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There are 3011 results for: content related to: Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha-1-anti-trypsin deficiency

  1. Novel Treatment Strategies for Liver Disease Due to a1-Antitrypsin Deficiency

    Clinical and Translational Science

    Volume 5, Issue 3, June 2012, Pages: 289–294, Nicholas Maurice and David H. Perlmutter

    Article first published online : 10 JAN 2012, DOI: 10.1111/j.1752-8062.2011.00363.x

  2. You have full text access to this OnlineOpen article
    Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease

    EMBO Molecular Medicine

    Volume 5, Issue 5, May 2013, Pages: 691–706, Carmine Spampanato, Erin Feeney, Lishu Li, Monica Cardone, Jeong-A Lim, Fabio Annunziata, Hossein Zare, Roman Polishchuk, Rosa Puertollano, Giancarlo Parenti, Andrea Ballabio and Nina Raben

    Article first published online : 18 APR 2013, DOI: 10.1002/emmm.201202176

  3. You have full text access to this OnlineOpen article
    Selective clearance of aberrant tau proteins and rescue of neurotoxicity by transcription factor EB

    EMBO Molecular Medicine

    Volume 6, Issue 9, September 2014, Pages: 1142–1160, Vinicia A Polito, Hongmei Li, Heidi Martini-Stoica, Baiping Wang, Li Yang, Yin Xu, Daniel B Swartzlander, Michela Palmieri, Alberto di Ronza, Virginia M-Y Lee, Marco Sardiello, Andrea Ballabio and Hui Zheng

    Article first published online : 28 JUL 2014, DOI: 10.15252/emmm.201303671

  4. You have full text access to this OnlineOpen article
    A lysosome-to-nucleus signalling mechanism senses and regulates the lysosome via mTOR and TFEB

    The EMBO Journal

    Volume 31, Issue 5, March 7, 2012, Pages: 1095–1108, Carmine Settembre, Roberto Zoncu, Diego L Medina, Francesco Vetrini, Serkan Erdin, SerpilUckac Erdin, Tuong Huynh, Mathieu Ferron, Gerard Karsenty, Michel C Vellard, Valeria Facchinetti, David M Sabatini and Andrea Ballabio

    Article first published online : 17 FEB 2012, DOI: 10.1038/emboj.2012.32

  5. You have full text access to this OnlineOpen article
    Regulation of TFEB and V-ATPases by mTORC1

    The EMBO Journal

    Volume 30, Issue 16, August 17, 2011, Pages: 3242–3258, Samuel Peña-Llopis, Silvia Vega-Rubin-de-Celis, Jacob C Schwartz, Nicholas C Wolff, Tram Anh T Tran, Lihua Zou, Xian-Jin Xie, David R Corey and James Brugarolas

    Article first published online : 29 JUL 2011, DOI: 10.1038/emboj.2011.257

  6. You have free access to this content
    Molecular pathogenesis of liver disease in α1-antitrypsin deficiency


    Volume 24, Issue 6, December 1996, Pages: 1504–1516, J H Teckman, D Qu and D H Perlmutter

    Article first published online : 30 DEC 2003, DOI: 10.1002/hep.510240635

  7. Alpha-1-Antitrypsin Deficiency and Related Disorders

    Textbook of Hepatology: From Basic Science to Clinical Practice, Third Edition

    Juan Rodés, Jean-Pierre Benhamou, Andres T. Blei, Jürg Reichen, Mario Rizzetto, Pages: 1330–1335, 2008

    Published Online : 15 APR 2008, DOI: 10.1002/9780470691861.ch16c

  8. You have free access to this content
    Quantitative isolation of α1AT mutant Z protein polymers from human and mouse livers and the effect of heat


    Volume 41, Issue 1, January 2005, Pages: 160–167, Jae-Koo An, Keith Blomenkamp, Douglas Lindblad and Jeffrey H. Teckman

    Article first published online : 23 DEC 2004, DOI: 10.1002/hep.20508

  9. An efficient and scalable process for helper-dependent adenoviral vector production using polyethylenimine-adenofection

    Biotechnology and Bioengineering

    Volume 102, Issue 3, 15 February 2009, Pages: 800–810, E. Dormond, A. Meneses-Acosta, D. Jacob, Y. Durocher, R. Gilbert, M. Perrier and A. Kamen

