Gene therapy and genome editing for primary immunodeficiency diseases
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...
| Main Authors: | , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
KeAi Communications Co., Ltd.
2020-03-01
|
| Series: | Genes and Diseases |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2352304219300510 |
| _version_ | 1827267296318455808 |
|---|---|
| author | Zhi-Yong Zhang Adrian J. Thrasher Fang Zhang |
| author_facet | Zhi-Yong Zhang Adrian J. Thrasher Fang Zhang |
| author_sort | Zhi-Yong Zhang |
| collection | DOAJ |
| description | 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. Keywords: 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 |
| first_indexed | 2024-03-12T07:06:22Z |
| format | Article |
| id | doaj.art-a05b5bdc0a054ba89f5ab09fae0fa2ca |
| institution | Directory Open Access Journal |
| issn | 2352-3042 |
| language | English |
| last_indexed | 2025-03-22T04:31:29Z |
| publishDate | 2020-03-01 |
| publisher | KeAi Communications Co., Ltd. |
| record_format | Article |
| series | Genes and Diseases |
| spelling | doaj.art-a05b5bdc0a054ba89f5ab09fae0fa2ca2024-04-28T03:27:09ZengKeAi Communications Co., Ltd.Genes and Diseases2352-30422020-03-01713851Gene therapy and genome editing for primary immunodeficiency diseasesZhi-Yong Zhang0Adrian J. Thrasher1Fang Zhang2Department of Immunology and Rheumatology, Children's Hospital of Chongqing Medical University, ChinaMolecular and Cellular Immunology, Great Ormond Street Institute of Child Health, University Colleage London, UKMolecular and Cellular Immunology, Great Ormond Street Institute of Child Health, University Colleage London, UK; Corresponding author.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. Keywords: Adenosine deaminase deficient, Chronic granulomatous disease, Gene therapy, Genome editing, Hematopoietic progenitor stem cells, Primary immunodeficiency diseases, Wiskott-Aldrich syndrome, X-liked severe combined immunodeficiencyhttp://www.sciencedirect.com/science/article/pii/S2352304219300510 |
| spellingShingle | Zhi-Yong Zhang Adrian J. Thrasher Fang Zhang Gene therapy and genome editing for primary immunodeficiency diseases Genes and Diseases |
| title | Gene therapy and genome editing for primary immunodeficiency diseases |
| title_full | Gene therapy and genome editing for primary immunodeficiency diseases |
| title_fullStr | Gene therapy and genome editing for primary immunodeficiency diseases |
| title_full_unstemmed | Gene therapy and genome editing for primary immunodeficiency diseases |
| title_short | Gene therapy and genome editing for primary immunodeficiency diseases |
| title_sort | gene therapy and genome editing for primary immunodeficiency diseases |
| url | http://www.sciencedirect.com/science/article/pii/S2352304219300510 |
| work_keys_str_mv | AT zhiyongzhang genetherapyandgenomeeditingforprimaryimmunodeficiencydiseases AT adrianjthrasher genetherapyandgenomeeditingforprimaryimmunodeficiencydiseases AT fangzhang genetherapyandgenomeeditingforprimaryimmunodeficiencydiseases |