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Review

Therapy Prospects for Mitochondrial DNA Maintenance Disorders

1
Research Group on Neuromuscular and Mitochondrial Diseases, Vall d’Hebron Research Institute, Universitat Autònoma de Barcelona, 08035 Barcelona, Spain
2
Biomedical Network Research Centre on Rare Diseases (CIBERER), Instituto de Salud Carlos III, 28029 Madrid, Spain
*
Author to whom correspondence should be addressed.
Academic Editor: María Eugenia Soriano
Int. J. Mol. Sci. 2021, 22(12), 6447; https://doi.org/10.3390/ijms22126447
Received: 22 May 2021 / Revised: 10 June 2021 / Accepted: 11 June 2021 / Published: 16 June 2021
(This article belongs to the Special Issue Molecular Research on Mitochondrial Dysfunction)
Mitochondrial DNA depletion and multiple deletions syndromes (MDDS) constitute a group of mitochondrial diseases defined by dysfunctional mitochondrial DNA (mtDNA) replication and maintenance. As is the case for many other mitochondrial diseases, the options for the treatment of these disorders are rather limited today. Some aggressive treatments such as liver transplantation or allogeneic stem cell transplantation are among the few available options for patients with some forms of MDDS. However, in recent years, significant advances in our knowledge of the biochemical pathomechanisms accounting for dysfunctional mtDNA replication have been achieved, which has opened new prospects for the treatment of these often fatal diseases. Current strategies under investigation to treat MDDS range from small molecule substrate enhancement approaches to more complex treatments, such as lentiviral or adenoassociated vector-mediated gene therapy. Some of these experimental therapies have already reached the clinical phase with very promising results, however, they are hampered by the fact that these are all rare disorders and so the patient recruitment potential for clinical trials is very limited. View Full-Text
Keywords: mitochondria; mtDNA; replication; depletion; multiple deletions; therapy; nucleoside; gene therapy mitochondria; mtDNA; replication; depletion; multiple deletions; therapy; nucleoside; gene therapy
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MDPI and ACS Style

Ramón, J.; Vila-Julià, F.; Molina-Granada, D.; Molina-Berenguer, M.; Melià, M.J.; García-Arumí, E.; Torres-Torronteras, J.; Cámara, Y.; Martí, R. Therapy Prospects for Mitochondrial DNA Maintenance Disorders. Int. J. Mol. Sci. 2021, 22, 6447. https://doi.org/10.3390/ijms22126447

AMA Style

Ramón J, Vila-Julià F, Molina-Granada D, Molina-Berenguer M, Melià MJ, García-Arumí E, Torres-Torronteras J, Cámara Y, Martí R. Therapy Prospects for Mitochondrial DNA Maintenance Disorders. International Journal of Molecular Sciences. 2021; 22(12):6447. https://doi.org/10.3390/ijms22126447

Chicago/Turabian Style

Ramón, Javier, Ferran Vila-Julià, David Molina-Granada, Miguel Molina-Berenguer, Maria J. Melià, Elena García-Arumí, Javier Torres-Torronteras, Yolanda Cámara, and Ramon Martí. 2021. "Therapy Prospects for Mitochondrial DNA Maintenance Disorders" International Journal of Molecular Sciences 22, no. 12: 6447. https://doi.org/10.3390/ijms22126447

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