MitoTALEN reduces mutant mtDNA load and restores tRNAAla levels in a mouse model of heteroplasmic mtDNA mutation
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Title
MitoTALEN reduces mutant mtDNA load and restores tRNAAla levels in a mouse model of heteroplasmic mtDNA mutation
Authors
Keywords
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Journal
NATURE MEDICINE
Volume -, Issue -, Pages -
Publisher
Springer Nature America, Inc
Online
2018-09-19
DOI
10.1038/s41591-018-0166-8
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Note: Only part of the references are listed.- Linear mitochondrial DNA is rapidly degraded by components of the replication machinery
- (2018) Viktoriya Peeva et al. Nature Communications
- The mitochondrial DNA polymerase gamma degrades linear DNA fragments precluding the formation of deletions
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- Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo
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- Near-complete elimination of mutant mtDNA by iterative or dynamic dose-controlled treatment with mtZFNs
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- A Phenotype-Driven Approach to Generate Mouse Models with Pathogenic mtDNA Mutations Causing Mitochondrial Disease
- (2016) Johanna H.K. Kauppila et al. Cell Reports
- Pathogenic mitochondrial mt-tRNAAla variants are uniquely associated with isolated myopathy
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- MitoTALEN: A General Approach to Reduce Mutant mtDNA Loads and Restore Oxidative Phosphorylation Function in Mitochondrial Diseases
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- Specific elimination of mutant mitochondrial genomes in patient-derived cells by mitoTALENs
- (2013) Sandra R Bacman et al. NATURE MEDICINE
- Directed evolution of the TALE N-terminal domain for recognition of all 5′ bases
- (2013) Brian M. Lamb et al. NUCLEIC ACIDS RESEARCH
- MRPS27 is a pentatricopeptide repeat domain protein required for the translation of mitochondrially encoded proteins
- (2012) Stefan M.K. Davies et al. FEBS LETTERS
- Mitochondrial DNA copy number is regulated in a tissue specific manner by DNA methylation of the nuclear-encoded DNA polymerase gamma A
- (2012) Richard D. W. Kelly et al. NUCLEIC ACIDS RESEARCH
- Retro-orbital injections in mice
- (2011) Tal Yardeni et al. LAB ANIMAL
- Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting
- (2011) Tomas Cermak et al. NUCLEIC ACIDS RESEARCH
- Organ-specific shifts in mtDNA heteroplasmy following systemic delivery of a mitochondria-targeted restriction endonuclease
- (2010) S R Bacman et al. GENE THERAPY
- Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures
- (2010) Yannick Doyon et al. NATURE METHODS
- TAL nucleases (TALNs): hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain
- (2010) Ting Li et al. NUCLEIC ACIDS RESEARCH
- Intra- and inter-molecular recombination of mitochondrial DNA after in vivo induction of multiple double-strand breaks
- (2009) S. R. Bacman et al. NUCLEIC ACIDS RESEARCH
- Mechanisms of formation and accumulation of mitochondrial DNA deletions in aging neurons
- (2008) Hirokazu Fukui et al. HUMAN MOLECULAR GENETICS
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