Treatment of brain disease in the mucopolysaccharidoses

Molecular Genetics and Metabolism

Volumn 122, Issue , 2017, Pages 25-34

  Scarpa, Maurizio ^a,b   Orchard, Paul J ^c   Schulz, Angela ^d   Dickson, Patricia I ^e   Haskins, Mark E ^f   Escolar, Maria L ^g   Giugliani, Roberto ^h  

^a DR HORST SCHMIDT KLINIKEN   (Germany)

^b UNIVERSITY OF PADOVA   (Italy)

^c UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^d UNIVERSITY MEDICAL CENTER HAMBURG EPPENDORF   (Germany)

^e HARBOR UCLA MEDICAL CENTER   (United States)

^f UNIVERSITY OF PENNSYLVANIA   (United States)

^g CHILDREN S HOSPITAL OF PITTSBURGH   (United States)

^h HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

Author keywords

Blood brain barrier; Enzyme replacement therapy; Gene therapy; Mucopolysaccharidoses; Transplantation

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; ATALUREN; CHAPERONE; CHLORAMPHENICOL; GENE THERAPY AGENT; GENTAMICIN; IDURONATE 2 SULFATASE; LARONIDASE; LENTIVIRUS VECTOR; LEVO IDURONIDASE; RECOMBINANT ENZYME; RECOMBINANT HEPARAN N SULFATASE; SULFATASE; UNCLASSIFIED DRUG; DRUG CARRIER; GLYCOSAMINOGLYCAN; NANOPARTICLE; RECOMBINANT PROTEIN;

BLOOD BRAIN BARRIER; BRAIN DISEASE; CENTRAL NERVOUS SYSTEM; COGNITIVE DEFECT; DRUG DELIVERY SYSTEM; ENZYME REPLACEMENT; EX VIVO GENE TRANSFER; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; IMMUNE RESPONSE; MUCOPOLYSACCHARIDOSIS; MUCOPOLYSACCHARIDOSIS TYPE 7; NEUROLOGIC DISEASE; NONHUMAN; PRIORITY JOURNAL; PROBLEM BEHAVIOR; REVIEW; SANFILIPPO SYNDROME; STOP CODON; TREATMENT OUTCOME; VIRAL GENE THERAPY; ANIMAL; BRAIN; CHEMISTRY; CHILD; CHILD BEHAVIOR; CHILD DEVELOPMENT; CLINICAL TRIAL (TOPIC); CYTOLOGY; DRUG EFFECT; GENE THERAPY; GENETICS; INTRACEREBROVENTRICULAR DRUG ADMINISTRATION; INTRASPINAL DRUG ADMINISTRATION; METABOLISM; ORGANIZATION; PATHOLOGY; PRESCHOOL CHILD; PROCEDURES; VASCULARIZATION;

ANIMALS; BLOOD-BRAIN BARRIER; BRAIN; CHILD; CHILD BEHAVIOR; CHILD DEVELOPMENT; CHILD, PRESCHOOL; CLINICAL TRIALS AS TOPIC; COGNITIVE DYSFUNCTION; CONGRESSES AS TOPIC; DRUG CARRIERS; ENZYME REPLACEMENT THERAPY; GENETIC THERAPY; GLYCOSAMINOGLYCANS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INJECTIONS, INTRAVENTRICULAR; INJECTIONS, SPINAL; MOLECULAR CHAPERONES; MUCOPOLYSACCHARIDOSES; NANOPARTICLES; RECOMBINANT PROTEINS;

EID: 85033789412     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2017.10.007     Document Type: Review

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