Long-term in vivo editing of PCSK9 in NHPs
收藏NIAID Data Ecosystem2026-03-12 收录
下载链接：
https://www.ncbi.nlm.nih.gov/bioproject/PRJNA695573
下载链接
链接失效反馈官方服务：
资源简介：
Knockdown of PCSK9 expression is a suitable approach to treat hypercholesterolemia. We sought to determine the efficacy and safety of PCSK9 gene disruption by a DNA sequence specific meganuclease delivered by adeno-associated virus (AAV). Ten NHPs administered AAV-meganuclease using two different AAV serotypes or in combination with corticosteroid treatment were monitored for a period of up to three years. This study demonstrates that targeted in vivo gene disruption exerts a lasting therapeutic effect and provides data for safety considerations regarding off-target editing.
创建时间：
2021-01-28
