ArticleEMBO molecular medicine2014
A magic bullet to specifically eliminate mutated mitochondrial genomes from patients' cells.
Article in EMBO molecular medicine, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
13 citing papers in PubMed.
- Mitochondrial-based therapies for neurodegenerative diseases: a review of the current literature.Naunyn-Schmiedeberg's archives of pharmacology · 2025Review
- A New Perspective on the Role of Alterations in Mitochondrial Proteins Involved in ATP Synthesis and Mobilization in Cardiomyopathies.International journal of molecular sciences · 2025Review
- Mitochondrial diseases: from molecular mechanisms to therapeutic advances.Signal transduction and targeted therapy · 2025Review
- Trends and prospects in mitochondrial genome editing.Experimental & molecular medicine · 2023Review
- Current Progress of Mitochondrial Genome Editing by CRISPR.Frontiers in physiology · 2022Article
- "Mitochondrial Toolbox" - A Review of Online Resources to Explore Mitochondrial Genomics.Frontiers in genetics · 2020Review
- TALEN-mediated shift of mitochondrial DNA heteroplasmy in MELAS-iPSCs with m.13513G>A mutation.Scientific reports · 2017Article
- Genetic manipulation for inherited neurodegenerative diseases: myth or reality?The British journal of ophthalmology · 2016Review
- Near-complete elimination of mutant mtDNA by iterative or dynamic dose-controlled treatment with mtZFNs.Nucleic acids research · 2016Article
- Mitochondria as a therapeutic target in Alzheimer's disease.Genes & diseases · 2016Review
- Modifying the Mitochondrial Genome.Cell metabolism · 2016Review
- Evidence to support mitochondrial neuroprotection, in severe traumatic brain injury.Journal of bioenergetics and biomembranes · 2015Review
- Maternally inherited mitochondrial respiratory disorders: from pathogenetic principles to therapeutic implications.Molecular genetics and metabolismReview
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Authors and funding
1 author.
Funding
No grant is acknowledged in the PubMed record.
Abstract
When mitochondrial diseases result from mutations found in the mitochondrial DNA, engineered mitochondrial-targeted nucleases such as mitochondrial-targeted zinc finger nucleases are shown to specifically eliminate the mutated molecules, leaving the wild-type mitochondrial DNA intact to replicate and restore normal copy number. In this issue, Gammage and colleagues successfully apply this improved technology on patients' cells with two types of genetic alterations responsible for neuropathy ataxia and retinitis pigmentosa (NARP) syndrome and Kearns Sayre syndrome and progressive external ophthalmoplegia (PEO).
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.