Compositions and methods to program therapeutic cells using targeted nucleic acid nanocarriers
申请人:FRED HUTCHINSON CANCER RESEARCH CENTER
公开号:US10188749B2
公开(公告)日:2019-01-29
Compositions and methods that rapidly and selectively modify hematopoietic stem cells (or cells derived therefrom) to achieve therapeutic objectives by providing for transient expression of nucleic acids are described. The transient expression leads to permanent therapeutic changes in the modified cells, referred to herein as “hit and run” effects.
本文描述了通过提供核酸的瞬时表达,快速、选择性地改变造血干细胞(或其衍生细胞)以达到治疗目的的组合物和方法。瞬时表达可导致被修饰细胞发生永久性治疗变化,在此称为 "一击即中 "效应。