Alessandro Aiuti - To make an appointment - GSD

Biography

Professor Alessandro Aiuti is the Head of the Pediatric Immunohaematology Unit at IRCCS Ospedale San Raffaele in Milan and Professor of Pediatrics at the University Vita-Salute San Raffaele. In addition, he is deputy director of the Istituto San Raffaele Telethon for Gene Therapy SR-Tiget, where he is also the head of the Pathogenesis and Therapy of Primary Immunodeficiencies research unit of the same.

He is a graduate in Medicine and Surgery of the Sapienza University of Rome, where he obtains PhD in Molecular and Cellular Biology. In 1998, he obtains specialization in Hematology at the University of Milan.

His clinical and research activity is focused on pediatric hematology and immunology, with particular emphasis on primary immune deficiencies and other genetic diseases, and, in particular, their treatment through advanced gene and cell therapies. Among the most notable achievements, Professor Aiuti has conducted clinical trials of gene therapy for ADA-SCID, the first hematopoietic (or blood) stem cell gene therapy to be approved for marketing in the world.

Professor Aiuti is an author of more than 180 scientific articles (H-Index Scopus 48, Impact Factor 1.509) published on journals such as Science, New England Journal of Medicine, Nature Medicine, Journal of Clinical Investigation, Blood, EMBO Molecular Medicine and Science Translational Medicine. He is a reviewer for Nature, Blood, The Journal of Allergy and Clinical Immunology and other journals.

For his research activities, Dr. Aiuti was granted numerous awards throughout his career, such as the Award for an outstanding career and pioneering contributions to the field in 2005 from the European Society for Cell and Gene Therapies. He is a member of the Committee of Advanced Therapy (CAT) of the European Medicines Agency (EMA), Inborn Errors Working Party (IEWP) of the Studies Committee of the European Society for Blood and Marrow Transplantation (EBMT), member of Italian Working Group of Immunodeficiencies (IPINET) and ASGCT Hematologic and Immunologic Gene and Cell Therapy.

Education

La Sapienza University of Rome
Degree in Medicine and Surgery - 1990

La Sapienza University of Rome
Doctor’s Degree in Molecular and Cellular Biology - 1996

University of Milan
Specialization in Hematology - 1998

Multimedia gallery

The ADA-SCID gene therapy drug will be producted and distributed by Telethon

Publications

Professor Aiuti is an author of more than 180 scientific articles (H-Index Scopus 48, Impact Factor 1.509) published on journals such as Science, New England Journal of Medicine, Nature Medicine, Journal of Clinical Investigation, Blood, EMBO Molecular Medicine and Science Translational Medicine. He is a reviewer for Nature, Blood, The Journal of Allergy and Clinical Immunology and other journals

Latest publications

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia.

Nat Med (2019) 25:234-241.

Marktel S., Scaramuzza S., Cicalese M.P., Giglio F., Galimberti S., Lidonnici M.R., Calbi V., Assanelli A., Bernardo M.E., Rossi C., Calabria A., Milani R., Gattillo S., Benedicenti F., Spin

Lentiviral haematopoietic stem/progenitor cell gene therapy for the treatment of Wiskott-Aldrich syndrome: interim results of a non-randomized, open-label, phase 1/2 clinical study.

Lancet Hematology (2019) 6: e239-e253. Epub 2019 April 10.

Ferrua F., Cicalese M.P., Galimberti S., Giannelli S., Dionisio F., Barzaghi F., Migliavacca M., Bernardo M.E., Calbi V., Assanelli A.A., Facchini M., Fossati C., Albertazzi E., Scaramuzza S

Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial.

Lancet (2016) 388: 476-487. Epub June 8, 2016.

Sessa M, Lorioli L, Fumagalli F, Acquati S, Redaelli D, Baldoli C, Canale S, Lopez ID, Morena F, Calabria A, Fiori R, Silvani P, Rancoita PM, Gabaldo M, Benedicenti F, Antonioli G, Assanelli

Dynamics of hematopoietic stem/progenitor cells after autologous transplantation in humans.

Nat Med (2018) 24:1683-1690. Epub 2018 Oct 1

Scala S., Basso-Ricci L., Dionisio F., Pellin D., Giannelli S., Salerio F.A., Leonardelli L., Cicalese M.P., Ferrua F., Aiuti A., and Biasco L.

Lentiviral vector gene therapy protects XCGD mice from Acute Staphylococcus aureus pneumonia and inflammatory response.

Mol Ther. (2016) 24: 1873-1880.

Farinelli G., Hernandez RJ, Rossi A, Ranucci S, Sanvito F, Migliavacca M,Brombin C, Pramov A, Serio CD, Bovolenta C, Gentner B, Bragonzi A,and Aiuti A.

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency.

Blood (2016) 128: 45-54. Epub April 29, 2016.

Cicalese M.P., Ferrua F., Castagnaro L., Pajno R., Barzaghi F., Giannelli S., Dionisio F., Brigida I., Bonopane M., Casiraghi M., Tabucchi A., Carlucci F., Grunebaum E., Adeli M., Bredius R.G., Puck J.M., Stepensky P., Tezcan I., Rolfe K., De Boever E., Reinhardt R.R., Appleby J., Ciceri F., Roncarolo M.G., and Aiuti A.

In vivo tracking of human hematopoiesis reveals patterns of clonal dynamics during early and steady-state seconstitution phases.

Cell Stem Cell (2016) 19: 107-119. Epub May 25, 2016.

Biasco L., Pellin D., Scala S., Dionisio F., Basso-Ricci L.,Leonardelli L., Scaramuzza S., Baricordi C., Ferrua F., Cicalese M.P., Giannelli S., Neduva V., Dow D.J., Schmidt M., Von Kalle C.,Roncarolo M.G., Ciceri F., Vicard P., Wit E., Di Serio C., Naldini L., and Aiuti A.

In vivo tracking of T cells in humans unveils decade-long survival and activity of genetically modified T memory stem cells.

Sci Transl Med (2015) 7: 273ra13.

Biasco L., Scala S., Basso Ricci L., Dionisio F., Baricordi C., Calabria A., Giannelli S., Cieri N., Barzaghi F., Pajno R., Al-Mousa H., Scarselli A., Cancrini C., Bordignon C., Roncarolo M.G., Monti E., Bonini C., and Aiuti A.

Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis.

Mol Ther. (2014) 22: 1472-1483. Epub 2014 May 29.

Chiriaco M., Farinelli G., Capo V., Di Matteo G., Zonari E., Scaramuzza S., Sergi Sergi L., Migliavacca M.,Hernandez R.J., Bombelli F., Giorda E., Kajaste-Rudnitski A., Trono D., Grez M., Rossi P., Finocchi A., Naldini L., Gentner B., and Aiuti A.

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.

Science (2013) 341(6148):1233158. Epub 2013 Jul 11.

Biffi A., Montini E., Lorioli L., Cesani M., Fumagalli F., Plati T., Baldoli C., Martino S., Calabria A., Canale S., Benedicenti F., Vallanti G., Biasco L., Leo S., Kabbara N., Zanetti G., Rizzo W.B., Mehta N., Cicalese M.P., Casiraghi M., Boelens J.J., Del Carro U., Dow D.J., Schmidt M., Assanelli A., Neduva V., Di Serio C., Stupka E., Gardner J., Van Kalle C., Bordignon C., Ciceri F., Rovelli A.