title: Current understanding of the Wiskott-Aldrich syndrome and prospects for gene therapy.
abstract: Gene therapy, based on the transplantation of genetically corrected autologous hematopoietic stem cells (HSCs), has proven to be an effective therapeutic approach as an alternative to allogenic HSC transplantation for the cure of severe combined immunodeficiencies (SCID). In this article, the recent preclinical studies aiming towards gene therapy trials for the Wiskott-Aldrich syndrome (WAS), a life-threatening immunodeficiency characterized by infections, hemorrhages, autoimmune disorders and lymphomas, will be reviewed. An update of the safety and efficacy data obtained in studies performed in murine disease models and in cells from WAS patients will be presented. Based on these data and on the clinical results of the recent trials for SCID, the most critical issues regarding the implementation of a gene therapy approach for WAS will be discussed.
authors.affiliation: San Raffaele-Telethon Institute for Gene Therapy (HSR-TIGET), Via Olgettina 58, 20132 Milan, Italy. s.trifari@hsr.it
authors.forename: Sara
authors.initials: S
authors.lastname: Trifari
authors.forename: Francesco
authors.initials: F
authors.lastname: Marangoni
authors.forename: Samantha
authors.initials: S
authors.lastname: Scaramuzza
authors.forename: Alessandro
authors.initials: A
authors.lastname: Aiuti
authors.forename: Maria Grazia
authors.initials: MG
authors.lastname: Roncarolo
authors.forename: Loïc
authors.initials: L
authors.lastname: Dupré