Developing therapeutic approaches for metachromatic leukodystrophy

Drug Design, Development and Therapy

Volumn 7, Issue , 2013, Pages 729-745

  Patil, Shilpa A ^a   Maegawa, Gustavo H B ^a  

^a JOHNS HOPKINS UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Arylsulfatase A; Enzyme enhancement therapy; Enzyme replacement therapy; Gene therapy; Metachromatic leukodystrophy; Small molecules

Indexed keywords

BENZYLOXYCARBONYLLEUCYLLEUCYLLEUCINAL; CELASTROL; CEREBROSIDE SULFATASE; CHAPERONE; HISTONE DEACETYLASE INHIBITOR; METAZYM; MIGLUSTAT; MUTANT PROTEIN; MYELIN; RECOMBINANT CEREBROSIDE SULFATASE; RECOMBINANT ENZYME; RETROVIRUS VECTOR; SPHINGOLIPID; SULFATIDE; UNCLASSIFIED DRUG; WARFARIN; ENZYME ACTIVATOR;

AUTOSOMAL RECESSIVE DISORDER; BLOOD BRAIN BARRIER; BONE MARROW; CENTRAL NERVOUS SYSTEM; CLINICAL FEATURE; DEMYELINATION; DISABILITY; DISEASE COURSE; DRUG SAFETY; DRUG TARGETING; ENZYME DEFICIENCY; ENZYME REPLACEMENT; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; LIPID DEGRADATION; LYSOSOME STORAGE DISEASE; METACHROMATIC LEUKODYSTROPHY; MICROENCAPSULATION; MOLECULAR PATHOLOGY; NERVE CELL; NEUROLOGIC DISEASE; NONHUMAN; REVIEW; UMBILICAL CORD BLOOD; ALLELE; GENETICS; LEUKODYSTROPHY, METACHROMATIC;

ALLELES; BLOOD-BRAIN BARRIER; CEREBROSIDE-SULFATASE; ENZYME REACTIVATORS; GENETIC THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; LEUKODYSTROPHY, METACHROMATIC;

EID: 84881270781     PISSN: 11778881     EISSN: None     Source Type: Journal    
DOI: 10.2147/DDDT.S15467     Document Type: Review

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