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EMBO Molecular Medicine

Cover image for Vol. 6 Issue 2

February 2014

Volume 6, Issue 2

Pages 155–295

  1. Closeup

    1. Top of page
    2. Closeup
    3. Review
    4. Research Articles
    5. Corrigendum
    1. You have full text access to this OnlineOpen article
      Mixing and matching mitochondrial aminoacyl synthetases and their tRNAs: a new way to treat respiratory chain disorders? (pages 155–157)

      Henna Tyynismaa and Eric A. Schon

      Version of Record online: 29 JAN 2014 | DOI: 10.1002/emmm.201303586

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      Tyynismaa and Schon share their thoughts on two back-to-back papers published in this issue describing the ability of a human C-terminal mtLeuRS fragment to rescue pathogenic tRNA mutations in human patient-derived cytoplasmic hybrid cell models

  2. Review

    1. Top of page
    2. Closeup
    3. Review
    4. Research Articles
    5. Corrigendum
    1. You have full text access to this OnlineOpen article
      Tuberculosis drug discovery in the post-post-genomic era (pages 158–168)

      Benoit Lechartier, Jan Rybniker, Alimuddin Zumla and Stewart T Cole

      Version of Record online: 8 JAN 2014 | DOI: 10.1002/emmm.201201772

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      As tuberculosis remains one of the most devastating infectious diseases in the world, efficacious treatment is more than ever needed. This review provides a well-balanced and comprehensive overview of the tuberculosis drug discovery pipeline.

  3. Research Articles

    1. Top of page
    2. Closeup
    3. Review
    4. Research Articles
    5. Corrigendum
    1. You have full text access to this OnlineOpen article
      The isolated carboxy-terminal domain of human mitochondrial leucyl-tRNA synthetase rescues the pathological phenotype of mitochondrial tRNA mutations in human cells (pages 169–182)

      Elena Perli, Carla Giordano, Annalinda Pisano, Arianna Montanari, Antonio F Campese, Aurelio Reyes, Daniele Ghezzi, Alessia Nasca, Helen A Tuppen, Maurizia Orlandi, Patrizio Di Micco, Elena Poser, Robert W Taylor, Gianni Colotti, Silvia Francisci, Veronica Morea, Laura Frontali, Massimo Zeviani and Giulia d'Amati

      Version of Record online: 10 JAN 2014 | DOI: 10.1002/emmm.201303198

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      Non-cognate mitochondrial aminoacyl-tRNA syntethases improve viability and bionergetic proficiency of human cells with pathogenic mutations in the mt-tRNAIle gene. The isolated carboxy-terminal domain of human mt-leucyl tRNA synthetase improves the pathologic phenotype.

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      Human mitochondrial leucyl tRNA synthetase can suppress non cognate pathogenic mt-tRNA mutations (pages 183–193)

      Hue Tran Hornig-Do, Arianna Montanari, Agata Rozanska, Helen A Tuppen, Abdulraheem A Almalki, Dyg P Abg-Kamaludin, Laura Frontali, Silvia Francisci, Robert N Lightowlers and Zofia M Chrzanowska-Lightowlers

      Version of Record online: 10 JAN 2014 | DOI: 10.1002/emmm.201303202

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      A non-cognate mitochondrial aminoacyl tRNA synthetase can overcome the respiratory defect caused by an mt-tRNA mutation. The pathogenic mutation can be suppressed by a short peptide corresponding to the C-terminus of leucyl tRNA synthetase.

    3. You have full text access to this OnlineOpen article
      Effective delivery of large genes to the retina by dual AAV vectors (pages 194–211)

      Ivana Trapani, Pasqualina Colella, Andrea Sommella, Carolina Iodice, Giulia Cesi, Sonia de Simone, Elena Marrocco, Settimio Rossi, Massimo Giunti, Arpad Palfi, Gwyneth J Farrar, Roman Polishchuk and Alberto Auricchio

      Version of Record online: 16 DEC 2013 | DOI: 10.1002/emmm.201302948

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      The Authors provide proof-of-concept of gene therapy for two inherited retinal degeneration conditions by using dual AAV vectors. This allows the expansion of AAV cargo capacity for gene therapy of syndromes that require the transfer of large genes.

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      Rapid target gene validation in complex cancer mouse models using re-derived embryonic stem cells (pages 212–225)

      Ivo J Huijbers, Rahmen Bin Ali, Colin Pritchard, Miranda Cozijnsen, Min-Chul Kwon, Natalie Proost, Ji-Ying Song, Hilda de Vries, Jitendra Badhai, Kate Sutherland, Paul Krimpenfort, Ewa M Michalak, Jos Jonkers and Anton Berns

      Version of Record online: 15 JAN 2014 | DOI: 10.1002/emmm.201303297

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      The GEMM-ESC approach describes and validates an improved method for generating mouse cancer models directly from embryonic stem cells. This technology speeds up the generation/modification of mouse models, while minimizing cost and breeding efforts.

