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Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities
Mitochondria are critical organelles that form networks within our cells, generate energy dynamically, contribute to diverse cell and organ function, and produce a variety of critical signaling molecules, such as cortisol. This intracellular microbiome can differ between cells, tissues, and organs....
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10056815/ https://www.ncbi.nlm.nih.gov/pubmed/36982871 http://dx.doi.org/10.3390/ijms24065798 |
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author | Kar, Bibekananda Castillo, Santiago R. Sabharwal, Ankit Clark, Karl J. Ekker, Stephen C. |
author_facet | Kar, Bibekananda Castillo, Santiago R. Sabharwal, Ankit Clark, Karl J. Ekker, Stephen C. |
author_sort | Kar, Bibekananda |
collection | PubMed |
description | Mitochondria are critical organelles that form networks within our cells, generate energy dynamically, contribute to diverse cell and organ function, and produce a variety of critical signaling molecules, such as cortisol. This intracellular microbiome can differ between cells, tissues, and organs. Mitochondria can change with disease, age, and in response to the environment. Single nucleotide variants in the circular genomes of human mitochondrial DNA are associated with many different life-threatening diseases. Mitochondrial DNA base editing tools have established novel disease models and represent a new possibility toward personalized gene therapies for the treatment of mtDNA-based disorders. |
format | Online Article Text |
id | pubmed-10056815 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-100568152023-03-30 Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities Kar, Bibekananda Castillo, Santiago R. Sabharwal, Ankit Clark, Karl J. Ekker, Stephen C. Int J Mol Sci Review Mitochondria are critical organelles that form networks within our cells, generate energy dynamically, contribute to diverse cell and organ function, and produce a variety of critical signaling molecules, such as cortisol. This intracellular microbiome can differ between cells, tissues, and organs. Mitochondria can change with disease, age, and in response to the environment. Single nucleotide variants in the circular genomes of human mitochondrial DNA are associated with many different life-threatening diseases. Mitochondrial DNA base editing tools have established novel disease models and represent a new possibility toward personalized gene therapies for the treatment of mtDNA-based disorders. MDPI 2023-03-18 /pmc/articles/PMC10056815/ /pubmed/36982871 http://dx.doi.org/10.3390/ijms24065798 Text en © 2023 by the authors. https://creativecommons.org/licenses/by/4.0/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/). |
spellingShingle | Review Kar, Bibekananda Castillo, Santiago R. Sabharwal, Ankit Clark, Karl J. Ekker, Stephen C. Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities |
title | Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities |
title_full | Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities |
title_fullStr | Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities |
title_full_unstemmed | Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities |
title_short | Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities |
title_sort | mitochondrial base editing: recent advances towards therapeutic opportunities |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10056815/ https://www.ncbi.nlm.nih.gov/pubmed/36982871 http://dx.doi.org/10.3390/ijms24065798 |
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