Genome editing for primary immunodeficiencies: A therapeutic perspective on Wiskott-Aldrich syndrome.
Genome editing for primary immunodeficiencies: A therapeutic perspective on Wiskott-Aldrich syndrome.
复制标题
DOI：
10.3389/fimmu.2022.966084
复制
发表时间：
2022
影响因子：
7.3
通讯作者：
中科院分区：
文献类型：
--
作者：
原发免疫缺陷疾病(PIDs)是一组罕见的影响免疫系统的遗传性疾病，传统上可以通过异基因造血干细胞移植和实验性的自体基因治疗来治疗。由于这两种方法仍面临着重大挑战，基因编辑最近已成为治疗遗传疾病的一种潜在的有价值的替代方案，并且在其最初开发的相对较短的时间内，已经进入了一些旨在治疗几种危及生命的疾病的里程碑式的临床试验。在这篇综述中，我们讨论了基于基因编辑的PIDs治疗策略的发展进展，特别是Wiskott-Aldrich综合征，并概述了他们的主要挑战以及已经建立的治疗方法的未来方向。
Primary immunodeficiency diseases (PIDs) are a group of rare inherited disorders affecting the immune system that can be conventionally treated with allogeneic hematopoietic stem cell transplantation and with experimental autologous gene therapy. With both approaches still facing important challenges, gene editing has recently emerged as a potential valuable alternative for the treatment of genetic disorders and within a relatively short period from its initial development, has already entered some landmark clinical trials aimed at tackling several life-threatening diseases. In this review, we discuss the progress made towards the development of gene editing-based therapeutic strategies for PIDs with a special focus on Wiskott - Aldrich syndrome and outline their main challenges as well as future directions with respect to already established treatments.
登录
查看更多内容
DOI：
10.1016/j.jaci.2017.11.063
发表时间：
2018-11
期刊：
The Journal of allergy and clinical immunology
影响因子：
--
作者：
Cervantes-Luevano KE;Caronni N;Castiello MC;Fontana E;Piperno GM;Naseem A;Uva P;Bosticardo M;Marcovecchio GE;Notarangelo LD;Cicalese MP;Aiuti A;Villa A;Benvenuti F
通讯作者：
Benvenuti F
影响因子：
7.5
作者：
Barker, Juliet N.;Boughan, Kirsten;Politikos, Ioannis
通讯作者：
Politikos, Ioannis
影响因子：
17.1
作者：
Chan YK;Wang SK;Chu CJ;Copland DA;Letizia AJ;Costa Verdera H;Chiang JJ;Sethi M;Wang MK;Neidermyer WJ Jr;Chan Y;Lim ET;Graveline AR;Sanchez M;Boyd RF;Vihtelic TS;Inciong RGCO;Slain JM;Alphonse PJ;Xue Y;Robinson-McCarthy LR;Tam JM;Jabbar MH;Sahu B;Adeniran JF;Muhuri M;Tai PWL;Xie J;Krause TB;Vernet A;Pezone M;Xiao R;Liu T;Wang W;Kaplan HJ;Gao G;Dick AD;Mingozzi F;McCall MA;Cepko CL;Church GM
通讯作者：
Church GM
影响因子：
56.9
作者：
Boch, Jens;Scholze, Heidi;Bonas, Ulla
通讯作者：
Bonas, Ulla
影响因子：
64.8
作者：
Anzalone, Andrew V.;Randolph, Peyton B.;Liu, David R.
通讯作者：
Liu, David R.