Ontology highlight
ABSTRACT:
SUBMITTER: Pereira CV
PROVIDER: S-EPMC6127889 | biostudies-other | 2018 Sep
REPOSITORIES: biostudies-other
Pereira Claudia V CV Bacman Sandra R SR Arguello Tania T Zekonyte Ugne U Williams Sion L SL Edgell David R DR Moraes Carlos T CT
EMBO molecular medicine 20180901 9
Pathogenic mitochondrial DNA (mtDNA) mutations often co-exist with wild-type molecules (mtDNA heteroplasmy). Phenotypes manifest when the percentage of mutant mtDNA is high (70-90%). Previously, our laboratory showed that mitochondria-targeted transcription activator-like effector nucleases (mitoTALENs) can eliminate mutant mtDNA from heteroplasmic cells. However, mitoTALENs are dimeric and relatively large, making it difficult to package their coding genes into viral vectors, limiting their cli ...[more]