Zhang, ZY;
Thrasher, AJ;
Zhang, F;
(2019)
Gene therapy and genome editing for primary immunodeficiency diseases.
Genes and Diseases
10.1016/j.gendis.2019.07.007.
(In press).
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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.
| Type: | Article |
|---|---|
| Title: | Gene therapy and genome editing for primary immunodeficiency diseases |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1016/j.gendis.2019.07.007 |
| Publisher version: | https://doi.org/10.1016/j.gendis.2019.07.007 |
| Language: | English |
| Additional information: | This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/). |
| 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 |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept |
| URI: | https://discovery-pp.ucl.ac.uk/id/eprint/10083455 |
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