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Gene therapy for inherited metabolic diseases

Baruteau, J; Gurung, S; Perocheau, D; Counsell, J; (2020) Gene therapy for inherited metabolic diseases. Journal of Mother and Child , 24 (2) 10.34763/jmotherandchild.20202402si.2004.000009. Green open access

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Abstract

Over the last two decades, gene therapy has been successfully translated to many rare diseases. The number of clinical trials is rapidly expanding and some gene therapy products have now received market authorisation in the western world. Inherited metabolic diseases (IMD) are orphan diseases frequently associated with a severe debilitating phenotype with limited therapeutic perspective. Gene therapy is progressively becoming a diseasechanging therapeutic option for these patients. In this review, we aim to summarise the development of this emerging field detailing the main gene therapy strategies, routes of administration, viral and non-viral vectors and gene editing tools. We discuss the respective advantages and pitfalls of these gene therapy strategies and review their application in IMD, providing examples of clinical trials with lentiviral or adeno-associated viral gene therapy vectors in rare diseases. The rapid development of the field and implementation of gene therapy as a realistic therapeutic option for various IMD in a short term also require a good knowledge and understanding of these technologies from physicians to counsel the patients at best.

Type: Article
Title: Gene therapy for inherited metabolic diseases
Open access status: An open access version is available from UCL Discovery
DOI: 10.34763/jmotherandchild.20202402si.2004.000009
Publisher version: http://dx.doi.org/10.34763/jmotherandchild.2020240...
Language: English
Additional information: Copyright © 2020 Seker Yilmaz et al. This is an open-access article distributed under the terms of the Creative Commons Attribution 4.0 Public License (CC BY) (https://creativecommons.org/licenses/by/4.0/). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
Keywords: adeno-associated virus, gene therapy, inherited metabolic disease, lentivirus, messenger RNA, zinc finger nuclease
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 Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Surgery and Interventional Sci
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 > Genetics and Genomic Medicine Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10109887
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