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Mitochondrial DNA Replacement Techniques to Prevent Human Mitochondrial Diseases

Background: Mitochondrial DNA (mtDNA) diseases are a group of maternally inherited genetic disorders caused by a lack of energy production. Currently, mtDNA diseases have a poor prognosis and no known cure. The chance to have unaffected offspring with a genetic link is important for the affected fam...

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Autores principales: Sendra, Luis, García-Mares, Alfredo, Herrero, María José, Aliño, Salvador F.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7827455/
https://www.ncbi.nlm.nih.gov/pubmed/33430493
http://dx.doi.org/10.3390/ijms22020551
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author Sendra, Luis
García-Mares, Alfredo
Herrero, María José
Aliño, Salvador F.
author_facet Sendra, Luis
García-Mares, Alfredo
Herrero, María José
Aliño, Salvador F.
author_sort Sendra, Luis
collection PubMed
description Background: Mitochondrial DNA (mtDNA) diseases are a group of maternally inherited genetic disorders caused by a lack of energy production. Currently, mtDNA diseases have a poor prognosis and no known cure. The chance to have unaffected offspring with a genetic link is important for the affected families, and mitochondrial replacement techniques (MRTs) allow them to do so. MRTs consist of transferring the nuclear DNA from an oocyte with pathogenic mtDNA to an enucleated donor oocyte without pathogenic mtDNA. This paper aims to determine the efficacy, associated risks, and main ethical and legal issues related to MRTs. Methods: A bibliographic review was performed on the MEDLINE and Web of Science databases, along with searches for related clinical trials and news. Results: A total of 48 publications were included for review. Five MRT procedures were identified and their efficacy was compared. Three main risks associated with MRTs were discussed, and the ethical views and legal position of MRTs were reviewed. Conclusions: MRTs are an effective approach to minimizing the risk of transmitting mtDNA diseases, but they do not remove it entirely. Global legal regulation of MRTs is required.
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spelling pubmed-78274552021-01-25 Mitochondrial DNA Replacement Techniques to Prevent Human Mitochondrial Diseases Sendra, Luis García-Mares, Alfredo Herrero, María José Aliño, Salvador F. Int J Mol Sci Review Background: Mitochondrial DNA (mtDNA) diseases are a group of maternally inherited genetic disorders caused by a lack of energy production. Currently, mtDNA diseases have a poor prognosis and no known cure. The chance to have unaffected offspring with a genetic link is important for the affected families, and mitochondrial replacement techniques (MRTs) allow them to do so. MRTs consist of transferring the nuclear DNA from an oocyte with pathogenic mtDNA to an enucleated donor oocyte without pathogenic mtDNA. This paper aims to determine the efficacy, associated risks, and main ethical and legal issues related to MRTs. Methods: A bibliographic review was performed on the MEDLINE and Web of Science databases, along with searches for related clinical trials and news. Results: A total of 48 publications were included for review. Five MRT procedures were identified and their efficacy was compared. Three main risks associated with MRTs were discussed, and the ethical views and legal position of MRTs were reviewed. Conclusions: MRTs are an effective approach to minimizing the risk of transmitting mtDNA diseases, but they do not remove it entirely. Global legal regulation of MRTs is required. MDPI 2021-01-07 /pmc/articles/PMC7827455/ /pubmed/33430493 http://dx.doi.org/10.3390/ijms22020551 Text en © 2021 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 (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Sendra, Luis
García-Mares, Alfredo
Herrero, María José
Aliño, Salvador F.
Mitochondrial DNA Replacement Techniques to Prevent Human Mitochondrial Diseases
title Mitochondrial DNA Replacement Techniques to Prevent Human Mitochondrial Diseases
title_full Mitochondrial DNA Replacement Techniques to Prevent Human Mitochondrial Diseases
title_fullStr Mitochondrial DNA Replacement Techniques to Prevent Human Mitochondrial Diseases
title_full_unstemmed Mitochondrial DNA Replacement Techniques to Prevent Human Mitochondrial Diseases
title_short Mitochondrial DNA Replacement Techniques to Prevent Human Mitochondrial Diseases
title_sort mitochondrial dna replacement techniques to prevent human mitochondrial diseases
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7827455/
https://www.ncbi.nlm.nih.gov/pubmed/33430493
http://dx.doi.org/10.3390/ijms22020551
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