CONWAY, ANTHONY,LEE, GARY K.,PASCHON, DAVID,ZHANG, LEI
申请号：
CA3035534
公开号：
CA3035534A1
申请日:
2017.09.07
申请国别(地区):
CA
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.
