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Volumn 58, Issue 1, 2016, Pages 30-36

Large-Scale Production of Adeno-Associated Viral Vector Serotype-9 Carrying the Human Survival Motor Neuron Gene

Author keywords

Adeno associated viral vector; Electron microscopy; Gene therapy; Human embryonic kidney 293 cells; Spinal muscular atrophy (SMA); Survival Motor Neuron (SMN) gene; Transient transfection

Indexed keywords

BRAIN; CELLS; CLONING; CYTOLOGY; ELECTRON MICROSCOPES; ELECTRON MICROSCOPY; GENE EXPRESSION; GENES; MOLECULAR BIOLOGY; NEURONS; QUALITY CONTROL; VECTORS;

EID: 84954376750     PISSN: 10736085     EISSN: 15590305     Source Type: Journal    
DOI: 10.1007/s12033-015-9899-5     Document Type: Article
Times cited : (21)

References (15)
  • 1
    • 0036154959 scopus 로고    scopus 로고
    • Quantitative analyses of smn1 and smn2 based on real-time lightcycler pcr: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy
    • Feldkötter, M., Schwarzer, V., Wirth, R., Wienker, T. F., & Wirth, B. (2002). Quantitative analyses of smn1 and smn2 based on real-time lightcycler pcr: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy. The American Journal of Human Genetics,70, 358–368.
    • (2002) The American Journal of Human Genetics , vol.70 , pp. 358-368
    • Feldkötter, M.1    Schwarzer, V.2    Wirth, R.3    Wienker, T.F.4    Wirth, B.5
  • 2
    • 70249095607 scopus 로고    scopus 로고
    • Spinal muscular atrophy and a model for survival of motor neuron protein function in axonal ribonucleoprotein complexes
    • COI: 1:CAS:528:DC%2BC3cXhsF2rtLrI
    • Rossoll, W., & Bassell, G. J. (2009). Spinal muscular atrophy and a model for survival of motor neuron protein function in axonal ribonucleoprotein complexes. Results and Problems in Cell Differentiation,48, 289–326.
    • (2009) Results and Problems in Cell Differentiation , vol.48 , pp. 289-326
    • Rossoll, W.1    Bassell, G.J.2
  • 5
    • 77749249680 scopus 로고    scopus 로고
    • Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
    • COI: 1:CAS:528:DC%2BC3cXisFelurs%3D
    • Foust, K. D., Wang, X., McGovern, V. L., Braun, L., Bevan, A. K., Haidet, A. M., et al. (2010). Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nature Biotechnology,28, 271–274.
    • (2010) Nature Biotechnology , vol.28 , pp. 271-274
    • Foust, K.D.1    Wang, X.2    McGovern, V.L.3    Braun, L.4    Bevan, A.K.5    Haidet, A.M.6
  • 7
    • 84873410199 scopus 로고    scopus 로고
    • Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice
    • COI: 1:CAS:528:DC%2BC3sXkt1Wntw%3D%3D
    • Benkhelifa-Ziyyat, S., Besse, A., Roda, M., Duque, S., Astord, S., Carcenac, R., et al. (2013). Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice. Molecular Therapy,21, 282–290.
    • (2013) Molecular Therapy , vol.21 , pp. 282-290
    • Benkhelifa-Ziyyat, S.1    Besse, A.2    Roda, M.3    Duque, S.4    Astord, S.5    Carcenac, R.6
  • 8
    • 84904412918 scopus 로고    scopus 로고
    • Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy
    • COI: 1:CAS:528:DC%2BC2cXhtFKmurrF
    • Passini, M. A., Bu, J., Richards, A. M., Treleaven, C. M., Sullivan, J. A., O’Riordan, C. R., et al. (2014). Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy. Human Gene Therapy,25, 619–630.
    • (2014) Human Gene Therapy , vol.25 , pp. 619-630
