Gene therapy and genome editing for primary immunodeficiency diseases

Zhi-Yong Zhang; Adrian J. Thrasher; Fang Zhang

doi:10.1016/j.gendis.2019.07.007

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Gene therapy and genome editing for primary immunodeficiency diseases

更新时间：2022-06-24

- Gene therapy and genome editing for primary immunodeficiency diseases
- Genes & Diseases Vol. 7, Issue 1, (2020)
- 作者机构：
  
  1. Department of Immunology and Rheumatology, Children's Hospital of Chongqing Medical University,China
  2. Molecular and Cellular Immunology, Great Ormond Street Institute of Child Health, University Colleage London,UK
- 作者简介：
- 基金信息：
- DOI：10.1016/j.gendis.2019.07.007
  CLC：
- Published：2020，
  
  Published Online：1 March 2020，
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Zhi-Yong Zhang, Adrian J. Thrasher, Fang Zhang. Gene therapy and genome editing for primary immunodeficiency diseases. [J]. Genes & Diseases 7(1).(2020)
DOI：

Zhi-Yong Zhang, Adrian J. Thrasher, Fang Zhang. Gene therapy and genome editing for primary immunodeficiency diseases. [J]. Genes & Diseases 7(1).(2020) DOI： 10.1016/j.gendis.2019.07.007.

摘要

Abstract

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.

关键词

Keywords

Adenosine deaminase deficientChronic granulomatous diseaseGene therapyGenome editingHematopoietic progenitor stem cellsPrimary immunodeficiency diseasesWiskott-Aldrich syndromeX-liked severe combined immunodeficiency

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