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US 9,956,247 B2Granted
Method of treating lysosomal storage diseases
AssigneeSangamo Therapeutics, Inc.
InventorEdward J. Rebar
Life of this patent
Estimating the term…
Abstract
Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for the provision of proteins lacking or deficient in subjects with a lysosomal storage disease and treatment and/or prevention of lysosomal storage diseases.
Claims4
A method for expressing and secreting exogenous glucocerebrosidase (GBA), α galactosidase (GLA), iduronate-2-sulfatase (IDS) or alpha-L iduronidase (IUDA) protein in a liver cell of a human or a mouse in need thereof, the method comprising:
- (a) intravenously administering one or more AAV vectors encoding a pair of zinc finger nucleases that cleaves in intron 1 or 12 of an endogenous albumin gene into said human or mouse containing the liver cell and disrupts expression of the albumin gene;
- and
- (b) intravenously administering an AAV vector comprising a donor sequence comprising a transgene encoding:
- (i) an exogenous GBA protein;
- (ii) an exogenous GLA protein;
- (iii) an exogenous IDS protein;
- or (iv) an exogenous IDUA protein, into the human or mouse liver cell of said human or mouse, wherein the transgene encoding the protein is flanked by regions of homology to intron 1 or 12 of the cleaved endogenous albumin gene, such that the transgene is integrated by homology directed repair into intron 1 or 12 of the cleaved endogenous albumin gene, and the liver cell expresses and secretes the exogenous GBA, GLA, IDS, or IDUA protein.
4 more elements
Dependencies are inferred from the claim text.