A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I
Li Ou, Michael J. Przybilla, Ozan Ahlat, Sarah Kim-McOlash, Paula Overn, Jeanine R. Jarnes, Maeve O’Sullivan, Chester B. Whitley
Topics & Concepts
Mucopolysaccharidosis type IGenome editingGenetic enhancementBiologyGeneTransgeneCRISPRViral vectorGene deliveryMucopolysaccharidosisCancer researchMedicineGeneticsInternal medicineBiochemistryRecombinant DNAEnzyme replacement therapyDiseaseCRISPR and Genetic EngineeringLysosomal Storage Disorders ResearchAutism Spectrum Disorder Research