miR-143 Regulates Lysosomal Enzyme Transport across the Blood-Brain Barrier and Transforms CNS Treatment for Mucopolysaccharidosis Type I
Yi Lin, Xiaohong Wang, Kevin Rose, Mei Dai, Jingfen Han, Mei Xin, Dao Pan
Topics & Concepts
Blood–brain barrierLysosomeEnzyme replacement therapyLysosomal storage diseaseEnzymeMucopolysaccharidosisMucopolysaccharidosis ICell biologyBiologyNeuroscienceCentral nervous systemMedicineBiochemistryInternal medicineDiseaseLysosomal Storage Disorders ResearchInfant Nutrition and HealthNeonatal and fetal brain pathology