Di Donfrancesco, Alessia;
Massaro, Giulia;
Di Meo, Ivano;
Tiranti, Valeria;
Bottani, Emanuela;
Brunetti, Dario;
(2022)
Gene Therapy for Mitochondrial Diseases: Current Status and Future Perspective.
Pharmaceutics
, 14
(6)
, Article 1287. 10.3390/pharmaceutics14061287.
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Abstract
Mitochondrial diseases (MDs) are a group of severe genetic disorders caused by mutations in the nuclear or mitochondrial genome encoding proteins involved in the oxidative phosphorylation (OXPHOS) system. MDs have a wide range of symptoms, ranging from organ-specific to multisystemic dysfunctions, with different clinical outcomes. The lack of natural history information, the limits of currently available preclinical models, and the wide range of phenotypic presentations seen in MD patients have all hampered the development of effective therapies. The growing number of pre-clinical and clinical trials over the last decade has shown that gene therapy is a viable precision medicine option for treating MD. However, several obstacles must be overcome, including vector design, targeted tissue tropism and efficient delivery, transgene expression, and immunotoxicity. This manuscript offers a comprehensive overview of the state of the art of gene therapy in MD, addressing the main challenges, the most feasible solutions, and the future perspectives of the field.
| Type: | Article |
|---|---|
| Title: | Gene Therapy for Mitochondrial Diseases: Current Status and Future Perspective |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.3390/pharmaceutics14061287 |
| Publisher version: | https://doi.org/10.3390/pharmaceutics14061287 |
| Language: | English |
| Additional information: | Copyright: © 2022 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: | mitochondria; mitochondrial DNA; mitochondrial disease; gene therapy; precision medicine |
| UCL classification: | UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > UCL School of Pharmacy > Pharmacology UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > UCL School of Pharmacy |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10151038 |
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