Cargando…

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....

Descripción completa

Detalles Bibliográficos
Autores principales: Bacman, Sandra R., Williams, Siôn L., Pinto, Milena, Peralta, Susana, Moraes, Carlos T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2013
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
_version_ 1782333280986595328
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
work_keys_str_mv AT bacmansandrar specificeliminationofmutantmitochondrialgenomesinpatientderivedcellsbymitotalens
AT williamssionl specificeliminationofmutantmitochondrialgenomesinpatientderivedcellsbymitotalens
AT pintomilena specificeliminationofmutantmitochondrialgenomesinpatientderivedcellsbymitotalens
AT peraltasusana specificeliminationofmutantmitochondrialgenomesinpatientderivedcellsbymitotalens
AT moraescarlost specificeliminationofmutantmitochondrialgenomesinpatientderivedcellsbymitotalens