    • Passini, M.A.1    Bu, J.2    Richards, A.M.3    Treleaven, C.M.4    Sullivan, J.A.5    O’Riordan, C.R.6
  • 9
    • 83455195614 scopus 로고    scopus 로고
    • Exploiting natural diversity of AAV for the design of vectors with novel properties
    • COI: 1:CAS:528:DC%2BC38XhsFGls7nK
    • Gao, G. P., Zhong, L., & Danos, O. (2011). Exploiting natural diversity of AAV for the design of vectors with novel properties. Methods in Molecular Biology,807, 93–118.
    • (2011) Methods in Molecular Biology , vol.807 , pp. 93-118
    • Gao, G.P.1    Zhong, L.2    Danos, O.3
  • 10
    • 84868158039 scopus 로고    scopus 로고
    • introducing genes into mammalian cells: Viral vectors
    • Green MR, Sambrook J, (eds), 2, Cold Spring Harbor Laboratory Press, New York
    • Gao, G. P., & Sena-Esteves, M. (2012). introducing genes into mammalian cells: Viral vectors. In M. R. Green & J. Sambrook (Eds.), Molecular cloning: A laboratory manual (Vol. 2, pp. 1209–1313). New York: Cold Spring Harbor Laboratory Press.
    • (2012) Molecular cloning: A laboratory manual , pp. 1209-1313
    • Gao, G.P.1    Sena-Esteves, M.2
  • 11
    • 84874640802 scopus 로고    scopus 로고
    • Viral vectors: a look back and ahead on gene transfer technology
    • COI: 1:CAS:528:DC%2BC3sXhvV2mu7zO
    • Vannucci, L., Lai, M., Chiuppesi, F., & Pistello, M. (2013). Viral vectors: a look back and ahead on gene transfer technology. New Microbiologica,36, 1–22.
    • (2013) New Microbiologica , vol.36 , pp. 1-22
    • Vannucci, L.1    Lai, M.2    Chiuppesi, F.3    Pistello, M.4
  • 12
    • 84857602982 scopus 로고    scopus 로고
    • Adeno-associated virus vectorology, manufacturing, and clinical applications
    • COI: 1:CAS:528:DC%2BC38XlvFKjs74%3D
    • Grieger, J. C., & Samulski, R. J. (2012). Adeno-associated virus vectorology, manufacturing, and clinical applications. Methods in Enzymology,507, 229–254.
    • (2012) Methods in Enzymology , vol.507 , pp. 229-254
    • Grieger, J.C.1    Samulski, R.J.2
  • 13
    • 0034202522 scopus 로고    scopus 로고
    • Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro
    • COI: 1:CAS:528:DC%2BD38XhvF2htr0%3D
    • Miao, C. H., Ohashi, K., Patijn, G. A., Meuse, L., Ye, X., Thompson, A. R., et al. (2000). Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro. Molecular Therapy,1, 522–532.
    • (2000) Molecular Therapy , vol.1 , pp. 522-532
    • Miao, C.H.1    Ohashi, K.2    Patijn, G.A.3    Meuse, L.4    Ye, X.5    Thompson, A.R.6
  • 14
    • 0036592086 scopus 로고    scopus 로고
    • A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo
    • COI: 1:CAS:528:DC%2BD38XktFyrsrY%3D
    • Ehrhardt, A., & Kay, M. A. (2002). A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo. Blood,99, 3923–3930.
    • (2002) Blood , vol.99 , pp. 3923-3930
    • Ehrhardt, A.1    Kay, M.A.2
  • 15
    • 77958104835 scopus 로고    scopus 로고
    • Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale
    • COI: 1:CAS:528:DC%2BC3cXht12ju7nJ
    • Lock, M., Alvira, M., Vandenberghe, L. H., Samanta, A., Toelen, J., Debyser, Z., et al. (2010). Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. Human Gene Therapy,21, 1259–1271.
    • (2010) Human Gene Therapy , vol.21 , pp. 1259-1271
    • Lock, M.1    Alvira, M.2    Vandenberghe, L.H.3    Samanta, A.4    Toelen, J.5    Debyser, Z.6


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.