Article
Characterization of chemically modified oligonucleotides targeting a pathogenic mutation in human mitochondrial DNA.
Biochimie - 1 May 2014
Tonin Yann, Heckel Anne-Marie, Dovydenko Ilya, Meschaninova Mariya, Comte Caroline, Venyaminova Alya, Pyshnyi Dmitrii, Tarassov Ivan, Entelis Nina
Abstract excerpt
Defects in mitochondrial genome can cause a wide range of clinical disorders, mainly neuromuscular diseases. Most of the deleterious mitochondrial mutations are heteroplasmic, meaning that wild type and mutated forms of mtDNA coexist in the same cell. Therefore, a shift in the proportion between mutant and wild type molecules could restore mitochondrial functions. The anti-replicative strategy aims to induce such...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.