Showing items related by title, author, creator and subject.

• Wiskott-Aldrich Syndrome in four male siblings from a consanguineous family from Lebanon 

  Mansour, Rana; El-Orfali, Youmna; Saber, Antoine; Noun, Dolly; Youssef, Nour J.; Youssef, Yolla; Hanna-Wakim, Rima H.; Dbaibo, Ghassan S.; Abboud, Miguel Raul; Massaad, Michel J. (Academic Press Inc., 2020)

  Background: Wiskott-Aldrich syndrome (WAS) is a rare X-linked primary immunodeficiency disorder (PID) characterized by microthrombocytopenia, bloody diarrhea, eczema, recurrent infections, and a high incidence of autoimmunity ...
• Gene therapy for Wiskott-Aldrich syndrome-long-term efficacy and genotoxicity 

  Braun, Christian Joerg; Boztug, Kaan; Paruzynski, Anna; Witzel, Maximilian Georg Wolfgang; Schwarzer, Adrian; Rothe, Michael; Modlich, Ute; Beier, Rita; Göhring, Gudrun; Steinemann, Doris; Fronza, Raffaele; Ball, Claudia Regina; Haemmerle, Reinhard; Naundorf, Sonja; Kühlcke, Klaus; Rose, Martina; Fraser, Chris J.; Mathias, Liesl; Ferrari, Rudolf; Abboud, Miguel Raul; Al-Herz, Waleed; Kondratenko, Irina Vadimovna; Máródi, László D.R.; Glimm, Hanno; Schlegelberger, Brigitte; Schambach, Axel; Albert, Michael Heinrich; Schmidt, Manfred C.; van Kalle, Christof; Klein, Christoph A. (American Association for the Advancement of Science, 2014)

  Wiskott-Aldrich syndrome (WAS) is characterized by microthrombocytopenia, immunodeficiency, autoimmunity, and susceptibility to malignancies. In our hematopoietic stem cell gene therapy (GT) trial using a γ-retroviral ...
• Altered regulation of cell migration in IRF6-mutated orofacial cleft patients-derived primary cells reveals a novel role of Rho GTPases in cleft/lip palate development 

  Ghassibe-Sabbagh, Michella; El Hajj, Joelle; Al Saneh, Mounir; El Baba, Nada; Abou Issa, Jamil; Al-Haddad, Maria; El Atat, Oula; Sabbagh, Joseph M.; Abou-Chebel, Naji; El-Sibai, Mirvat (Elsevier B.V., 2021)

  Orofacial clefts are the most common congenital craniofacial birth defects. They occur from a failure in cell proliferation and fusion of neural crest cells of the lip buds and/or palatal shelves. In this study, we investigate ...