Miniaturized dystrophins and uses thereof
AssigneeBristol-Myers Squibb Company
InventorsGlen Banks, Jonathan Harry Davis, Paul Charles Levesque
Life of this patent
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Abstract
Disclosed herein are nucleic acid molecules, polypeptides, cells, vectors, and pharmaceutical compositions relating to miniaturized dystrophin. Methods of production and methods of therapeutic use of the miniaturized dystrophin are also disclosed.
Claims27
A nucleic acid molecule comprising a nucleotide sequence, which encodes a miniaturized dystrophin polypeptide comprising from N terminus to C terminus hinge 1 (H1) domain, spectrin repeat 1 (R1) domain, spectrin repeat 3 (R3) domain, hinge 2 (H2) domain, spectrin repeat 16 (R16) domain, spectrin repeat 17 (R17) domain, spectrin repeat 24 (R24) domain, and hinge 4 (H4) domain of dystrophin, wherein the miniaturized dystrophin polypeptide does not comprise spectrin repeat 2 (R2) of dystrophin, wherein the R1 domain and the R3 domain are fused by amino acids ARGVAL (RV) and wherein the H2 domain and the R16 domain are fused by the amino acid sequence set forth in SEQ ID NOs:
- 74 or 75, and wherein (i) the H1 domain and the R1 domain are fused directly, (ii) the R3 domain and the H2 domain are fused directly, (iii) the R16 and R17 domains are fused directly, (iv) the R17 and R24 domains are fused directly, and (v) the R24 and H4 domains are fused directly.
A recombinant adeno-associated virus (rAAV) vector particle comprising an AAV capsid and a nucleic acid molecule comprising a nucleotide sequence, which encodes a miniaturized dystrophin polypeptide comprising the amino acid sequence of SEQ ID NO:
- 118.
Dependencies are inferred from the claim text.