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Specific elimination of mutant mitochondrial genomes in patient–derived cells by mitoTALENs
Mitochondrial diseases are commonly caused by mutations in the mitochondrial DNA (mtDNA), which in most cases co–exists with the wild–type (mtDNA heteroplasmy). We have engineered TAL–effector nucleases (TALENs) to localize to mitochondria and cleave different classes of pathogenic mtDNA mutations....
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
2013
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4153471/ https://www.ncbi.nlm.nih.gov/pubmed/23913125 http://dx.doi.org/10.1038/nm.3261 |
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author | Bacman, Sandra R. Williams, Siôn L. Pinto, Milena Peralta, Susana Moraes, Carlos T. |
author_facet | Bacman, Sandra R. Williams, Siôn L. Pinto, Milena Peralta, Susana Moraes, Carlos T. |
author_sort | Bacman, Sandra R. |
collection | PubMed |
description | Mitochondrial diseases are commonly caused by mutations in the mitochondrial DNA (mtDNA), which in most cases co–exists with the wild–type (mtDNA heteroplasmy). We have engineered TAL–effector nucleases (TALENs) to localize to mitochondria and cleave different classes of pathogenic mtDNA mutations. MitoTALEN expression led to permanent reductions in deletion or point mutant mtDNA in patient–derived cells, raising the possibility that they can be curative to some of these diseases. |
format | Online Article Text |
id | pubmed-4153471 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2013 |
record_format | MEDLINE/PubMed |
spelling | pubmed-41534712014-09-03 Specific elimination of mutant mitochondrial genomes in patient–derived cells by mitoTALENs Bacman, Sandra R. Williams, Siôn L. Pinto, Milena Peralta, Susana Moraes, Carlos T. Nat Med Article Mitochondrial diseases are commonly caused by mutations in the mitochondrial DNA (mtDNA), which in most cases co–exists with the wild–type (mtDNA heteroplasmy). We have engineered TAL–effector nucleases (TALENs) to localize to mitochondria and cleave different classes of pathogenic mtDNA mutations. MitoTALEN expression led to permanent reductions in deletion or point mutant mtDNA in patient–derived cells, raising the possibility that they can be curative to some of these diseases. 2013-08-04 2013-09 /pmc/articles/PMC4153471/ /pubmed/23913125 http://dx.doi.org/10.1038/nm.3261 Text en Users may view, print, copy, download and text and data- mine the content in such documents, for the purposes of academic research, subject always to the full Conditions of use: http://www.nature.com/authors/editorial_policies/license.html#terms |
spellingShingle | Article Bacman, Sandra R. Williams, Siôn L. Pinto, Milena Peralta, Susana Moraes, Carlos T. Specific elimination of mutant mitochondrial genomes in patient–derived cells by mitoTALENs |
title | Specific elimination of mutant mitochondrial genomes in patient–derived cells by mitoTALENs |
title_full | Specific elimination of mutant mitochondrial genomes in patient–derived cells by mitoTALENs |
title_fullStr | Specific elimination of mutant mitochondrial genomes in patient–derived cells by mitoTALENs |
title_full_unstemmed | Specific elimination of mutant mitochondrial genomes in patient–derived cells by mitoTALENs |
title_short | Specific elimination of mutant mitochondrial genomes in patient–derived cells by mitoTALENs |
title_sort | specific elimination of mutant mitochondrial genomes in patient–derived cells by mitotalens |
topic | Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4153471/ https://www.ncbi.nlm.nih.gov/pubmed/23913125 http://dx.doi.org/10.1038/nm.3261 |
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