P1392: INTERIM ANALYSIS OF FIRST IN HUMAN PHASE I-II CLINICAL TRIAL OF EX-VIVO GENE THERAPY FOR HURLER SYNDROME: AN UPDATE AT 3 YEAR FOLLOW-UP

• Francesca Tucci,
• Giulia Consiglieri,
• Chiara Filisetti,
• Maurizio De Pellegrin,
• Renata Mellone,
• Francesca Fumagalli,
• Silvia Darin,
• Marina Sarzana,
• Stefano Scarparo,
• Francesca Ciotti,
• Paolo Silvani,
• Cristina Baldoli,
• Silvia Pontesilli,
• Rossella Parini,
• Giancarlo la Marca,
• Fabio Ciceri,
• Luigi Naldini,
• Alessandro Aiuti,
• Bernhard Gentner,
• Maria Ester Bernardo

Affiliations

Francesca Tucci

1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute Milan, Milano, Italy

Giulia Consiglieri

1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute Milan, Milano, Italy

Chiara Filisetti

1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute Milan, Milano, Italy

Maurizio De Pellegrin

1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute Milan, Milano, Italy

Renata Mellone

2 IRCCS San Raffaele Scientific Institute Milan, Radiology Unit, Milano, Italy

Francesca Fumagalli

1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute Milan, Milano, Italy

Silvia Darin

1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute Milan, Milano, Italy

Marina Sarzana

1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute Milan, Milano, Italy

Stefano Scarparo

1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute Milan, Milano, Italy

Francesca Ciotti

1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute Milan, Milano, Italy

Paolo Silvani

3 IRCCS San Raffaele Scientific Institute Milan, Anesthesia and Critical Care, Milano, Italy

Cristina Baldoli

2 IRCCS San Raffaele Scientific Institute Milan, Radiology Unit, Milano, Italy

Silvia Pontesilli

2 IRCCS San Raffaele Scientific Institute Milan, Radiology Unit, Milano, Italy

Rossella Parini

1 San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute Milan, Milano, Italy

Giancarlo la Marca

4 Meyer Children’s University Hospital, Florence, Italy

Fabio Ciceri

5 IRCCS San Raffaele Scientific Institute, Hematology and Bone Marrow Transplantation, Milano, Italy

Luigi Naldini

7 IRCCS San Raffaele Scientific Institute Milan, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milano, Italy

Alessandro Aiuti

6 “Vita-Salute” San Raffaele University Milan, Italy

Bernhard Gentner

7 IRCCS San Raffaele Scientific Institute Milan, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milano, Italy

Maria Ester Bernardo

6 “Vita-Salute” San Raffaele University Milan, Italy