In vivo base editing gene therapy for heterozygous familial hypercholesterolemia: a phase 1 trial
Ping Wan, Siyuan Tang, Dongni Lin, Yuming Lu, Mei Long, Ling Xiao, Yanhong Jiang, Jiaoyang Liao, Xiaoying Ma, Ying Liu, Wensu Yu, Michael Ott, Zi Jun Wang, Yuxuan Wu, Taihua Yang, Qiang Xia
Topics & Concepts
Familial hypercholesterolemiaPCSK9MedicineIn vivoGenetic enhancementAdverse effectClinical trialInternal medicineCholesterolGenome editingPharmacologyLDL receptorEndocrinologyPhases of clinical researchApolipoprotein BGenetic disorderHeterozygote advantageCohortLipoproteinBioinformaticsLiver enzymeClinical significanceOncologyGeneVirus-based gene therapy researchLipoproteins and Cardiovascular HealthCRISPR and Genetic Engineering