作者
Ramona Bella, Rafal Kaminski, Pietro Mancuso, Won-Bin Young, Chen Chen, Rahsan Sariyer, Tracy Fischer, Shohreh Amini, Pasquale Ferrante, Jeffrey M Jacobson, Fatah Kashanchi, Kamel Khalili
发表日期
2018/9/7
期刊
Molecular Therapy-Nucleic Acids
卷号
12
页码范围
275-282
出版商
Elsevier
简介
We used NOD/SCID mice, also known as NRG, to assess the ability of lentivirus-mediated intravenous delivery of CRISPR in editing the HIV-1 genome from the circulating PBMC engrafts, some of which homed within several animal solid tissues. Lentivirus-mediated delivery of a multiplex of guide RNAs accompanied by Cas9 endonuclease led to the excision of the targeted region of the viral genome positioned within the HIV-1 LTR from the in-vitro-infected human peripheral blood mononuclear cells (PBMCs) embedded in the spleens of NRG mice. Similarly, the treatment of NRG mice harboring PBMC engrafts derived from HIV-1-positive patients with the therapeutic lentivirus eliminated the presence of the viral DNA fragment in the blood, as well as in the spleen, lung, and liver, of the engrafted animals. Sanger sequence analysis of the viral DNA after treatment with the lentiviral vectors expressing Cas9 and …
引用总数
201820192020202120222023202412523221577
学术搜索中的文章
R Bella, R Kaminski, P Mancuso, WB Young, C Chen… - Molecular Therapy-Nucleic Acids, 2018