Microdystrophin nucleic acid gene therapy constructs and uses thereof
AssigneeREGENXBIO Inc.
InventorsChunping Qiao, Devin McDougald, Ye Liu, Olivier Danos
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Abstract
Provided are gene constructs that encode a microdystrophin protein for use in gene therapy. The microdystrophin gene constructs and expression cassettes were engineered for improved therapy with respect to efficacy, potency and safety to the subject when expressed by a viral vector in muscle cells and/or CNS cells.
Claims33
A nucleic acid comprising a nucleotide sequence encoding a microdystrophin protein comprising the amino acid sequence of SEQ ID NO:
- 79, or the reverse complement of the nucleotide sequence.
A nucleic acid comprising a nucleotide sequence encoding a microdystrophin protein comprising the amino acid sequence of SEQ ID NO:
- 1, or the reverse complement of the nucleotide sequence.
A plasmid comprising a nucleic acid comprising a nucleotide sequence encoding a microdystrophin protein comprising the amino acid sequence of SEQ ID NO:
- 1, or the reverse complement of the nucleotide sequence.
A host cell comprising a plasmid comprising a nucleic acid comprising a nucleotide sequence encoding a microdystrophin protein comprises the amino acid sequence of SEQ ID NO:
- 1, or the reverse complement of the nucleotide sequence.
A nucleic acid comprising the nucleotide sequence of SEQ ID NO:
- 53.
A plasmid comprising a nucleic acid comprising the nucleotide sequence of SEQ ID NO:
- 53.
A host cell comprising a plasmid comprising a nucleic acid comprising the nucleotide sequence of SEQ ID NO:
- 53.
Dependencies are inferred from the claim text.