Miniaturized dystrophins having spectrin fusion domains and uses thereof
AssigneeBristol-Myers Squibb Company
InventorsGlen B. Banks, Jonathan Harry Davis, Paul Charles Levesque
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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.
Claims73
A nucleic acid molecule comprising a nucleotide sequence, which encodes a miniaturized dystrophin polypeptide comprising a modified spectrin repeat 16 (R16) domain, wherein a part of spectrin repeat 16 (R16) domain is replaced by a corresponding part of a different spectrin repeat domain.
A nucleic acid molecule comprising a nucleotide sequence, which encodes a miniaturized dystrophin polypeptide comprising an amino acid sequence of SEQ ID NO:
- 83.
A nucleic acid molecule comprising a nucleotide sequence comprising in order a C5-12(T) promoter of SEQ ID NO:
- 109, an SV40 intron of SEQ ID NO:
- 110, a coding sequence for miniaturized dystrophin BXA-220931 of SEQ ID NO:
- 111, a 3′ UTR of SEQ ID NO:
- 112, and a polyA sequence of SEQ ID NO:
- 113.
Dependencies are inferred from the claim text.