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Title:
PCSK9の塩基編集および疾患の処置のためにこれを使用する方法
Document Type and Number:
Japanese Patent JP7457832
Kind Code:
B2
Abstract:
Provided herein are compositions for gene modification or editing and methods of using same to treat or prevent certain conditions. Specific compositions and methods capable of safely and effectively editing gene targets expressed in the liver to durably lower LDL-C thereby treating a leading cause of cardiovascular disease are disclosed.

Inventors:
Chadwick, Alexandra
Rajeev, Karantota Chil Gee
Rhode, Ellen
Chen, Christopher
Race, Caroline
Application Number:
JP2022562027A
Publication Date:
March 28, 2024
Filing Date:
April 09, 2021
Export Citation:
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Assignee:
Verve Therapeutics, Inc.
International Classes:
C12N15/09; C12N15/113; C12N15/62
Domestic Patent References:
JP2015514076A
JP2016530294A
Foreign References:
WO2019025431A1
WO2019217941A1
WO2019213183A1
Other References:
Gaudelli, N. M. et al.,"Directed evolution of adenine base editors with increased activity and therapeutic application",Nat. Biotechnol.,2020年04月13日,Vol. 38,pp. 892-900, Methods,SUPPLEMENTARY INFORMATION
Attorney, Agent or Firm:
Osamu Yamamoto
Toru Miyamae
Junichi Matsuo
Takumi Terachi