Back to results
US 2025/0205367 A1Published application
Compositions comprising a muscle active promoter and methods of using the same
AssigneeDuke University
InventorsDwight Koeberl, Baodong Sun, Benjamin Arnson
Life of this patent
Estimating the term…
Abstract
Disclosed herein are novel promoters that drive expression of a gene product preferentially in the heart and in skeletal muscle. Disclosed herein are vectors comprising these novel promoters and vectors comprising these novel promoters. Disclosed herein are methods of gene editing and gene therapy that employ these novel promoters and novel vectors.
Claims20
A glucose-6-phosphatase (G6PC) minimal promoter comprising the sequence set forth in SEQ ID NO:02 or SEQ ID NO:03.
A gene editing system, comprising:
- a viral vector comprising a glucose-6-phosphatase (G6PC)minimal promoter operably linked to a nucleic acid sequence encoding a transgene, a nucleic acid sequence encoding a guide RNA (gRNA) targeting a gene locus having one or more defects, a second promoter operably linked to the gRNA, and a pair of sequences flanking the nucleic acid sequence encoding the transgene,
- wherein the pair of sequences are homologous to a portion of the gene locus having one or more defects;
- a viral vector comprising a G6PC minimal promoter operably linked to a nucleic acid sequence encoding a Cas9 endonuclease.
Dependencies are inferred from the claim text.