The Wiskott-Aldrich syndrome

Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome.

Nature medicine·2022

Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency.

Scientific reports·2018

Current progress on gene therapy for primary immunodeficiencies.

Gene therapy·2013

Critical variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency.

Gene therapy·2012

Lack of T-cell responses following autologous tumour lysate pulsed dendritic cell vaccination, in patients with relapsed osteosarcoma.

Clinical & translational oncology : official publication of the Federation of Spanish Oncology Societies and of the National Cancer Institute of Mexico·2012