CRISPR/Cas9‐mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse - Guan, Yuting; Ma, Yanlin; Li, Qi; Sun, Zhenliang; Ma, Lie; Wu, Lijuan; Wang, Liren; Zeng, Li; Shao, Yanjiao; Chen, Yuting; Ma, Ning; Lu, Wenqing; et al. (EMBO molecular medicine, 2016)
Keywords: Adenoviridae - genetics; Animal models; Animals; Child; Coagulation factors; CRISPR; CRISPR-Cas Systems; Deoxyribonucleic acid; Disease; Disease Models, Animal; DNA; EMBO16; EMBO18; Expression vectors; Factor IX - genetics; Factor IX deficiency; Gene Editing - methods; Gene loci; Gene therapy; Genetic disorders; Genetic Therapy - methods; Genetic Vectors; Genome editing; Genomes; Hemophilia; hemophilia B; Hemophilia B - pathology; Hemophilia B - therapy; Hemostasis; Hepatocytes; Hereditary diseases; Humans; Male; Males; Mice; monogenetic disease; Mutation; Mutation, Missense; Phenotypes; Point mutation; Recombination, Genetic; Research Article; Toxicity; Treatment Outcome; Twins; Mali; China; genome editing; gene therapy; monogenetic disease; hemostasis; hemophilia B