Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease

Proceedings of the National Academy of Sciences of the United States of America

Volumn 102, Issue 49, 2005, Pages 17822-17827

  Dodge, James C ^a   Clarke, Jennifer ^a   Song, Antonius ^a   Bu, Jie ^a   Yang, Wendy ^a   Taksir, Tatyana V ^a   Griffiths, Denise ^a   Zhao, Michael A ^a   Schuchman, Edward H ^b   Cheng, Seng H ^a   O'Riordan, Catherine R ^a   Shihabuddin, Lamya S ^a   Passini, Marco A ^a   Stewart, Gregory R ^a  

^a GENZYME CORPORATION   (United States)

^b MOUNT SINAI SCHOOL OF MEDICINE   (United States)

Author keywords

Adeno associated virus; Axonal transport; Deep cerebellar nuclei; Gene therapy; Lysosomal storage diseases

Indexed keywords

PARVOVIRUS VECTOR; SPHINGOMYELIN PHOSPHODIESTERASE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BRAIN STEM; CEREBELLUM NUCLEUS; CONTROLLED STUDY; GENE TRANSFER; HISTOLOGY; HUMAN; KNOCKOUT MOUSE; LYSOSOME STORAGE DISEASE; MALE; MESENCEPHALON; MOTOR DYSFUNCTION; MOUSE; NEUROPATHOLOGY; NIEMANN PICK DISEASE; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; SEROTYPE; SPINAL CORD; VIRAL GENE THERAPY;

ANIMALS; BRAIN; CALCIUM-BINDING PROTEIN, VITAMIN D-DEPENDENT; CELL COUNT; CENTRAL NERVOUS SYSTEM; CHOLESTEROL; DISEASE MODELS, ANIMAL; GENE THERAPY; HUMANS; MALE; MICE; MICE, KNOCKOUT; MOTOR NEURONS; NIEMANN-PICK DISEASES; PURKINJE CELLS; SPHINGOMYELIN PHOSPHODIESTERASE; SPHINGOMYELINS;

ADENO-ASSOCIATED VIRUS; ANIMALIA; PARVOVIRUS;

EID: 29144442863     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.0509062102     Document Type: Article

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