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US 2018/0064827 A1Published application
Modulation of liver genes
AssigneeSangamo Therapeutics, Inc.
InventorsAnthony Conway, Gary K. Lee, David Paschon, Lei Zhang
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Abstract
Described herein are compositions and methods for modulation of gene expression in the liver including modulation of PCSK9, TTR, SERPINA1, KLKB1 and/or HAO1.
Claims21
A liver cell in which expression of an endogenous PCSK9, TTR, SERPINA1, KLKB1 and/or HAO1 gene is altered as compared to wild-type.
A fusion molecule comprising a DNA-binding domain that binds to a target site in an endogenous PCSK9, TTR, SERPINA1, KLKB1 and/or HAO1 gene and a functional domain.
Dependencies are inferred from the claim text.