Vectors encoding a glucose-6-phosphatase (G6PASE-A) for gene therapy
AssigneesGenethon, Institut National De La Sante Et De La Recherche Medicale (Inserm), Universite D'Evry Val D'Essonne, Universite Claude Bernard Lyon
InventorsLouisa Jauze, Fabienne Rajas, Giuseppe Ronzitti
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Abstract
The invention relates to an adeno-associated virus (AAV) vector comprising a nucleic acid construct for the expression of a glucose-6-phosphatase-a (G6Pase-a) in a cell, the construct comprising a nucleic acid sequence encoding the G6Pase-a, wherein the nucleic acid sequence encoding the G6Pase-a is operably linked to a human alpha-1 antitrypsin (hAAT) promoter, a cell transformed with the vector of the invention, a composition comprising the vector or the cell of the invention, and the use thereof.
Claims22
An adeno-associated virus (AAV) vector comprising a nucleic acid construct for the expression of a glucose-6-phosphatase-a (G6Pase-a) in a cell, the construct comprising a nucleic acid sequence encoding the G6Pase-a, wherein the nucleic acid sequence encoding the G6Pase-a is operably linked to a human alpha-1 antitrypsin (hAAT) promoter, wherein the nucleic acid sequence encoding the G6Pase-a is codon optimized by decreasing the content of GC and decreasing GC dimers in said nucleic acid sequence encoding the G6Pase-a.
Dependencies are inferred from the claim text.