Safety of direct administration of AAV2CUhCLN2, a candidate treatment for the central nervous system manifestations of late infantile neuronal ceroid lipofuscinosis, to the brain of rats and nonhuman primates

Human Gene Therapy

Volumn 16, Issue 12, 2005, Pages 1484-1503

  Hackett, Neil R ^a   Redmond, D Eugene ^b,c   Sondhi, Dolan ^a   Giannaris, E Lela ^a   Vassallo, Elizabeth ^a   Stratton, Jamie ^a   Qiu, Jianping ^a   Kaminsky, Stephen M ^a   Lesser, Martin L ^d   Fisch, Gene S ^d   Rouselle, Serge D ^e   Crystal, Ronald G ^a  

^a WEILL CORNELL MEDICAL COLLEGE   (United States)

^b YALE UNIVERSITY   (United States)

^c Axion Research Foundation   (United States)

^d LONG ISLAND JEWISH MEDICAL CENTER   (United States)

^e CHARLES RIVER LABORATORIES   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; GENE PRODUCT; NEUTRALIZING ANTIBODY; PARVOVIRUS VECTOR; PEPTIDASE; PHOSPHATE; PROTEIN CLN2; SODIUM CHLORIDE; TRIPEPTIDYL PEPTIDASE I; UNCLASSIFIED DRUG;

ADENOASSOCIATED VIRUS VECTOR 2; ANIMAL TISSUE; ARTICLE; AUTOSOMAL RECESSIVE DISORDER; BRAIN CORTEX; BRAIN EDEMA; CAUDATE NUCLEUS; CELL DESTRUCTION; CENTRAL NERVOUS SYSTEM; CERCOPITHECIDAE; CLINICAL FEATURE; CLN2 GENE; CONTROLLED STUDY; DEGENERATIVE DISEASE; DRUG ADMINISTRATION ROUTE; DRUG SAFETY; DRUG TARGETING; FEMALE; GENE; GENE EXPRESSION; GENE MUTATION; GENE TRANSFER; GLIA CELL; HIPPOCAMPUS; HISTOPATHOLOGY; INTRASTRIATAL DRUG ADMINSTRATION; LYSOSOME STORAGE DISEASE; MALE; NEUROLOGIC EXAMINATION; NEURONAL CEROID LIPOFUSCINOSIS; NEURONAL CEROID LIPOFUSCINOSIS LATE INFANTILE TYPE; NONHUMAN; NUCLEOTIDE SEQUENCE; PRIMATE; QUANTITATIVE ANALYSIS; RAT; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS PARTICLE; WHITE MATTER;

ANIMALS; BRAIN EDEMA; CENTRAL NERVOUS SYSTEM; CERCOPITHECUS AETHIOPS; DEPENDOVIRUS; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; MALE; NEURONAL CEROID-LIPOFUSCINOSES; PLASMIDS; RATS; RATS, INBRED F344;

ANIMALIA; CERCOPITHECIDAE; CERCOPITHECUS AETHIOPS; PARVOVIRUS; PRIMATES;

EID: 29244460417     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2005.16.1484     Document Type: Article

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