A magic bullet to specifically eliminate mutated mitochondrial genomes from patients' cells.

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).

• Authors: Carlos T Moraes
• Journal: EMBO molecular medicine (EMBO Mol Med) Vol. 6 Issue 4 Pg. 434-5 (04 2014) ISSN: 1757-4684 [Electronic] England
• PMID: 24623377 (Publication Type: Journal Article, Comment)

Chemical References

• DNA, Mitochondrial

Topics

• DNA, Mitochondrial (metabolism)
• Genetic Therapy
• Genome, Mitochondrial
• Humans
• Mitochondria (enzymology)
• Mitochondrial Diseases (genetics, therapy)
• Point Mutation
• Sequence Deletion