Hasil Pencarian - Seng H Cheng

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  1. 1
    Gene therapy for the neurological manifestations in lysosomal storage disorders

    Gene therapy for the neurological manifestations in lysosomal storage disorders oleh Seng H. Cheng

    Diterbitkan 2014-09-01
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  2. 2
    Proteasome inhibition is partially effective in attenuating pre-existing immunity against recombinant adeno-associated viral vectors.

    Proteasome inhibition is partially effective in attenuating pre-existing immunity against recombinant adeno-associated viral vectors. oleh Jozsef Karman, Nathan K Gumlaw, Jinhua Zhang, Ji-Lei Jiang, Seng H Cheng, Yunxiang Zhu

    Diterbitkan 2012-01-01
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  3. 3
    Rationale for using centralized transduction inhibition assays in three phase 3 rAAV gene therapy clinical trials

    Rationale for using centralized transduction inhibition assays in three phase 3 rAAV gene therapy clinical trials oleh Martin Schulz, George Bashirians, Seng H. Cheng, Daniel I. Levy, Mark Lundie, Lisa Wilcox, Ian Winburn, Suryanarayan Somanathan

    Diterbitkan 2023-12-01
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  4. 4
    Dysregulation of multiple facets of glycogen metabolism in a murine model of Pompe disease.

    Dysregulation of multiple facets of glycogen metabolism in a murine model of Pompe disease. oleh Kristin M Taylor, Elizabeth Meyers, Michael Phipps, Priya S Kishnani, Seng H Cheng, Ronald K Scheule, Rodney J Moreland

    Diterbitkan 2013-01-01
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  5. 5
    Comparative analysis of acid sphingomyelinase distribution in the CNS of rats and mice following intracerebroventricular delivery.

    Comparative analysis of acid sphingomyelinase distribution in the CNS of rats and mice following intracerebroventricular delivery. oleh Christopher M Treleaven, Thomas Tamsett, Jonathan A Fidler, Tatyana V Taksir, Seng H Cheng, Lamya S Shihabuddin, James C Dodge

    Diterbitkan 2011-01-01
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  6. 6
    Glucosylceramide synthase inhibition reduces ganglioside GM3 accumulation, alleviates amyloid neuropathology, and stabilizes remote contextual memory in a mouse model of Alzheimer’s disease

    Glucosylceramide synthase inhibition reduces ganglioside GM3 accumulation, alleviates amyloid neuropathology, and stabilizes remote contextual memory in a mouse model of Alzheimer’... oleh James C. Dodge, Thomas J. Tamsett, Christopher M. Treleaven, Tatyana V. Taksir, Peter Piepenhagen, S. Pablo Sardi, Seng H. Cheng, Lamya S. Shihabuddin

    Diterbitkan 2022-02-01
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  7. 7
    A novel mixing device for the reproducible generation of nonviral gene therapy formulations

    A novel mixing device for the reproducible generation of nonviral gene therapy formulations oleh Lee A. Davies, Graciela A. Nunez-Alonso, Henry L. Hebel, Ron K. Scheule, Seng H. Cheng, Stephen C. Hyde, Deborah R. Gill

    Diterbitkan 2010-09-01
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  8. 8
    A bispecific protein capable of engaging CTLA-4 and MHCII protects non-obese diabetic mice from autoimmune diabetes.

    A bispecific protein capable of engaging CTLA-4 and MHCII protects non-obese diabetic mice from autoimmune diabetes. oleh Hongmei Zhao, Jozsef Karman, Ji-Lei Jiang, Jinhua Zhang, Nathan Gumlaw, John Lydon, Qun Zhou, Huawei Qiu, Canwen Jiang, Seng H Cheng, Yunxiang Zhu

    Diterbitkan 2013-01-01
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  9. 9
    IGF-1 delivery to CNS attenuates motor neuron cell death but does not improve motor function in type III SMA mice

    IGF-1 delivery to CNS attenuates motor neuron cell death but does not improve motor function in type III SMA mice oleh Li-Kai Tsai, Yi-Chun Chen, Wei-Cheng Cheng, Chen-Hung Ting, James C. Dodge, Wuh-Liang Hwu, Seng H. Cheng, Marco A. Passini

    Diterbitkan 2012-01-01
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  10. 10
    The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo

    The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo oleh Sirkka Kyostio-Moore, Patricia Berthelette, Susan Piraino, Cathleen Sookdeo, Bindu Nambiar, Robert Jackson, Brenda Burnham, Catherine R O'Riordan, Seng H Cheng, Donna Armentano

    Diterbitkan 2016-01-01
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  11. 11
    Increased hepatic insulin action in diet-induced obese mice following inhibition of glucosylceramide synthase.

