Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy

Science Translational Medicine

Volumn 2, Issue 35, 2010, Pages

  Valori, Chiara F ^a   Ning, Ke ^a   Wyles, Matthew ^a   Mead, Richard J ^a   Grierson, Andrew J ^a   Shaw, Pamela J ^a   Azzouz, Mimoun ^a  

^a UNIVERSITY OF SHEFFIELD   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

GREEN FLUORESCENT PROTEIN; PARVOVIRUS VECTOR; SURVIVAL MOTOR NEURON PROTEIN; SURVIVAL MOTOR NEURON PROTEIN 1;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CODON; CONTROLLED STUDY; FIBROBLAST; GENE REPLACEMENT THERAPY; HUMAN; HUMAN CELL; IMMUNOBLOTTING; IMMUNOCYTOCHEMISTRY; IMMUNOHISTOCHEMISTRY; IN VIVO GENE TRANSFER; MOTOR PERFORMANCE; MOUSE; NEWBORN; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; PROTEIN EXPRESSION; SINGLE DRUG DOSE; SPINAL MUSCULAR ATROPHY; SURVIVAL RATE; VIRAL GENE DELIVERY SYSTEM; ADENO ASSOCIATED VIRUS; ANIMAL; DISEASE MODEL; GENE THERAPY; GENE TRANSFER; GENETICS; METABOLISM; MOLECULAR GENETICS; MOTOR ACTIVITY; MUTATION; NUCLEOTIDE SEQUENCE; PATHOLOGY; PATHOPHYSIOLOGY; PHYSIOLOGY; SURVIVAL;

ANIMALS; ANIMALS, NEWBORN; BASE SEQUENCE; CODON; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; FIBROBLASTS; GENE THERAPY; GENE TRANSFER TECHNIQUES; MICE; MOLECULAR SEQUENCE DATA; MOTOR ACTIVITY; MUSCULAR ATROPHY, SPINAL; MUTATION; PHENOTYPE; SURVIVAL ANALYSIS; SURVIVAL OF MOTOR NEURON 1 PROTEIN;

EID: 77955602597     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.3000830     Document Type: Article

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30. Acknowledgments: We thank K. Mitrophanous for his help with codon optimization of SMN. Lentiviral scAAV-GFP and pAAV2/9 plasmids were gifts from N. Deglon, J. Gray, and J. Wilson, respectively. We also thank J. Haylor who allowed us to use his stereomicroscope for the intravenous injection and S. Wharton for helpful advice on heart pathology. Funding: Jennifer Trust for SMA, SMA Trust, Sheffield Hospitals Charitable Trust, Wellcome Trust (P.J.S.), and FightSMA (M.W.). Author contributions: C.F.V. and K.N. designed, performed, and analyzed the in vitro and in vivo experiments; M.W. was involved in lentiviral production and titration; R.J.M., A.J.G., and P.J.S. dealt with the UK Home Office regulation and edited the paper; M.A. designed the experiments; M.A. and C.F.V. wrote and edited the paper. Competing interests: The authors declare that they have no competing interests.