LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study
Alexandria M. Doerfler, So Hyun Park, Julia M. Assini, Amer Youssef, Lavanya Saxena, Adam B. Yaseen, Marco De Giorgi, Marcel Chuecos, Ayrea Hurley, Ang Li, Santica M. Marcovina, Gang Bao, Michael B. Boffa, Marlys L. Koschinsky, William R. Lagor
Abstract
, and highlights the importance of examining the diverse consequences of CRISPR cutting within repetitive loci and in the genome globally.
Topics & Concepts
CRISPRTransgeneGenetically modified mouseProof of conceptChemistryCell biologyBiologyComputational biologyMolecular biologyBiochemistryComputer scienceGeneOperating systemCRISPR and Genetic EngineeringPluripotent Stem Cells ResearchVirus-based gene therapy research