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EMBO Molecular Medicine

Cover image for Vol. 5 Issue 11

November 2013

Volume 5, Issue 11

Pages 1637–1793, i–ii

  1. Perspective

    1. Top of page
    2. Perspective
    3. Reviews
    4. Research Articles
    5. Masthead
    1. You have full text access to this OnlineOpen article
  2. Reviews

    1. Top of page
    2. Perspective
    3. Reviews
    4. Research Articles
    5. Masthead
    1. You have full text access to this OnlineOpen article
      Gene therapy on the move (pages 1642–1661)

      Kerstin B. Kaufmann, Hildegard Büning, Anne Galy, Axel Schambach and Manuel Grez

      Article first published online: 17 SEP 2013 | DOI: 10.1002/emmm.201202287

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      A timely review on the application of gene therapy for the correction of inherited diseases, including a description of the drawbacks from the early clinical trials, how the inherent risks are being successfully addressed and future perspectives.

  3. Research Articles

    1. Top of page
    2. Perspective
    3. Reviews
    4. Research Articles
    5. Masthead
    1. You have full text access to this OnlineOpen article
      p53's choice of myocardial death or survival: Oxygen protects infarct myocardium by recruiting p53 on NOS3 promoter through regulation of p53-Lys118 acetylation (pages 1662–1683)

      Rajan Gogna, Esha Madan, Mahmood Khan, Uttam Pati and Periannan Kuppusamy

      Article first published online: 1 OCT 2013 | DOI: 10.1002/emmm.201202055

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      Oxygen therapy of infarcted hearts is shown to lead to increased survival of cardiomyocytes, inhibition of p53-dependent apoptosis mediated by p53-Lys118 and high expression of NOS3 via p53-dependent transcription and improved cardiac function.

    2. You have full text access to this OnlineOpen article
      Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice (pages 1684–1697)

      Andrea Annoni, Alessio Cantore, Patrizia Della Valle, Kevin Goudy, Mahzad Akbarpour, Fabio Russo, Sara Bartolaccini, Armando D'Angelo, Maria Grazia Roncarolo and Luigi Naldini

      Article first published online: 16 SEP 2013 | DOI: 10.1002/emmm.201302857

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      Liver-directed gene therapy can reverse pre-existing anti-factor IX humoral immunity in hemophilia B mice, eradicate factor IX inhibitors, stably reconstitute factor IX expression and restore hemostasis to near-normal levels.

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      Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies (pages 1698–1709)

      David M. Markusic, Brad E. Hoffman, George Q. Perrin, Sushrusha Nayak, Xiaomei Wang, Paul A. LoDuca, Katherine A. High and Roland W. Herzog

      Article first published online: 16 SEP 2013 | DOI: 10.1002/emmm.201302859

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      Hepatic adeno-associated viral factor IX gene transfer rapidly reversed pre-existing high-titer inhibitors in hemophilia B mice, eliminated antibody production by B cells, desensitized from anaphylaxis and provided long-term correction.

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      Rescue of amyotrophic lateral sclerosis phenotype in a mouse model by intravenous AAV9-ADAR2 delivery to motor neurons (pages 1710–1719)

      Takenari Yamashita, Hui Lin Chai, Sayaka Teramoto, Shoji Tsuji, Kuniko Shimazaki, Shin-ichi Muramatsu and Shin Kwak

      Article first published online: 24 SEP 2013 | DOI: 10.1002/emmm.201302935

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      A single intravenous injection of AAV9-ADAR2 in a mechanistic mouse model of sporadic ALS prevents progressive motor neurons dysfunction and death by enhancing ADAR2 enzymatic activity and expression of GluA2R instead of GluA2Q, marker of disease.

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      Activin receptor-like kinase5 inhibition suppresses mouse melanoma by ubiquitin degradation of Smad4, thereby derepressing eomesodermin in cytotoxic T lymphocytes (pages 1720–1739)

      Jeong-Hwan Yoon, Su Myung Jung, Seok Hee Park, Mitsuyasu Kato, Tadashi Yamashita, In-Kyu Lee, Katsuko Sudo, Susumu Nakae, Jin Soo Han, Ok-Hee Kim, Byung-Chul Oh, Takayuki Sumida, Masahiko Kuroda, Ji-Hyeon Ju, Kyeong Cheon Jung, Seong Hoe Park, Dae-Kee Kim and Mizuko Mamura

      Article first published online: 11 OCT 2013 | DOI: 10.1002/emmm.201302524

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      A novel orally bioavailable ALK5 inhibitor is shown to increase antimelanoma CTL-response by inducing Smad4 degradation and Eomes derepression in CD8+ T cells, in addition to systemic inhibition of RSmad phosphorylation by TGF-β type I receptor.

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      Genome-wide identification and functional analyses of microRNA signatures associated with cancer pain (pages 1740–1758)

      Kiran Kumar Bali, Deepitha Selvaraj, Venkata P. Satagopam, Jianning Lu, Reinhard Schneider and Rohini Kuner

      Article first published online: 18 OCT 2013 | DOI: 10.1002/emmm.201302797

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      The reversal of cancer-mediated overexpression of miR-1a-3p or miR-34c-5p or downregulation of miR-483-3p in sensory neurons reduces tumor-associated pain. Furthermore, Clcn3 is a target of miR- 1a-3p and a novel pain modulator.

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      Inhibition of p38 MAPK sensitizes tumour cells to cisplatin-induced apoptosis mediated by reactive oxygen species and JNK (pages 1759–1774)

      Lorena Pereira, Ana Igea, Begoña Canovas, Ignacio Dolado and Angel R. Nebreda

      Article first published online: 24 SEP 2013 | DOI: 10.1002/emmm.201302732

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      p38alpha/beta inhibition is shown here to sensitize tumor cells to cisplatin-induced apoptosis by increasing ROS levels and activating the JNK pathway, providing an interesting therapeutic strategy of combining p38 inhibitors to anti-cancer drugs.

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      CCR2+ monocytes infiltrate atrophic lesions in age-related macular disease and mediate photoreceptor degeneration in experimental subretinal inflammation in Cx3cr1 deficient mice (pages 1775–1793)

      Florian Sennlaub, Constance Auvynet, Bertrand Calippe, Sophie Lavalette, Lucie Poupel, Shulong J. Hu, Elisa Dominguez, Serge Camelo, Olivier Levy, Elodie Guyon, Noah Saederup, Israel F. Charo, Nico Van Rooijen, Emeline Nandrot, Jean-Louis Bourges, Francine Behar-Cohen, José-Alain Sahel, Xavier Guillonneau, William Raoul and Christophe Combadiere

      Article first published online: 21 OCT 2013 | DOI: 10.1002/emmm.201302692

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      The eyes of patients with atrophic AMD feature high CCL2 and CCR2+ monocytes. This is modeled in Cx3cr1 KO mice in which Ccl2 and Ccr2 deletion, CCR2 inhibition and monocyte depletion diminished subretinal inflammation and photoreceptor degeneration.

  4. Masthead

    1. Top of page
    2. Perspective
    3. Reviews
    4. Research Articles
    5. Masthead
    1. You have full text access to this OnlineOpen article
      Masthead (EMBO Mol. Med. 11/2013) (pages i–ii)

      Article first published online: 4 NOV 2013 | DOI: 10.1002/emmm.201370111

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