Compositions and methods for treating laminopathies
AssigneeEncoded Therapeutics, Inc.
InventorsSirika Wood, Kartik Ramamoorthi, Stephanie Tagliatela, Anne Tanenhaus
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Abstract
The present disclosure provides compositions and methods for treating, preventing, or inhibiting laminopathies. In one aspect, the disclosure provides nucleic acid constructs and/or vectors comprising a nucleotide sequence encoding lamin A and/or lamin C.
Claims29
A nucleic acid construct comprising a nucleotide sequence encoding a lamin A polypeptide and a lamin C polypeptide, wherein said construct comprises introns 8-11 of the wildtype human LMNA gene and lacks introns 1-7 of the wildtype-human LMNA gene, wherein the lamin A polypeptide comprises an amino acid sequence that is at least 95% identical to the amino acid sequence of SEQ ID NO:
- 12 or SEQ ID NO:
- 21, and wherein said lamin C polypeptide comprises an amino acid sequence that is at least 95% identical to the amino acid sequence of SEQ ID NO:
- 13.
Dependencies are inferred from the claim text.