Gene therapy and genome editing for primary immunodeficiency diseases
作者机构：Molecular and Cellular ImmunologyGreat Ormond Street Institute of Child HealthUniversity Colleage LondonUK Department of Immunology and RheumatologyChildren’s Hospital of Chongqing Medical UniversityChina
出 版 物：《Genes & Diseases》 (基因与疾病（英文）)
年 卷 期：2020年第7卷第1期
页 面：38-51页
核心收录：
学科分类：0710[理学-生物学] 0831[工学-生物医学工程（可授工学、理学、医学学位）] 1002[医学-临床医学] 1001[医学-基础医学(可授医学、理学学位)] 100102[医学-免疫学] 0703[理学-化学] 10[医学]
基 金：We thank Dr Alessia Cavazza for helping in the manuscript correction.FZ is supported by the Wellcome Trust(104807/Z/14/Z) ZYZ is supported by National Natural Science Foundation of China(NO.81202316) Foundation from Children’s Hospital of Chongqing Medical University.AJT is supported by both the Wellcome Trust(104807/Z/14/Z) by the National Institute for Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London.
主 题：Adenosine deaminase deficient Chronic granulomatous disease Gene therapy Genome editing Hematopoietic progenitor stem cells Primary immunodeficiency diseases Wiskott-Aldrich syndrome X-liked severe combined immunodeficiency
摘 要：In past two decades the gene therapy using genetic modified autologous hematopoietic stem cells(HSCs)transduced with the viral vector has become a promising alternative option for treating primary immunodeficiency diseases(PIDs).Despite of some pitfalls at early stage clinical trials,the field of gene therapy has advanced significantly in the last decade with improvements in viral vector safety,preparatory regime for manufacturing high quality virus,automated CD34 cell purification.Hence,the overall outcome from the clinical trials for the different PIDs has been very encouraging.In addition to the viral vector based gene therapy,the recent fast moving forward developments in genome editing using engineered nucleases in HSCs has provided a new promising platform for the treatment of PIDs.This review provides an overall outcome and progress in gene therapy clinical trials for SCID-X,ADA-SCID,WAS,X-CGD,and the recent developments in genome editing technology applied in HSCs for developing potential therapy,particular in the key studies for PIDs.
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