    Increased hepatic insulin action in diet-induced obese mice following inhibition of glucosylceramide synthase. oleh Nelson S Yew, Hongmei Zhao, Eun-Gyoung Hong, I-Huan Wu, Malgorzata Przybylska, Craig Siegel, James A Shayman, Cynthia M Arbeeny, Jason K Kim, Canwen Jiang, Seng H Cheng

    Diterbitkan 2010-06-01
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  12. 12
    Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease

    Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease oleh Nicholas P Clayton, Carol A Nelson, Timothy Weeden, Kristin M Taylor, Rodney J Moreland, Ronald K Scheule, Lucy Phillips, Andrew J Leger, Seng H Cheng, Bruce M Wentworth

    Diterbitkan 2014-01-01
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  13. 13
    Dysregulated DNA methylation in the pathogenesis of Fabry disease

    Dysregulated DNA methylation in the pathogenesis of Fabry disease oleh Jin-Song Shen, Uthra Balaji, Kunitoshi Shigeyasu, Yoshinaga Okugawa, Siamak Jabbarzadeh-Tabrizi, Taniqua S. Day, Erland Arning, John Marshall, Seng H. Cheng, Jinghua Gu, Raphael Schiffmann, Teodoro Bottiglieri, Ajay Goel

    Diterbitkan 2022-12-01
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  14. 14
    Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes

    Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes oleh Shelley A. Nass, Maryellen A. Mattingly, Denise A. Woodcock, Brenda L. Burnham, Jeffrey A. Ardinger, Shayla E. Osmond, Amy M. Frederick, Abraham Scaria, Seng H. Cheng, Catherine R. O’Riordan

    Diterbitkan 2018-06-01
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  15. 15
    Systemic delivery of a glucosylceramide synthase inhibitor reduces CNS substrates and increases lifespan in a mouse model of type 2 Gaucher disease.

    Systemic delivery of a glucosylceramide synthase inhibitor reduces CNS substrates and increases lifespan in a mouse model of type 2 Gaucher disease. oleh Mario A Cabrera-Salazar, Matthew Deriso, Scott D Bercury, Lingyun Li, John T Lydon, William Weber, Nilesh Pande, Mandy A Cromwell, Diane Copeland, John Leonard, Seng H Cheng, Ronald K Scheule

    Diterbitkan 2012-01-01
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  16. 16
    Substrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry disease.

    Substrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry disease. oleh John Marshall, Karen M Ashe, Dinesh Bangari, KerryAnne McEachern, Wei-Lien Chuang, Joshua Pacheco, Diane P Copeland, Robert J Desnick, James A Shayman, Ronald K Scheule, Seng H Cheng

    Diterbitkan 2010-11-01
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  17. 17
    Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease

    Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease oleh Piotr Hadaczek, Lisa Stanek, Agnieszka Ciesielska, Vivek Sudhakar, Lluis Samaranch, Philip Pivirotto, John Bringas, Catherine O'Riordan, Bryan Mastis, Waldy San Sebastian, John Forsayeth, Seng H Cheng, Krystof S Bankiewicz, Lamya S Shihabuddin

    Diterbitkan 2016-01-01
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  18. 18
    GBA2-encoded β-glucosidase activity is involved in the inflammatory response to Pseudomonas aeruginosa.

    GBA2-encoded β-glucosidase activity is involved in the inflammatory response to Pseudomonas aeruginosa. oleh Nicoletta Loberto, Maela Tebon, Ilaria Lampronti, Nicola Marchetti, Massimo Aureli, Rosaria Bassi, Maria Grazia Giri, Valentino Bezzerri, Valentina Lovato, Cinzia Cantù, Silvia Munari, Seng H Cheng, Alberto Cavazzini, Roberto Gambari, Sandro Sonnino, Giulio Cabrini, Maria Cristina Dechecchi

    Diterbitkan 2014-01-01
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  19. 19
    Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease.

    Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease. oleh Karen M Ashe, Dinesh Bangari, Lingyun Li, Mario A Cabrera-Salazar, Scott D Bercury, Jennifer B Nietupski, Christopher G F Cooper, Johannes M F G Aerts, Edward R Lee, Diane P Copeland, Seng H Cheng, Ronald K Scheule, John Marshall

    Diterbitkan 2011-01-01
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  20. 20
    A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis

    A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with... oleh Eric WFW Alton, David K Armstrong, Deborah Ashby, Katie J Bayfield, Diana Bilton, Emily V Bloomfield, A Christopher Boyd, June Brand, Ruaridh Buchan, Roberto Calcedo, Paula Carvelli, Mario Chan, Seng H Cheng, David S Collie, Steve Cunningham, Heather E Davidson, Gwyneth Davies, Jane C Davies, Lee A Davies, Maria H Dewar, Ann Doherty, Jackie Donovan, Natalie S Dwyer, Hala I Elgmati, Rosanna F Featherstone, Jemyr Gavino, Sabrina Gea-Sorli, Duncan M Geddes, James SR Gibson, Deborah R Gill, Andrew P Greening, Uta Griesenbach, David M Hansell, Katharine Harman, Tracy E Higgins, Samantha L Hodges, Stephen C Hyde, Laura Hyndman, J Alastair Innes, Joseph Jacob, Nancy Jones, Brian F Keogh, Maria P Limberis, Paul Lloyd-Evans, Alan W Maclean, Michelle C Manvell, Dominique McCormick, Michael McGovern, Gerry McLachlan, Cuixiang Meng, M Angeles Montero, Hazel Milligan, Laura J Moyce, Gordon D Murray, Andrew G Nicholson, Tina Osadolor, Javier Parra-Leiton, David J Porteous, Ian A Pringle, Emma K Punch, Kamila M Pytel, Alexandra L Quittner, Gina Rivellini, Clare J Saunders, Ronald K Scheule, Sarah Sheard, Nicholas J Simmonds, Keith Smith, Stephen N Smith, Najwa Soussi, Samia Soussi, Emma J Spearing, Barbara J Stevenson, Stephanie G Sumner-Jones, Minna Turkkila, Rosa P Ureta, Michael D Waller, Marguerite Y Wasowicz, James M Wilson, Paul Wolstenholme-Hogg, on behalf of the UK Cystic Fibrosis Gene Therapy Consortium

    Diterbitkan 2016-07-01
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