Cas9-mediated replacement of expanded CAG repeats in a pig model of Huntington’s disease
Sen Yan, Xiaoming Zheng, Yingqi Lin, Caijuan Li, Zhaoming Liu, Jiawei Li, Zhuchi Tu, Yu Zhao, Chunhui Huang, Yizhi Chen, Jun Li, Xichen Song, Bofeng Han, Wei Wang, Weien Liang, Liangxue Lai, Xiao‐Jiang Li, Shihua Li
Topics & Concepts
NeurodegenerationMutantBiologyGeneTrinucleotide repeat expansionDiseaseGenetic enhancementTransgeneHuntingtinCas9AlleleMutationHuntington's diseaseGeneticsMolecular biologyMedicineCRISPRPathologyGenetic Neurodegenerative DiseasesCRISPR and Genetic EngineeringVirus-based gene therapy research