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US 2014/0112896 A1Published application
Methods and compositions for the provision of proteins deficient in lysosomal storage diseases
AssigneeSangqmo BioSciences, Inc.
InventorEdward J. Rebar
Life of this patent
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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.
Claims16
A method for generating a cell that produces a protein deficient in a lysosomal storage disease, the method comprising:
- integrating a transgene encoding the protein into an endogenous locus of the cell using a non-naturally occurring nuclease, such that the cell produces the protein that is deficient in the lysosomal storage disease.
Dependencies are inferred from the claim text.