Seker Yilmaz, Berna;
Gissen, Paul;
(2023)
Genetic Therapy Approaches for Ornithine Transcarbamylase Deficiency.
Biomedicines
, 11
(8)
, Article 2227. 10.3390/biomedicines11082227.
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Abstract
Ornithine transcarbamylase deficiency (OTCD) is the most common urea cycle disorder with high unmet needs, as current dietary and medical treatments may not be sufficient to prevent hyperammonemic episodes, which can cause death or neurological sequelae. To date, liver transplantation is the only curative choice but is not widely available due to donor shortage, the need for life-long immunosuppression and technical challenges. A field of research that has shown a great deal of promise recently is gene therapy, and OTCD has been an essential candidate for different gene therapy modalities, including AAV gene addition, mRNA therapy and genome editing. This review will first summarise the main steps towards clinical translation, highlighting the benefits and challenges of each gene therapy approach, then focus on current clinical trials and finally outline future directions for the development of gene therapy for OTCD.
| Type: | Article |
|---|---|
| Title: | Genetic Therapy Approaches for Ornithine Transcarbamylase Deficiency |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.3390/biomedicines11082227 |
| Publisher version: | https://doi.org/10.3390/biomedicines11082227 |
| Language: | English |
| Additional information: | © 2023 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/). |
| Keywords: | ornithine transcarbamylase deficiency; gene therapy; adeno-associated virus; messenger RNA |
| 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/10175362 |
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