CONWAY, Anthony,LEE, Gary K.,PASCHON, David,ZHANG, Lei
申请号：
USUS2017/050446
公开号：
WO2018/049009A3
申请日:
2017.09.07
申请国别(地区):
US
年份:
2018
代理人:
摘要：
Described herein are compositions and methods for modulation of gene expression in the liver including modulation of PCSK9, TTR, SERPINA1, KLKB1 and/or HA01. Specifically, the disclosure provides methods for modulating the expression of the PCSK9, TTR, SERPINA1, KLKB1 and/or HA01 genes in the liver using one or more engineered nucleases.L'invention concerne des compositions et des procédés de modulation de l'expression génique dans le foie notamment la modulation de PCSK9, TTR, SERPINA1, KLKB1 et/ou HAO1.
