Methods and compositions for the treatment of fabry disease
AssigneeSangamo Therapeutics, Inc.
InventorMarshall W. Huston
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Abstract
Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic α-Gal A protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of Fabry disease.
Claims20
A pharmaceutical composition comprising an adeno-associated virus (AAV) expression construct, wherein the AAV expression construct comprises an apolipoprotein E (APOE) enhancer operably linked to an alpha 1-antitrypsin (hAAT) promoter, a human hemoglobin beta (HBB)-IGG intron, and a cDNA transgene encoding at least one a galactosidase A (α-Gal A) protein, and a pharmaceutically acceptable carrier or excipient.
An adeno-associated virus (AAV) expression construct, wherein the AAV expression construct comprises an apolipoprotein E (APOE) enhancer operably linked to an alpha 1-antitrypsin (hAAT) promoter, a human hemoglobin beta (HBB)-IGG intron, and a cDNA transgene encoding at least one a galactosidase A (α-Gal A) protein.
The AAV expression construct of 6, wherein the AAV expression construct further comprises a poly A signal sequence.
An adeno-associated virus (AAV) expression construct comprising an apolipoprotein E (APOE) enhancer linked to an alpha 1-antitrypsin (hAAT) promoter, a human hemoglobin beta (HBB)-IGG intron, a sequence encoding an α-Gal A signal peptide, a cDNA transgene encoding at least one alpha-galactosidase A (α-Gal A) protein, and a bovine growth hormone poly A signal sequence.
Dependencies are inferred from the claim text.