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Therapy Prospects for Mitochondrial DNA Maintenance Disorders
- Source :
- International Journal of Molecular Sciences, International Journal of Molecular Sciences, Vol 22, Iss 6447, p 6447 (2021), Dipòsit Digital de Documents de la UAB, Universitat Autònoma de Barcelona, Scientia
- Publication Year :
- 2021
- Publisher :
- MDPI, 2021.
-
Abstract
- Esgotament; Teràpia gènica; Mitocondris Mitochondria; Depletion; Gene therapy Agotamiento; Terapia génica; Mitocondrias 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.
- Subjects :
- 0301 basic medicine
Mitochondrial Diseases
Genetic enhancement
medicine.medical_treatment
Genetic Phenomena::DNA Replication [PHENOMENA AND PROCESSES]
Multiple deletions
Review
Nutritional and Metabolic Diseases::Metabolic Diseases::Mitochondrial Diseases [DISEASES]
Mitochondrion
Liver transplantation
Bioinformatics
0302 clinical medicine
Nucleoside
Biology (General)
Otros calificadores::/terapia [Otros calificadores]
Spectroscopy
depletion
mtDNA
Disease Management
General Medicine
multiple deletions
Combined Modality Therapy
gene therapy
Computer Science Applications
Mitochondria
mitochondria
Chemistry
Mitocondris - Malalties - Tractament
Disease Susceptibility
Stem cell
enfermedades nutricionales y metabólicas::enfermedades metabólicas::enfermedades mitocondriales [ENFERMEDADES]
DNA Replication
Mitochondrial DNA
replication
ADN - Duplicació
QH301-705.5
Replication
Dysfunctional family
nucleoside
DNA, Mitochondrial
Catalysis
Mitochondrial Proteins
Inorganic Chemistry
03 medical and health sciences
Depletion
fenómenos genéticos::replicación del ADN [FENÓMENOS Y PROCESOS]
Gene therapy
medicine
Animals
Humans
Physical and Theoretical Chemistry
QD1-999
Molecular Biology
therapy
business.industry
Organic Chemistry
Other subheadings::/therapy [Other subheadings]
Mtdn
Transplantation
Clinical trial
030104 developmental biology
Gene Expression Regulation
Mutation
Therapy
business
030217 neurology & neurosurgery
Subjects
Details
- Language :
- English
- ISSN :
- 14220067
- Volume :
- 22
- Issue :
- 12
- Database :
- OpenAIRE
- Journal :
- International Journal of Molecular Sciences
- Accession number :
- edsair.doi.dedup.....5a05d416ac82094cae00451c40ad52ab