Miniaturized dystrophins having spectrin fusion domains 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.
Claims26
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 the R16 domain is replaced by a corresponding part of a spectrin repeat 2 (R2) domain, and wherein the miniaturized dystrophin polypeptide comprises an amino acid sequence at least about 98% identical to the amino acid sequence set forth as SEQ ID NO:
- 83.
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 a modified R16 domain, wherein a part of the R16 domain is replaced by a corresponding part of a R2 domain, and wherein the miniaturized dystrophin polypeptide comprises an amino acid sequence at least about 98% identical to the amino acid sequence set forth as SEQ ID NO:
- 83.
A rAAV vector particle comprising an AAV capsid and a nucleic acid molecule comprising a nucleotide sequence comprising in order a C5-12(T) promoter having the nucleotide sequence set forth as SEQ ID NO:
- 109, an SV40 intron having the nucleotide sequence set forth as SEQ ID NO:
- 110, a coding sequence for a miniaturized dystrophin polypeptide having the nucleotide sequence set forth as SEQ ID NO:
- 111, a 3′ UTR having the nucleotide sequence set forth as SEQ ID NO:
- 112, and a polyA sequence having the nucleotide sequence set forth as SEQ ID NO:
- 113, wherein the nucleic acid molecule further comprises a first ITR and a second ITR both from the genome of AAV2 flanking the nucleotide sequence, and wherein the AAV capsid is from AAV8 or AAV9.
Dependencies are inferred from the claim text.