Lentiviral-mediated gene therapy results in sustained expression of β-Glucuronidase for up to 12 Months in the Gusmps/mpsand up to 18 Months in the Gustm(L175F)Slymouse models of mucopolysaccharidosis type VII

Human Gene Therapy

Volumn 25, Issue 9, 2014, Pages 798-810

  Derrick Roberts, Ainslie L K ^a,b   Pyragius, Carmen E ^a   Kaidonis, Xenia M ^a,c   Jackson, Matilda R ^a,c   Anson, Donald S ^a,b   Byers, Sharon ^a,b,c  

^a Genetics and Molecular Pathology   (Australia)

^b UNIVERSITY OF ADELAIDE   (Australia)

^c UNIVERSITY OF ADELAIDE   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GLUCURONIDASE; GANGLIOSIDE; GLYCOSAMINOGLYCAN; LENTIVIRUS VECTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BEHAVIOR; BIOCHEMISTRY; BONE MASS; BONE MINERAL; CONTROLLED STUDY; ENZYME ACTIVITY; ENZYMOLOGY; GENE THERAPY; HEART; KIDNEY; LIFESPAN; LIVER; LIVER NODULE; MOUSE; MUCOPOLYSACCHARIDOSIS TYPE 7; NONHUMAN; PHENOTYPE; PROTEIN EXPRESSION; SPATIAL LEARNING; SPLEEN; TRANSGENE; ANALYSIS OF VARIANCE; ANIMAL; BLOOD; BONE DENSITY; DISEASE MODEL; EXPLORATORY BEHAVIOR; GENE VECTOR; GENETICS; HEART MUSCLE; HISTOLOGY; LENTIVIRINAE; LONGEVITY; METABOLISM; MUCOPOLYSACCHARIDOSIS VII; PATHOLOGY; PHYSIOLOGY; PROCEDURES; SPECIES DIFFERENCE;

ANALYSIS OF VARIANCE; ANIMALS; BONE DENSITY; DISEASE MODELS, ANIMAL; EXPLORATORY BEHAVIOR; GENETIC THERAPY; GENETIC VECTORS; GLUCURONIDASE; HISTOLOGICAL TECHNIQUES; KIDNEY; LENTIVIRUS; LIVER; LONGEVITY; MICE; MUCOPOLYSACCHARIDOSIS VII; MYOCARDIUM; SPATIAL LEARNING; SPECIES SPECIFICITY; SPLEEN;

EID: 84907297514     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2013.141     Document Type: Article

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