    Article first published online : 2 SEP 2008, DOI: 10.1002/bit.22113

  10. Broad Spectrum of Hepatocyte Inclusions in Humans, Animals, and Experimental Models

    Standard Article

    Comprehensive Physiology

    Pavel Strnad, Renwar Nuraldeen, Nurdan Guldiken, Daniel Hartmann, Vineet Mahajan, Helmut Denk and Johannes Haybaeck

    Published Online : 22 OCT 2013, DOI: 10.1002/cphy.c120032

  11. Helper-Dependent Adenoviral Vectors


    Current Protocols in Human Genetics


    Kazuhiro Oka and Lawrence Chan

    Published Online : 1 NOV 2005, DOI: 10.1002/0471142905.hg1213s47

  12. You have free access to this content
    Helper-dependent adenoviral gene therapy mediates long-term correction of the clotting defect in the canine hemophilia A model

    Journal of Thrombosis and Haemostasis

    Volume 4, Issue 6, June 2006, Pages: 1218–1225, W. M. McCORMACK JR, M. P. SEILER, T. K. BERTIN, K. UBHAYAKAR, D. J. PALMER, P. NG, T. C. NICHOLS and B. LEE

    Article first published online : 17 MAY 2006, DOI: 10.1111/j.1538-7836.2006.01901.x

  13. You have free access to this content
    Indomethacin increases liver damage in a murine model of liver injury from alpha-1-antitrypsin deficiency


    Volume 44, Issue 4, October 2006, Pages: 976–982, David A. Rudnick, Olga Shikapwashya, Keith Blomenkamp and Jeffrey H. Teckman

    Article first published online : 27 SEP 2006, DOI: 10.1002/hep.21326

  14. Alpha-1 Antitrypsin Deficiency

    Schiff's Diseases of the Liver, Eleventh Edition

    Eugene R. Schiff, Willis C. Maddrey, Michael F. Sorrell, Pages: 845–867, 2011

    Published Online : 31 OCT 2011, DOI: 10.1002/9781119950509.ch31

  15. Molecular analysis of the Pi*Z allele in patients with liver disease

    American Journal of Medical Genetics

    Volume 104, Issue 4, 15 December 2001, Pages: 287–290, Luciane Cauduro Lima, Ursula Matte, Sandra Leistner, Ana Rosa Bopp, Viviane Colombo Scholl, Roberto Giugliani and Themis Reverbel da Silveira

    Article first published online : 29 OCT 2001, DOI: 10.1002/ajmg.10069

  16. Phenotypic correction of ornithine transcarbamylase deficiency using low dose helper-dependent adenoviral vectors

    The Journal of Gene Medicine

    Volume 10, Issue 8, August 2008, Pages: 890–896, Nicola Brunetti-Pierri, Christian Clarke, Viraj Mane, Donna J. Palmer, Brendan Lanpher, Qin Sun, William O'Brien and Brendan Lee

    Article first published online : 19 JUN 2008, DOI: 10.1002/jgm.1218

  17. Biopump: Autologous skin-derived micro-organ genetically engineered to provide sustained continuous secretion of therapeutic proteins

    Dermatologic Therapy


    Article first published online : 21 FEB 2012, DOI: 10.1111/j.1529-8019.2012.01457.x

  18. Characterization of t(6;11)(p21;q12) in a renal-cell carcinoma of an adult patient

    Genes, Chromosomes and Cancer

    Volume 46, Issue 5, May 2007, Pages: 419–426, Lorenza Pecciarini, M. Giulia Cangi, Crocifissa Lo Cunsolo, Ettore Macri', Elena Dal Cin, Guido Martignoni and Claudio Doglioni

    Article first published online : 6 FEB 2007, DOI: 10.1002/gcc.20422

  19. You have free access to this content
    Transcription factor EB: A central regulator of both the autophagosome and lysosome


    Volume 55, Issue 5, May 2012, Pages: 1632–1634, Enpeng Zhao and Mark J. Czaja

    Article first published online : 19 APR 2012, DOI: 10.1002/hep.25619

  20. Gene therapy strategies for hemophilia: benefits versus risks

    The Journal of Gene Medicine

    Volume 12, Issue 10, October 2010, Pages: 797–809, Inge Petrus, Marinee Chuah and Thierry VandenDriessche

    Article first published online : 16 SEP 2010, DOI: 10.1002/jgm.1500