Efficient CRISPR/Cas9 nickase-mediated genome editing in an in vitro model of mucopolysaccharidosis IVA
Andrés Felipe Leal, Carlos Javier Alméciga-Díaz
Topics & Concepts
CRISPRGenome editingBiologyCas9Keratan sulfateEnzyme replacement therapyGenetic enhancementLysosomal storage diseaseMucopolysaccharidosis IMucopolysaccharidosisGeneGeneticsGlycosaminoglycanEnzymeChondroitin sulfateBiochemistryMedicineDiseasePathologyCRISPR and Genetic EngineeringLysosomal Storage Disorders ResearchRetinal Development and Disorders