Patent ID: 11898158
Assignee: SANGAMO THERAPEUTICS, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 0:
1. A method of treating a mouse or human with Gaucher's, Fabry's, or Pompe's disease, the method comprising
intravenously injecting first and second AAV vectors encoding first and second ZFNs of a pair of zinc finger nucleases that cleave in an endogenous albumin gene, into the mouse or human with Gaucher's, Fabry's, or Pompe's disease;
intravenously injecting a third AAV vector comprising a transgene encoding:
(a) a glucocerebrosidase (GBA) protein into the mouse or human with Gaucher's disease;
(b) an α galactosidase deficiency (GLA) protein into the mouse or human with Fabry's disease;
(c) an alpha-glucosidase (GAA) protein into the mouse or human with Pompe's disease; or
wherein the transgene is flanked by sequences having homology with the endogenous albumin gene;
wherein the first, second and third AAV vectors are delivered at a ratio of 1:1:8 and further wherein the transgene is integrated into the endogenous albumin gene in liver cells of the mouse or human and the liver cells express and secrete therapeutic amounts of the protein and a symptom of the Gaucher's, Fabry's, or Pompe's disease is treated in the mouse or human.