Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier
Bethan J. Critchley, H. Bobby Gaspar, Sara Benedetti
Topics & Concepts
Central nervous systemEnzyme replacement therapyBlood–brain barrierGenetic enhancementStem cellLysosomal storage diseaseProgenitor cellBiologyNeuroscienceTransplantationIn vivoSubstrate reduction therapyMedicineBioinformaticsEnzymeCell biologyDiseasePathologyGeneInternal medicineBiotechnologyBiochemistryLysosomal Storage Disorders ResearchCellular transport and secretionRetinal Development and Disorders