Targeting the Apoa1 locus for liver-directed gene therapy
Marco De Giorgi, Ang Li, Ayrea Hurley, Mercedes Barzi, Alexandria M. Doerfler, Nikitha Cherayil, Harrison E. Smith, Jonathan D. Brown, Charles Y. Lin, Karl‐Dimiter Bissig, Gang Bao, William R. Lagor
Abstract
as a novel integration site that supports durable transgene expression in the liver for gene therapy applications.
Topics & Concepts
Locus (genetics)Genetic enhancementGeneGeneticsBiologyMedicineComputational biologyCRISPR and Genetic EngineeringVirus-based gene therapy researchPluripotent Stem Cells Research