Neuronopathic lysosomal storage disorders: Approaches to treat the central nervous system

Best Practice and Research: Clinical Endocrinology and Metabolism

Volumn 29, Issue 2, 2015, Pages 159-171

  Scarpa, Maurizio ^a,b,c   Bellettato, Cinzia Maria ^c   Lampe, Christina ^a   Begley, David J ^c,d  

^a DR HORST SCHMIDT KLINIKEN   (Germany)

^b UNIVERSITY OF PADOVA   (Italy)

^c Brains for Brain Foundation   (Italy)

^d KING'S COLLEGE LONDON   (United Kingdom)

Author keywords

blood brain barrier; central nervous system; drug delivery; lysosomal storage disorders; neurodegeneration

Indexed keywords

CARRIER PROTEIN; CHAPERONE; HYBRID PROTEIN; LIPOSOME; NANOCARRIER; NANOPARTICLE; PARVOVIRUS VECTOR; RECOMBINANT DNA; RECOMBINANT ENZYME; RECOMBINANT PROTEIN;

BLOOD BRAIN BARRIER; BONE MARROW TRANSPLANTATION; CELL MIGRATION; CENTRAL NERVOUS SYSTEM DISEASE; DEGENERATIVE DISEASE; DRUG ADMINISTRATION ROUTE; DRUG DELIVERY SYSTEM; ENZYME REPLACEMENT; GENE THERAPY; HUMAN; LYSOSOME STORAGE DISEASE; MONOCYTE; MONOCYTYE TRANSMIGRATION; NERVE CELL NECROSIS; NEURAL STEM CELL; NEUROPATHOLOGY; NONHUMAN; REVIEW; SUBSTRATE REDUCTION THERAPY; INTRACEREBROVENTRICULAR DRUG ADMINISTRATION; INTRASPINAL DRUG ADMINISTRATION; LYSOSOMAL STORAGE DISEASES, NERVOUS SYSTEM; METABOLISM; PROCEDURES;

BLOOD-BRAIN BARRIER; DRUG DELIVERY SYSTEMS; ENZYME REPLACEMENT THERAPY; HUMANS; INFUSIONS, INTRAVENTRICULAR; INFUSIONS, SPINAL; INJECTIONS, INTRAVENTRICULAR; INJECTIONS, SPINAL; LYSOSOMAL STORAGE DISEASES, NERVOUS SYSTEM; MOLECULAR CHAPERONES; NANOPARTICLES; RECOMBINANT PROTEINS;

EID: 84929518545     PISSN: 1521690X     EISSN: 15321908     Source Type: Journal    
DOI: 10.1016/j.beem.2014.12.001     Document Type: Review

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