Gene therapy strategies for rare monogenic disorders with nuclear or mitochondrial gene mutations
Yi Wang, Li‐Fan Hu, Tian‐Jiao Zhou, Lian‐Yu Qi, Lei Xing, Jaiwoo Lee, Fengzhen Wang, Yu‐Kyoung Oh, Hu‐Lin Jiang
Topics & Concepts
Genetic enhancementGene deliveryGenome editingGeneBioinformaticsBiologyMitochondrial diseaseGene mutationMedicineComputational biologyGeneticsMitochondrial DNAMutationGenomeCRISPR and Genetic EngineeringRNA regulation and diseaseMitochondrial Function and Pathology