Gene editing of pcsk9

An editing and encoding technology, applied in genetic engineering, metabolic diseases, extracellular fluid diseases, etc., can solve problems such as genome instability and off-target effects
CN110352242APending Publication Date: 2019-10-18PRESIDENT & FELLOWS OF HARVARD COLLEGE
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Patent Information

Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
PRESIDENT & FELLOWS OF HARVARD COLLEGE
Publication Date
2019-10-18

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Abstract

Provided herein are systems, compositions, and methods of introducing loss-of-function mutations in to protein factors involved in the LDL-R-mediated cholesterol clearance pathway, e.g., PCSK9, APOC3,LDL-R, or IDOL. Loss-of-function mutations may be introduced using a CRISPR / Cas9-based nucleobase editor described in. Further provided herein are compositions and methods of treating conditions related to high circulating cholesterol levels.
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Description

[0001] related application

[0002] This application claims priority under 35 U.S.C. § 119(e) to U.S. Provisional Application U.S.S.N. 62 / 438,869, filed December 23, 2016, which is incorporated herein by reference.

[0003] governmental support

[0004] This invention was made with Government support under Grant No. GM065865 awarded by the National Institutes of Health. The government has certain rights in this invention.

[0005] Background of the invention

[0006] Hepatic proprotein convertase subtilisin / Kexin type 9 (PCSK9) is a secreted, globular, autoactivating serine protease that functions as a protein-binding linker within endosomal vesicles to bind low-density lipoprotein (LDL ) bridges a pH-dependent interaction with the LDL receptor (LDL-R) during endocytosis of granules, thereby preventing recycling of LDL-R to the cell surface and resulting in reduced LDL-cholesterol clearance. Blocking or inhibiting the function of PCSK9 to enhance LDL-R-mediated clearance of ...

Claims

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