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      Inhibition of the immunoproteasome ameliorates experimental autoimmune encephalomyelitis (pages 226–238)

      Michael Basler, Sarah Mundt, Tony Muchamuel, Carlo Moll, Jing Jiang, Marcus Groettrup and Christopher J. Kirk

      Version of Record online: 7 JAN 2014 | DOI: 10.1002/emmm.201303543

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      The immunoproteasome is found implicated in the development of experimental autoimmune encephalomyelitis and LMP7 inhibitors shown promising for the treatment of multiple sclerosis and other autoimmune diseases that rely on Th1/Th17 response.

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      Targeting endothelial junctional adhesion molecule-A/ EPAC/ Rap-1 axis as a novel strategy to increase stem cell engraftment in dystrophic muscles (pages 239–258)

      Monica Giannotta, Sara Benedetti, Francesco Saverio Tedesco, Monica Corada, Marianna Trani, Rocco D'Antuono, Queensta Millet, Fabrizio Orsenigo, Beatriz G Gálvez, Giulio Cossu and Elisabetta Dejana

      Version of Record online: 30 DEC 2013 | DOI: 10.1002/emmm.201302520

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      Rap-1 inhibitors block junction tightening and allow muscle-cell progenitors extravasation, thereby enhancing their engraftment to dystrophic muscles, highlighting them as potential agents to optimize cell therapy protocols for muscular dystrophies.

    7. You have full text access to this OnlineOpen article
      Modelling and rescuing neurodevelopmental defect of Down syndrome using induced pluripotent stem cells from monozygotic twins discordant for trisomy 21 (pages 259–277)

      Youssef Hibaoui, Iwona Grad, Audrey Letourneau, M Reza Sailani, Sophie Dahoun, Federico A Santoni, Stefania Gimelli, Michel Guipponi, Marie Françoise Pelte, Frédérique Béna, Stylianos E Antonarakis and Anis Feki

      Version of Record online: 27 DEC 2013 | DOI: 10.1002/emmm.201302848

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      The generation and characterization of iPSCs from monozygotic twins discordant for trisomy 21 allows studying Down syndrome early embryonic development and pathogenesis. DYRK1A inhibition is further shown with therapeutic potentials for DS patients.

    8. You have full text access to this OnlineOpen article
      ADAM8 expression in invasive breast cancer promotes tumor dissemination and metastasis (pages 278–294)

      Mathilde Romagnoli, Nora D Mineva, Michael Polmear, Catharina Conrad, Srimathi Srinivasan, Delphine Loussouarn, Sophie Barillé-Nion, Irene Georgakoudi, Áine Dagg, Enda W McDermott, Michael J Duffy, Patricia M. McGowan, Uwe Schlomann, Maddy Parsons, Jörg W Bartsch and Gail E Sonenshein

      Version of Record online: 27 DEC 2013 | DOI: 10.1002/emmm.201303373

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      The transmembrane protein ADAM8 is shown to be a crucial player in multiple steps of breast tumorigenesis, notably in angiogenesis and cancer cell adhesion to the endothelium. The results validate ADAM8 as novel target for the treatment of aggressive triple-negative breast cancer.

  4. Corrigendum

    1. Top of page
    2. Closeup
    3. Review
    4. Research Articles
    5. Corrigendum
    1. You have full text access to this OnlineOpen article
      Stathmin regulates mutant p53 stability and transcriptional activity in ovarian cancer (page 295)

      Maura Sonego, Monica Schiappacassi, Sara Lovisa, Alessandra Dall'Acqua, Marina Bagnoli, Francesca Lovat, Massimo Libra, Sara D'Andrea, Vincenzo Canzonieri, Loredana Militello, Marco Napoli, Giorgio Giorda, Barbara Pivetta, Delia Mezzanzanica, Mattia Barbareschi, Barbara Valeri, Silvana Canevari, Alfonso Colombatti, Barbara Belletti, Giannino Del Sal and Gustavo Baldassarre

      Version of Record online: 7 FEB 2014 | DOI: 10.1002/emmm.201470020

      This article corrects:

      Stathmin regulates mutant p53 stability and transcriptional activity in ovarian cancer

      Vol. 5, Issue 5, 707–722, Version of Record online: 22 APR 2013

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