AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease

Science Translational Medicine

Volumn 7, Issue 313, 2015, Pages

  Katz, Martin L ^a   Tecedor, Luis ^b   Chen, Yonghong ^b   Williamson, Baye G ^c   Lysenko, Elena ^c   Wininger, Fred A ^c   Young, Whitney M ^c   Johnson, Gayle C ^d   Whiting, Rebecca E H ^e   Coates, Joan R ^e   Davidson, Beverly L ^a  

^a University of Missouri   (United States)

^b CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^c UNIVERSITY OF MISSOURI   (United States)

^d UNIVERSITY OF PENNSYLVANIA   (United States)

^e Veterinary Specialty Services   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; CYCLOSPORIN; ENHANCED GREEN FLUORESCENT PROTEIN; MYCOPHENOLATE MOFETIL; PROTON TRANSPORTING ADENOSINE TRIPHOSPHATE SYNTHASE; PROTON TRANSPORTING ADENOSINE TRIPHOSPHATE SYNTHASE C; TRIPEPTIDYL PEPTIDASE I; UNCLASSIFIED DRUG; AMINOPEPTIDASE; DIPEPTIDYL PEPTIDASE; SERINE PROTEINASE; TRIPEPTIDYL-PEPTIDASE 1;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY TITER; ARTICLE; ASTROCYTE; CELL ACTIVATION; CEREBELLAR ATAXIA; CONTROLLED STUDY; DACHSHUND (DOG); DRUG DOSE REDUCTION; DRUG MECHANISM; DRUG TOLERABILITY; ENZYME ACTIVITY; ENZYME REPLACEMENT; GENE EXPRESSION; IMMUNOREACTIVITY; LIFE EXTENSION; MENTAL DETERIORATION; MOUSE; NEURONAL CEROID LIPOFUSCINOSIS; NONHUMAN; PRIORITY JOURNAL; SIGNAL TRANSDUCTION; TPP1 GENE; VIRAL GENE DELIVERY SYSTEM; ADMINISTRATION AND DOSAGE; ANIMAL; BRAIN VENTRICLE; CEREBROSPINAL FLUID; DEFICIENCY; DEPENDOPARVOVIRUS; DISEASE MODEL; DOG; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; METABOLISM; NEURONAL CEROID-LIPOFUSCINOSES;

AMINOPEPTIDASES; ANIMALS; CEREBRAL VENTRICLES; DEPENDOVIRUS; DIPEPTIDYL-PEPTIDASES AND TRIPEPTIDYL-PEPTIDASES; DISEASE MODELS, ANIMAL; DOGS; ENZYME REPLACEMENT THERAPY; GENETIC VECTORS; NEURONAL CEROID-LIPOFUSCINOSES; SERINE PROTEASES; TRANSDUCTION, GENETIC;

EID: 84947975895     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.aac6191     Document Type: Article

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