Adenovirus vectors in hematopoietic stem cell genome editing.
Adenovirus vectors in hematopoietic stem cell genome editing.
复制标题
DOI：
10.1002/1873-3468.13668
复制
发表时间：
2019-12
期刊：
影响因子：
3.5
通讯作者：
Lieber, Andre
中科院分区：
文献类型：
--
作者：
Li, Chang;Lieber, Andre
关键词：
造血干细胞（hsc）的基因组编辑代表了许多血液遗传疾病的治疗选择，因为造血干细胞具有自我更新和分化为所有血细胞谱系的潜力。本文综述了利用腺病毒载体对造血干细胞进行基因组编辑的研究进展。我们关注的是衣壳修饰的、依赖辅助剂的腺病毒载体，它不含所有病毒基因，因此具有更高的安全性。我们讨论了几种遗传性疾病和传染病的HSC基因组工程，包括血红蛋白病、范可尼贫血、血友病和HIV-1感染，通过在转基因小鼠、非人灵长类动物以及人类CD34+细胞中进行体外和体内编辑。治疗性基因转移的机制包括设计核酸酶和碱基编辑器的外体表达，转座酶介导的随机整合，以及靶向同源定向修复引发的整合到选定的基因组安全港位点。
Genome editing of hematopoietic stem cells (HSCs) represents a therapeutic option for a number of hematological genetic diseases, as HSCs have the potential for self-renewal and differentiation into all blood cell lineages. This review presents advances of genome editing in HSCs utilizing adenovirus vectors as delivery vehicles. We focus on capsid-modified, helper-dependent adenovirus vectors that are devoid of all viral genes and therefore exhibit an improved safety profile. We discuss HSC genome engineering for several inherited disorders and infectious diseases including hemoglobinopathies, Fanconi anemia, hemophilia, and HIV-1 infection by ex vivo and in vivo editing in transgenic mice, nonhuman primates, as well as in human CD34+ cells. Mechanisms of therapeutic gene transfer including episomal expression of designer nucleases and base editors, transposase-mediated random integration, and targeted homology-directed repair triggered integration into selected genomic safe harbor loci are also reviewed.
登录
查看更多内容
DOI：
10.1016/j.omtn.2018.05.021
发表时间：
2018-09-07
期刊：
Molecular therapy. Nucleic acids
影响因子：
--
作者：
Bella R;Kaminski R;Mancuso P;Young WB;Chen C;Sariyer R;Fischer T;Amini S;Ferrante P;Jacobson JM;Kashanchi F;Khalili K
通讯作者：
Khalili K
影响因子：
3.7
作者：
Balamotis, MA;Huang, K;Mitani, K
通讯作者：
Mitani, K
影响因子：
3.8
作者：
Adams, William C.;Berenson, Ronald J.;Lore, Karin
通讯作者：
Lore, Karin
影响因子：
56.9
作者：
Boch, Jens;Scholze, Heidi;Bonas, Ulla
通讯作者：
Bonas, Ulla
影响因子：
16.6
作者：
Bahal, Raman;McNeer, Nicole Ali;Quijano, Elias;Liu, Yanfeng;Sulkowski, Parker;Turchick, Audrey;Lu, Yi-Chien;Bhunia, Dinesh C.;Manna, Arunava;Greiner, Dale L.;Brehm, Michael A.;Cheng, Christopher J.;Lopez-Giraldez, Francesc;Ricciardi, Adele;Beloor, Jagadish;Krause, Diane S.;Kumar, Priti;Gallagher, Patrick G.;Braddock, Demetrios T.;Saltzman, W. Mark;Ly, Danith H.;Glazer, Peter M.
通讯作者：
Glazer, Peter M.