Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type i

Human Gene Therapy

Volumn 28, Issue 7, 2017, Pages 576-587

  Belur, Lalitha R ^a   Temme, Alexa ^a   Podetz Pedersen, Kelly M ^a   Riedl, Maureen ^b   Vulchanova, Lucy ^b   Robinson, Nicholas ^b   Hanson, Leah R ^c   Kozarsky, Karen F ^d   Orchard, Paul J ^b   Frey, William H ^c   Low, Walter C ^b   Scott McIvor, R ^a  

^a UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^b UNIVERSITY OF MINNESOTA   (United States)

^c REGIONS HOSPITAL   (United States)

^d REGENXBIO INC   (United States)

Author keywords

AAV; brain; iduronidase; Intranasal; MPS I; neurocognitive

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; GLYCOSAMINOGLYCAN; LEVO IDURONIDASE; GREEN FLUORESCENT PROTEIN;

ADENO ASSOCIATED VIRUS 9; ADULT; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BARNES MAZE TEST; BLOOD BRAIN BARRIER; BRAIN CORTEX; BRAIN STEM; CENTRAL NERVOUS SYSTEM DISEASE; CEREBELLUM; CONTROLLED STUDY; CORPUS STRIATUM; ENZYME ACTIVITY; GENE EXPRESSION; HETEROZYGOTE; HIPPOCAMPUS; HURLER SYNDROME; IMMUNOFLUORESCENCE; IMMUNOHISTOLOGY; IMMUNOREACTIVITY; KUPFFER CELL; LIVER; LUNG; MOUSE; NONHUMAN; NOSE EPITHELIUM; OLFACTORY BULB; OLFACTORY EPITHELIUM; OLFACTORY RECEPTOR NEURON; PURKINJE CELL; SPATIAL ORIENTATION; THALAMUS; TISSUE SECTION; VIRAL GENE DELIVERY SYSTEM; ANIMAL; CENTRAL NERVOUS SYSTEM; COGNITION; DEPENDOPARVOVIRUS; GENE TRANSFER; GENETIC TRANSDUCTION; GENETICS; HUMAN; INTRANASAL DRUG ADMINISTRATION; LYSOSOME; METABOLISM; NERVE CELL; NEUROLOGIC DISEASE; NOSE MUCOSA; PATHOLOGY; PATHOPHYSIOLOGY;

ADMINISTRATION, INTRANASAL; ANIMALS; CENTRAL NERVOUS SYSTEM; COGNITION; DEPENDOVIRUS; GENE TRANSFER TECHNIQUES; GREEN FLUORESCENT PROTEINS; HUMANS; IDURONIDASE; LYSOSOMES; MICE; MUCOPOLYSACCHARIDOSIS I; NASAL MUCOSA; NERVOUS SYSTEM DISEASES; NEURONS; OLFACTORY BULB; TRANSDUCTION, GENETIC;

EID: 85024895661     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2017.187     Document Type: Article

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