Characteristics and outcome of early-onset, severe forms of Wiskott-Aldrich syndrome

A randomized, double-blind study of romiplostim to determine its safety and efficacy in children with immune thrombocytopenia

(2011) J. B. Bussel et al.   BLOOD

Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study

(2011) D. Moratto et al.   BLOOD

WASp-deficient B cells play a critical, cell-intrinsic role in triggering autoimmunity

(2011) Shirly Becker-Herman et al.   JOURNAL OF EXPERIMENTAL MEDICINE

X-linked thrombocytopenia (XLT) due to WAS mutations: clinical characteristics, long-term outcome, and treatment options

(2010) M. H. Albert et al.   BLOOD

The French national registry of primary immunodeficiency diseases

(2010)   CLINICAL IMMUNOLOGY

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

(2010) Kaan Boztug et al.   NEW ENGLAND JOURNAL OF MEDICINE

The European internet-based patient and research database for primary immunodeficiencies: results 2006-2008

(2009) B. Gathmann et al.   CLINICAL AND EXPERIMENTAL IMMUNOLOGY

Progress and prospects: gene therapy for inherited immunodeficiencies

(2009) W Qasim et al.   GENE THERAPY

Development of lentiviral gene therapy for Wiskott Aldrich syndrome

(2008) Anne Galy et al.   EXPERT OPINION ON BIOLOGICAL THERAPY