Telethon Foundation

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

Alessandro Aiuti, Luca Biasco, Samantha Scaramuzza et al. · Science · 2013 · 1K citations · Full text

Cell Therapy, Lentiviral Gene Therapy, Immunology +17

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

Alessandro Aiuti, Federica Cattaneo, Stefania Galimberti et al. · New England Journal of Medicine · 2009 · 997 citations · Full text

Host response to EBV infection in X-linked lymphoproliferative disease results from mutations in an SH2-domain encoding gene

Alison J. Coffey, Robert A. Brooksbank, Oliver Brandau et al. · Nature Genetics · 1998 · 754 citations

Host Response, Sh2-domain Encoding Gene, Molecular Virology +8

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

Eugenio Montini, Daniela Cesana, Manfred Schmidt et al. · Journal of Clinical Investigation · 2009 · 571 citations · Full text

MYH9-Related Disease

Marco Seri, Alessandro Pecci, Filomena Di Bari et al. · Medicine · 2003 · 307 citations · Full text

Mild Macrothrombocytopenia, Autoimmune Disease, Mendelian Disorder +14