Publication | Open Access

Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice

Knockout MouseGene TherapiesBone Disease FeaturesPathogenesisImmunologyPathologyGenetic EngineeringNeonatal Nonviral GeneGenome EngineeringGenome EditingGene EditingCrispr/cas9 SystemMedicineCell BiologyCrispr