Single AAV-mediated CRISPR-Nme2Cas9 efficiently reduces mutant hTTR expression in a transgenic mouse model of transthyretin amyloidosis
Jinkun Wen, Tianqi Cao, Jinni Wu, Yuxi Chen, Shengyao Zhi, Yanming Huang, Peilin Zhen, Guanglan Wu, Lars Aagaard, Jianxin Zhong, Puping Liang, Junjiu Huang
Topics & Concepts
MutantTransgeneTransthyretinGenetically modified mouseCRISPRCell biologyBiologyGene knockinMolecular biologyGeneGeneticsEndocrinologyCRISPR and Genetic EngineeringRNA regulation and diseaseCellular transport and secretion