Gene therapy for primary mitochondrial diseases: experimental advances and clinical challenges
Micol Falabella, Michal Minczuk, Michael G. Hanna, Carlo Viscomi, Robert D. S. Pitceathly
Topics & Concepts
Mitochondrial DNAGenetic enhancementGenome editingClinical trialMitochondrial diseaseDiseaseBiologyMedicineBioinformaticsComputational biologyGenomeGeneGeneticsPathologyMitochondrial Function and PathologyCRISPR and Genetic EngineeringMetabolism and Genetic Disorders