mitoTALEN reduces the mutant mtDNA load in neurons
Sandra R. Bacman, Jose Domingo Barrera-Paez, Milena Pinto, Derek Van Booven, James B. Stewart, Anthony J. Griswold, Carlos T. Moraes
Abstract
, paving the way for clinical trials in patients with mitochondrial encephalopathies.
Topics & Concepts
MutantMitochondrial DNABiologyGeneticsGeneMitochondrial Function and PathologyCRISPR and Genetic EngineeringRNA and protein synthesis mechanisms