID NO:
- 182
- SEQ ID NO:
- 133
- SEQ ID NO:
- 183
- ZFP KD:
- SEQ ID NO:
- 181
- SEQ ID NO:
- 134
- SEQ ID NO:
- 181
- ZFP KE:
- SEQ ID NO:
- 182
- SEQ ID NO:
- 134
- SEQ ID NO:
- 182
- ZFP KF:
- SEQ ID NO:
- 182
- SEQ ID NO:
- 134
- SEQ ID NO:
- 183
- ZFP KG:
- SEQ ID NO:
- 12
- SEQ ID NO:
- 181
- SEQ ID NO:
- 134
- ZFP KH:
- SEQ ID NO:
- 12
- SEQ ID NO:
- 182
- SEQ ID NO:
- 134
- ZFP KI:
- SEQ ID NO:
- 12
- SEQ ID NO:
- 183
- SEQ ID NO:
- 134
- ZFP KJ:
- SEQ ID NO:
- 133
- SEQ ID NO:
- 181
- SEQ ID NO:
- 134
- ZFP KK:
- SEQ ID NO:
- 133
- SEQ ID NO:
- 182
- SEQ ID NO:
- 134
- ZFP KL:
- SEQ ID NO:
- 133
- SEQ ID NO:
- 183
- SEQ ID NO:
- 134
- ZFP LP:
- SEQ ID NO:
- 184
- SEQ ID NO:
- 3
- SEQ ID NO:
- 184
- ZFP LQ:
- SEQ ID NO:
- 185
- SEQ ID NO:
- 3
- SEQ ID NO:
- 185
- ZFP LR:
- SEQ ID NO:
- 186
- SEQ ID NO:
- 3
- SEQ ID NO:
- 186
- ZFP LS:
- SEQ ID NO:
- 184
- SEQ ID NO:
- 12
- SEQ ID NO:
- 184
- ZFP LT:
- SEQ ID NO:
- 185
- SEQ ID NO:
- 12
- SEQ ID NO:
- 185
- ZFP LU:
- SEQ ID NO:
- 186
- SEQ ID NO:
- 12
- SEQ ID NO:
- 186.
A polypeptide comprising a zinc finger peptide having from 5 to 7 zinc finger domains (F1 to F7) according to Formula 2:
- X0-2 C X1-5 C X2-7 X−1 X+1 X+2 X+3 X+4 X+5 X+6 H X3-6 H/C where X is any amino acid, the numbers in subscript indicate the number of residues represented by X, and numbers in superscript indicate a position of the amino acid in the α-helix;
- wherein the polypeptide binds to a 5′-GGGGCC-3′ nucleic acid repeat sequence;
- and
- the zinc finger domains have a recognition sequence X−1 X+1 X+2 X+3 X+4 X+5 X+6 according to the following pattern:
- F1
- F2, F4, F6
- F3, F5, F7
- ZFP GO:
- SEQ ID NO:
- 109
- SEQ ID NO:
- 108
- SEQ ID NO:
- 109
- ZFP GN:
- SEQ ID NO:
- 109
- SEQ ID NO:
- 108
- SEQ ID NO:
- 110
- ZFP FW:
- SEQ ID NO:
- 107
- SEQ ID NO:
- 109
- SEQ ID NO:
- 107
- ZFP FV:
- SEQ ID NO:
- 107
- SEQ ID NO:
- 109
- SEQ ID NO:
- 108.
(canceled)
(canceled)
Dependencies are inferred from the claim text.