INVESTIGADORES
DEWEY Ricardo Alfredo
congresos y reuniones científicas
Título:
Gene therapy for Wiskott-Aldrich syndrome: preliminary results after treatment of the first patients.
Autor/es:
KAAN BOZTUG; INES AVEDILLO DÍEZ; RICARDO A. DEWEY; MANFRED SCHMIDT; CHRISTOPH VON KALLE; SONJA NAUNDORF; KLAUS KUEHLKE; CHRISTOPH KLEIN
Lugar:
Kartause Ittingen, Germany
Reunión:
Jornada; XXIV. Jahrestagung der Arbeitsgemeinschaft Pädiatrische Immunologie.; 2007
Institución organizadora:
Arbeitsgemeinschaft Pädiatrische Immunologie.
Resumen:
Wiskott-Aldrich syndrome is a complex primary immunodeficiency disorder
associated with microthrombocytopenia, eczema, autoimmune phenomena and
susceptibility to malignant lymphoma. Allogenic hematopoietic stem cell
transplantation (HSCT) represents a curative approach but remains problematic in
light of severe risks and side effects, in particular if no HLA-matched donor is
available. More recently, extensive preclinical studies conducted by us and others
have indicated that gene therapy may be a feasible and effective therapeutic option.
The first hematopoietic stem cell gene therapy trial for Wiskott-Aldrich syndrome has
been open for patient accrual since 2006. Two patients with classical, severe
Wiskott-Aldrich syndrome for whom an HLA-matched donor was unavailable have
been enrolled. Preliminary results on gene marking, efficacy, and functional
reconstitution suggest that transplantation of retrovirally modified hematopoietic stem
cells may be an alternative to conventional allogeneic bone marrow transplantation.
Long-term follow up will be necessary to assess sustained correction of the
hematopoietic system and to determine the risk-benefit ratio.