UCL Discovery
UCL home » Library Services » Electronic resources » UCL Discovery

Gene Therapy for Primary Immunodeficiency

Houghton, BC; Booth, C; (2021) Gene Therapy for Primary Immunodeficiency. Hemasphere , 5 (1) , Article e509. 10.1097/HS9.0000000000000509. Green open access

[img]
Preview
Text
Gene_Therapy_for_Primary_Immunodeficiency.16.pdf - Published version

Download (1MB) | Preview

Abstract

Over the past 3 decades, there has been significant progress in refining gene therapy technologies and procedures. Transduction of hematopoietic stem cells ex vivo using lentiviral vectors can now create a highly effective therapeutic product, capable of reconstituting many different immune system dysfunctions when reinfused into patients. Here, we review the key developments in the gene therapy landscape for primary immune deficiency, from an experimental therapy where clinical efficacy was marred by adverse events, to a commercialized product with enhanced safety and efficacy. We also discuss progress being made in preclinical studies for challenging disease targets and emerging gene editing technologies that are showing promising results, particularly for conditions where gene regulation is important for efficacy.

Type: Article
Title: Gene Therapy for Primary Immunodeficiency
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1097/HS9.0000000000000509
Publisher version: https://doi.org/10.1097/HS9.0000000000000509
Language: English
Additional information: This is an open-access article distributed under the terms of the Creative Commons Attribution-Non Commercial-No Derivatives License 4.0 (CCBY-NC-ND), where it is permissible to download and share the work provided it is properly cited. The work cannot be changed in any way or used commercially without permission from the journal. https://creativecommons.org/licenses/by-nc-nd/4.0/
UCL classification: UCL
UCL > Provost and Vice Provost Offices
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 > 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.ucl.ac.uk/id/eprint/10118907
Downloads since deposit
9Downloads
Download activity - last month
Download activity - last 12 months
Downloads by country - last 12 months

Archive Staff Only

View Item View Item