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AAV-mediated gene therapy for rare metabolic disorders: turning a promise into a reality

Seker Yilmaz, Berna; Gissen, Paul; (2023) AAV-mediated gene therapy for rare metabolic disorders: turning a promise into a reality. The Biochemist , 45 (4) pp. 2-5. 10.1042/bio_2023_128. Green open access

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Abstract

Gene therapy is emerging as the realistic treatment option for inborn errors of metabolism (IEMs) and, with the promising safety and efficacy evidence from the proof-of-concept studies, adeno-associated virus (AAV) has become the frontrunner among viral vector candidates for these monogenic disorders. Different AAV capsids exhibit specific tissue tropisms, which can considerably increase the efficiency of gene transfer to particular organs. Here, we will discuss two distinct diseases: ornithine transcarbamylase (OTC) deficiency and Niemann–Pick disease type C, in which significant advances have been achieved in AAV-based gene therapy trials.

Type: Article
Title: AAV-mediated gene therapy for rare metabolic disorders: turning a promise into a reality
Open access status: An open access version is available from UCL Discovery
DOI: 10.1042/bio_2023_128
Publisher version: https://doi.org/10.1042/bio_2023_128
Language: English
Additional information: © The Authors. Published by Portland Press Limited under the Creative Commons Attribution License 4.0 (https://creativecommons.org/licenses/by-nc-nd/4.0/deed.en_GB).
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 > Genetics and Genomic Medicine Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10176720
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