Genome editing-mediated knock-in of therapeutic genes ameliorates the disease phenotype in a model of hemophilia
Jeong Hyeon Lee, Hye-Kyung Oh, Beom Seok Choi, Ho Hyeon Lee, Kyu Jun Lee, Un Gi Kim, Jina Lee, Hye-Rim Lee, Geon Seong Lee, Se Jun Ahn, Jeong Pil Han, Seokjoong Kim, Su Cheong Yeom, Dong Woo Song
Abstract
-editing platform, providing insights into the development of further long-term therapeutics for diverse monogenic liver diseases.
Topics & Concepts
PhenotypeGenome editingGeneGeneticsDiseaseBiologyGenomeClinical phenotypeComputational biologyMedicinePathologyCRISPR and Genetic EngineeringCAR-T cell therapy researchVirus-based gene therapy research