EMBO Molecular Medicine

Cover image for Vol. 7 Issue 11

Early View (Online Version of Record published before inclusion in an issue)

Edited By: Stefanie Dimmeler (Chief Editor), Roberto Buccione and Céline Carret (EMBO Editors)

Online ISSN: 1757-4684


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  1. Research Articles

    1. You have full text access to this OnlineOpen article
      KLF4 is a key determinant in the development and progression of cerebral cavernous malformations

      Roberto Cuttano, Noemi Rudini, Luca Bravi, Monica Corada, Costanza Giampietro, Eleanna Papa, Marco Francesco Morini, Luigi Maddaluno, Nicolas Baeyens, Ralf H Adams, Mukesh K Jain, Gary K Owens, Martin Schwartz, Maria Grazia Lampugnani and Elisabetta Dejana

      Article first published online: 26 NOV 2015 | DOI: 10.15252/emmm.201505433

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      Current therapy for cerebral cavernous malformation (CCM) therapy is limited to neurosurgery. Transcription factor KLF4 is found to be a crucial determinant for the development of cavernomas and thus a future therapeutic target.

  2. Reviews

    1. You have full text access to this OnlineOpen article
      Mitochondrial disease in adults: what's old and what's new?

      Patrick F Chinnery

      Article first published online: 26 NOV 2015 | DOI: 10.15252/emmm.201505079

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      A state-of-the art, comprehensive overview on adult mitochondrial disorders including discussion of current directions for therapy and patient priorities for treatment. A must-read for basic and clinical researchers alike.

  3. Research Articles

    1. You have full text access to this OnlineOpen article
      Accumulated common variants in the broader fragile X gene family modulate autistic phenotypes

      Beata Stepniak, Anne Kästner, Giulia Poggi, Marina Mitjans, Martin Begemann, Annette Hartmann, Sandra Van der Auwera, Farahnaz Sananbenesi, Dilja Krueger-Burg, Gabriela Matuszko, Cornelia Brosi, Georg Homuth, Henry Völzke, Fritz Benseler, Claudia Bagni, Utz Fischer, Alexander Dityatev, Hans-Jörgen Grabe, Dan Rujescu, Andre Fischer and Hannelore Ehrenreich

      Article first published online: 26 NOV 2015 | DOI: 10.15252/emmm.201505696

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      Based on a novel approach, phenotype-based genetic association study, first evidence is provided that a particular constellation of completely normal genotypes in the “broader fragile X gene family” contributes to autistic phenotypes.

    2. You have full text access to this OnlineOpen article
      Cholesterol-loaded nanoparticles ameliorate synaptic and cognitive function in Huntington's disease mice

      Marta Valenza, Jane Y Chen, Eleonora Di Paolo, Barbara Ruozi, Daniela Belletti, Costanza Ferrari Bardile, Valerio Leoni, Claudio Caccia, Elisa Brilli, Stefano Di Donato, Marina M Boido, Alessandro Vercelli, Maria A Vandelli, Flavio Forni, Carlos Cepeda, Michael S Levine, Giovanni Tosi and Elena Cattaneo

      Article first published online: 20 NOV 2015 | DOI: 10.15252/emmm.201505413

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      Cholesterol in brain is largely derived by local synthesis. One affected pathway in Huntington's disease (HD) implicates that reduced production and/or availability of brain cholesterol may be detrimental for neuronal function.

    3. You have full text access to this OnlineOpen article
      Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy

      Giulio Cossu, Stefano C Previtali, Sara Napolitano, Maria Pia Cicalese, Francesco Saverio Tedesco, Francesca Nicastro, Maddalena Noviello, Urmas Roostalu, Maria Grazia Natali Sora, Marina Scarlato, Maurizio De Pellegrin, Claudia Godi, Serena Giuliani, Francesca Ciotti, Rossana Tonlorenzi, Isabella Lorenzetti, Cristina Rivellini, Sara Benedetti, Roberto Gatti, Sarah Marktel, Benedetta Mazzi, Andrea Tettamanti, Martina Ragazzi, Maria Adele Imro, Giuseppina Marano, Alessandro Ambrosi, Rossana Fiori, Maria Pia Sormani, Chiara Bonini, Massimo Venturini, Letterio S Politi, Yvan Torrente and Fabio Ciceri

      Article first published online: 5 NOV 2015 | DOI: 10.15252/emmm.201505636

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      This study reports a safe, first-in-human mesoangioblast cell therapy to treat Duchenne muscular dystrophy (DMD) in 5 young patients, using a successful preclinical strategy, as an exploratory non-randomized open-label phase I-IIa clinical trial of intra-arterial HLA-matched donor cell transplantation.

    4. You have full text access to this OnlineOpen article
      Aberrant epigenome in iPSC-derived dopaminergic neurons from Parkinson's disease patients

      Rubén Fernández-Santiago, Iria Carballo-Carbajal, Giancarlo Castellano, Roger Torrent, Yvonne Richaud, Adriana Sánchez-Danés, Roser Vilarrasa-Blasi, Alex Sánchez-Pla, José Luis Mosquera, Jordi Soriano, José López-Barneo, Josep M Canals, Jordi Alberch, Ángel Raya, Miquel Vila, Antonella Consiglio, José I Martín-Subero, Mario Ezquerra and Eduardo Tolosa

      Article first published online: 29 OCT 2015 | DOI: 10.15252/emmm.201505439

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      This is the first proof-of-principle that induced pluripotent stem cell (iPSC)-derived dopaminergic neurons (DAn) from sporadic and monogenetic Parkinson's disease (PD) patients show the same epigenomic changes as compared to healthy controls.


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