Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis

Human Gene Therapy Methods

Volumn 23, Issue 5, 2012, Pages 324-335

  Sondhi, Dolan ^a   Johnson, Linda ^b,c   Purpura, Keith ^a   Monette, Sebastien ^b   Souweidane, Mark M ^a   Kaplitt, Michael G ^a   Kosofsky, Barry ^a   Yohay, Kaleb ^a   Ballon, Douglas ^a   Dyke, Jonathan ^a   Kaminksy, Stephen M ^a   Hackett, Neil R ^a   Crystal, Ronald G ^a  

^a WEILL CORNELL MEDICAL COLLEGE   (United States)

^b ROCKEFELLER UNIVERSITY   (United States)

^c JAMES COOK UNIVERSITY   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

ALANINE AMINOTRANSFERASE; ASPARTATE AMINOTRANSFERASE; CLN2 PROTEIN; GENE THERAPY AGENT; PARVOVIRUS VECTOR; PEPTIDASE; TRIPEPTIDYL PEPTIDASE I; UNCLASSIFIED DRUG;

ALANINE AMINOTRANSFERASE BLOOD LEVEL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; AUTOSOMAL RECESSIVE DISORDER; BLOOD CELL COUNT; BRAIN EDEMA; CHLOROCEBUS; CONTROLLED STUDY; CORPUS STRIATUM; DRUG EFFICACY; DRUG SAFETY; ENCEPHALITIS; ENZYME ACTIVITY; FEMALE; FRONTAL CORTEX; GENE EXPRESSION; GENE MUTATION; GENOME; GLIOSIS; HISTOPATHOLOGY; LATE INFANTILE NEURONAL CEROID LIPOFUSCINOSIS; MALE; NEURONAL CEROID LIPOFUSCINOSIS; NEUROPIL; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; RAT; RISK ASSESSMENT; SEROTYPE; VIRAL GENE DELIVERY SYSTEM; WHITE MATTER;

AMINOPEPTIDASES; ANIMALS; ANTIBODIES, NEUTRALIZING; ANTIBODIES, VIRAL; BEHAVIOR, ANIMAL; BRAIN; CELL LINE; DEPENDOVIRUS; DIPEPTIDYL-PEPTIDASES AND TRIPEPTIDYL-PEPTIDASES; FEMALE; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MALE; NEURONAL CEROID-LIPOFUSCINOSES; PRIMATES; RATS; SERINE PROTEASES; TIME FACTORS; TRANSDUCTION, GENETIC;

EID: 84875443169     PISSN: 19466536     EISSN: 19466544     Source Type: Journal    
DOI: 10.1089/hgtb.2012.120     Document Type: Article

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