Current understanding of the Wiskott-Aldrich syndrome and prospects for gene therapy.
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.
Trifari
Sara
S
San Raffaele-Telethon Institute for Gene Therapy (HSR-TIGET), Via Olgettina 58, 20132 Milan, Italy. s.trifari@hsr.it
Marangoni
Francesco
F
Scaramuzza
Samantha
S
Aiuti
Alessandro
A
Roncarolo
Maria Grazia
MG
Dupré
Loïc
L