Contents

Contents

• Focus

  • Meeting the Demand for Pediatric Clinical Trials

    By Edward M. Connor, William E. Smoyer, Jonathan M. Davis, Anne Zajicek, Linda Ulrich, Mary Purucker, Steven Hirschfeld

    Science Translational Medicine12 Mar 2014 : 227fs11 Full AccessRestricted Access

    The bid for high-quality, cost-effective pediatric clinical trials requires robust research and regulatory playgrounds and an appropriately trained workforce.

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  • Maladapted Endothelial Cells Flip the Mesenchymal Switch

    By Daylon James, Shahin Rafii

    Science Translational Medicine12 Mar 2014 : 227fs12 Full AccessRestricted Access

    Endothelial cells in the neointima undergo a transition to the mesenchymal phenotype, suggesting a therapeutic target for vein graft restenosis (Cooley et al., this issue).

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• Research Articles

  • Gene Therapy for Wiskott-Aldrich Syndrome—Long-Term Efficacy and Genotoxicity

    By Christian Jörg Braun, Kaan Boztug, Anna Paruzynski, Maximilian Witzel, Adrian Schwarzer, Michael Rothe, Ute Modlich, Rita Beier, Gudrun Göhring, Doris Steinemann, Raffaele Fronza, Claudia Regina Ball, Reinhard Haemmerle, Sonja Naundorf, Klaus Kühlcke, Martina Rose, Chris Fraser, Liesl Mathias, Rudolf Ferrari, Miguel R. Abboud, Waleed Al-Herz, Irina Kondratenko, László Maródi, Hanno Glimm, Brigitte Schlegelberger, Axel Schambach, Michael Heinrich Albert, Manfred Schmidt, Christof von Kalle, Christoph Klein

    Science Translational Medicine12 Mar 2014 : 227ra33 Full AccessRestricted Access

    Wiskott-Aldrich syndrome gene therapy is feasible, but γ-retroviral vectors contribute a substantial risk of leukemogenesis.

    • Editor's Summary
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    • Supplementary Materials

  • TGF-β Signaling Mediates Endothelial-to-Mesenchymal Transition (EndMT) During Vein Graft Remodeling

    By Brian C. Cooley, Jose Nevado, Jason Mellad, Dan Yang, Cynthia St. Hilaire, Alejandra Negro, Fang Fang, Guibin Chen, Hong San, Avram D. Walts, Robin L. Schwartzbeck, Brandi Taylor, Jan D. Lanzer, Andrew Wragg, Abdalla Elagha, Leilani E. Beltran, Colin Berry, Robert Feil, Renu Virmani, Elena Ladich, Jason C. Kovacic, Manfred Boehm

    Science Translational Medicine12 Mar 2014 : 227ra34 Full AccessRestricted Access

    In vivo endothelial cell fate mapping demonstrates that TGF-β signaling is a central pathway regulating the endothelial-to-mesenchymal transition (EndMT) during vein graft remodeling.

    • Editor's Summary
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  • Postexposure Protection of Macaques from Vaginal SHIV Infection by Topical Integrase Inhibitors

    By Charles Dobard, Sunita Sharma, Urvi M. Parikh, Rolieria West, Andrew Taylor, Amy Martin, Chou-Pong Pau, Debra L. Hanson, Jonathan Lipscomb, James Smith, Francis Novembre, Daria Hazuda, J. Gerardo Garcia-Lerma, Walid Heneine

    Science Translational Medicine12 Mar 2014 : 227ra35 Full AccessRestricted Access

    Postexposure protection of macaques from SHIV infection by a gel containing integrase inhibitors.

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  • Intestinal Inhibition of the Na⁺/H⁺ Exchanger 3 Prevents Cardiorenal Damage in Rats and Inhibits Na⁺ Uptake in Humans

    By Andrew G. Spencer, Eric D. Labonte, David P. Rosenbaum, Craig F. Plato, Christopher W. Carreras, Michael R. Leadbetter, Kenji Kozuka, Jill Kohler, Samantha Koo-McCoy, Limin He, Noah Bell, Jocelyn Tabora, Kristin M. Joly, Marc Navre, Jeffrey W. Jacobs, Dominique Charmot

    Science Translational Medicine12 Mar 2014 : 227ra36 Full AccessRestricted Access

    An inhibitor of intestinal NHE3 reduces absorption of dietary sodium in rats and humans and prevents salt-induced cardiorenal injury in nephrectomized rats.

    • Editor's Summary
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• Editors' Choice

  • The Serpin Shield

    By Mercedes Balcells

    Science Translational Medicine12 Mar 2014 : 227ec43 Full AccessRestricted Access

    Serpins help breast and lung cancer cells metastasize to the brain by annulling stromal defenses and co-opting capillaries.

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  • Sifting for Diagnostic Gold

    By Edward Kai-Hua Chow

    Science Translational Medicine12 Mar 2014 : 227ec44 Full AccessRestricted Access

    Nanoporous silica thin films can enrich for low-abundance serum peptides in clinical samples.

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  • Got Milk Antibodies?

    By Mustafa Mahfuz

    Science Translational Medicine12 Mar 2014 : 227ec45 Full AccessRestricted Access

    Secreted immunoglobulins in breast milk may promote long-term benefits against intestinal inflammatory diseases.

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  • Are Two Heads Really Better than One?

    By Goutham Narla

    Science Translational Medicine12 Mar 2014 : 227ec46 Full AccessRestricted Access

    The PRKCI and SOX2 oncogenes are coamplified and cooperate to activate Hedgehog signaling in lung squamous cell carcinoma.

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  • New Biology Identified by Genetic Analysis of Nonalcoholic Fatty Liver Disease

    By Benjamin Neale

    Science Translational Medicine12 Mar 2014 : 227ec47 Full AccessRestricted Access

    Genetic variation in TM6SF2 predisposes to nonalcoholic fatty liver disease, potentially through impairment of VLDL secretion.

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