Metachromatic leukodystrophy: Disease spectrum and approaches for treatment

Best Practice and Research: Clinical Endocrinology and Metabolism

Volumn 29, Issue 2, 2015, Pages 261-273

  Van Rappard, Diane F ^a   Boelens, Jaap Jan ^b   Wolf, Nicole I ^a  

^a VU UNIVERSITY MEDICAL CENTER   (Netherlands)

^b UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

Author keywords

enzyme replacement therapy; gene therapy; hematopoietic stem cell transplantation; leukodystrophy; magnetic resonance imaging; metachromatic

Indexed keywords

ARYLSULFATASE; CEREBROSIDE SULFATASE; LENTIVIRUS VECTOR; PARVOVIRUS VECTOR; RETROVIRUS VECTOR; SULFATIDE;

ARSA GENE; CENTRAL NERVOUS SYSTEM; CLINICAL TRIAL (TOPIC); DEMYELINATION; DIAGNOSTIC PROCEDURE; ENZYME ACTIVITY; ENZYME REPLACEMENT; GENE MUTATION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; METACHROMATIC LEUKODYSTROPHY; MUTATOR GENE; NONHUMAN; ONSET AGE; PERIPHERAL NERVOUS SYSTEM; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); REVIEW; TREATMENT INDICATION; VIRAL GENE THERAPY; BRAIN; BRAIN DISEASES; COMPLICATION; GENE THERAPY; LEUKODYSTROPHY, METACHROMATIC; NUCLEAR MAGNETIC RESONANCE IMAGING; PATHOLOGY; PERIPHERAL NERVOUS SYSTEM DISEASES;

BRAIN; BRAIN DISEASES; ENZYME REPLACEMENT THERAPY; GENETIC THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; LEUKODYSTROPHY, METACHROMATIC; MAGNETIC RESONANCE IMAGING; PERIPHERAL NERVOUS SYSTEM DISEASES;

EID: 84929507063     PISSN: 1521690X     EISSN: 15321908     Source Type: Journal    
DOI: 10.1016/j.beem.2014.10.001     Document Type: Review

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