Enhanced genome editing to ameliorate a genetic metabolic liver disease through co-delivery of adeno-associated virus receptor
Shuming Yin, Lie Ma, Tingting Shao, Mei Zhang, Yuting Guan, Liren Wang, Yaqiang Hu, Xi Chen, Honghui Han, Nan Shen, Wenjuan Qiu, Hongquan Geng, Yongguo Yu, Shichang Li, Weishi Yu, Mingyao Liu, Dali Li
Topics & Concepts
Transduction (biophysics)Adeno-associated virusGenome editingIn vivoGenetic enhancementBiologyGene deliveryGeneGenomeIndelInsertionGeneticsMutationMolecular biologyBiochemistryVector (molecular biology)Single-nucleotide polymorphismRecombinant DNAGenotypeCRISPR and Genetic EngineeringVirus-based gene therapy researchRNA regulation and disease