Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery

Molecular Therapy

Volumn 25, Issue 3, 2017, Pages 792-802

  Fu, Haiyan ^a,b   Meadows, Aaron S ^a   Ware, Tierra ^a   Mohney, Robert P ^c   McCarty, Douglas M ^a,b  

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

^b OHIO STATE UNIVERSITY   (United States)

^c METABOLON INC   (United States)

Author keywords

AAV9; biomarkers; global metabolomics profiling; MPS IIIB; systemic gene delivery

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; ALPHA TOCOPHEROL; ASCORBIC ACID; ASPARTIC ACID; GLUTAMIC ACID; N ACETYLTYROSINE; NUCLEOTIDE; PLASMID DNA; PYRIDOXINE; RECOMBINANT ADENO ASSOCIATED VIRUS 9 HNAGLU; RECOMBINANT DNA; RIBOFLAVIN; SEROTONIN; TRYPTOPHAN; UNCLASSIFIED DRUG; ACETYLGLUCOSAMINIDASE; AGENTS INTERACTING WITH TRANSMITTER, HORMONE OR DRUG RECEPTORS; ALPHA-N-ACETYL-D-GLUCOSAMINIDASE; BIOLOGICAL MARKER;

ADENO ASSOCIATED VIRUS 9; AMINO ACID METABOLISM; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CARBOHYDRATE METABOLISM; CONTROLLED STUDY; DISEASE COURSE; ENERGY METABOLISM; FEMALE; GENE TARGETING; GENE THERAPY; LIPID METABOLISM; MALE; METABOLOMICS; MOLECULARLY TARGETED THERAPY; MOUSE; NONHUMAN; NUCLEOTIDE METABOLISM; RAAV9 HNAGLU GENE; SANFILIPPO SYNDROME; TREATMENT RESPONSE; VIRAL GENE DELIVERY SYSTEM; VIRUS GENE; VITAMIN METABOLISM; XENOBIOTIC METABOLISM; ADMINISTRATION AND DOSAGE; ANIMAL; CLUSTER ANALYSIS; DEPENDOPARVOVIRUS; DISEASE EXACERBATION; DISEASE MODEL; GENE TRANSFER; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; GLYCOSYLATION; HUMAN; METABOLISM; METABOLOME; PHENOTYPE; PROCEDURES; TREATMENT OUTCOME;

ACETYLGLUCOSAMINIDASE; ANIMALS; BIOMARKERS; CLUSTER ANALYSIS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DISEASE PROGRESSION; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; GLYCOSYLATION; HUMANS; METABOLIC NETWORKS AND PATHWAYS; METABOLOME; METABOLOMICS; MICE; MUCOPOLYSACCHARIDOSIS III; NEUROTRANSMITTER AGENTS; PHENOTYPE; TRANSDUCTION, GENETIC; TREATMENT OUTCOME;

EID: 85016257823     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2016.12.025     Document Type: Article

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