Gene delivery improvement for treating the lysosomal storage disorder metachromatic leukodystrophy

Human Gene Therapy

Volumn 23, Issue 8, 2012, Pages 793-795

  Ferraiuolo, Laura ^a   Kaspar, Brian K ^a,b  

^a THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN S HOSPITAL   (United States)

^b THE OHIO STATE UNIVERSITY COLLEGE OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR 2; ADENO ASSOCIATED VIRUS VECTOR 8; ADENO ASSOCIATED VIRUS VECTOR 9; ADENO ASSOCIATED VIRUS VECTOR RH 10; BETA ACTIN; BETA GLOBIN; CEREBROSIDE SULFATASE; PHOSPHOGLYCERATE KINASE; PROTEIN; PROTEIN RPE65; SULFATIDE; TRANSMEMBRANE CONDUCTANCE REGULATOR; UNCLASSIFIED DRUG; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; CENTRAL NERVOUS SYSTEM; CENTRAL NERVOUS SYSTEM DISEASE; CEREBROSPINAL FLUID; CLINICAL TRIAL (TOPIC); CYSTIC FIBROSIS; CYTOMEGALOVIRUS; DISEASE COURSE; ENZYME LOCALIZATION; GENE MUTATION; GENE THERAPY; GENE TRANSFER; GLIA CELL; HEMOPHILIA; HUMAN; INJECTION SITE; LEBER CONGENITAL AMAUROSIS; LIPOSOMAL GENE DELIVERY SYSTEM; LYSOSOME; METACHROMATIC LEUKODYSTROPHY; MUSCLE HYPOTONIA; MUSCLE WEAKNESS; NONHUMAN; NOTE; OLIGODENDROGLIA; OPTIC NERVE ATROPHY; PIGMENT EPITHELIUM; RESPIRATORY TRACT INFECTION; SEIZURE;

ANIMALS; BRAIN; DEPENDOVIRUS; HUMANS; LEUKODYSTROPHY, METACHROMATIC; OLIGODENDROGLIA;

EID: 84865221233     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2012.2516     Document Type: Note

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