12 March 2014
Vol 6, Issue 227
  • Contents

    • Focus

      • Meeting the Demand for Pediatric Clinical Trials

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

    • Research Articles

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

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

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

        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.

      • Postexposure Protection of Macaques from Vaginal SHIV Infection by Topical Integrase Inhibitors

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

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

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

    • Editors' Choice

      • The Serpin Shield

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

      • Sifting for Diagnostic Gold

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

      • Got Milk Antibodies?

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

      • Are Two Heads Really Better than One?

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

About The Cover

Cover image expansion

ONLINE COVER Gene Therapy Moves Mountains. Shown are T cell receptor (TCR) length distributions before and after gene therapy in Wiskott-Aldrich Syndrome (WAS) patients. Most patients with a disturbed TCR profile before gene therapy improved after gene therapy, with a more polyclonal pattern of TCR Vβ usage. In this issue, Braun et al. report that hematopoietic stem cell gene therapy for WAS is feasible and effective, but the use of γ-retroviral vectors is associated with a substantial risk of leukemogenesis. [CREDIT: CHRISTIAN BRAUN/UNIVERSITY CHILDREN’S HOSPITAL, LUDWIG MAXIMILLIAN UNIVERISTY, MUNICH, GERMANY]