Exploitation of circulating CD34+ cells and non-genotoxic conditioning to overcome major limitations to treatment for autosomal recessive osteopetrosis

• Valentina Capo,
• Sara Penna,
• Ivan Merelli,
• Matteo Barcella,
• Serena Scala,
• Luca Basso-Ricci,
• Elena Draghici,
• Eleonora Palagano,
• Erika Zonari,
• Paolo Uva,
• Roberto Cusano,
• Alessandro Aiuti,
• Francesca Ficara,
• Cristina Sobacchi,
• Bernhard Gentner,
• Anna Villa

Affiliations

Valentina Capo

San Raffaele Telethon Institute for Gene Therapy, IRCSS San Raffaele Scientific Institute, Milan, Italy; Corresponding author.

Sara Penna

San Raffaele Telethon Institute for Gene Therapy, IRCSS San Raffaele Scientific Institute, Milan, Italy; DIMET, University of Milano-Bicocca, Monza, Italy

Ivan Merelli

Institute for Biomedical Technologies, National Research Council, Segrate, Italy

Matteo Barcella

San Raffaele Telethon Institute for Gene Therapy, IRCSS San Raffaele Scientific Institute, Milan, Italy

Serena Scala

San Raffaele Telethon Institute for Gene Therapy, IRCSS San Raffaele Scientific Institute, Milan, Italy

Luca Basso-Ricci

San Raffaele Telethon Institute for Gene Therapy, IRCSS San Raffaele Scientific Institute, Milan, Italy

Elena Draghici

San Raffaele Telethon Institute for Gene Therapy, IRCSS San Raffaele Scientific Institute, Milan, Italy

Eleonora Palagano

CNR-IRGB, Milan Unit, Milan, Italy; Humanitas Clinical and Research Center - IRCCS, Rozzano, Italy

Erika Zonari

San Raffaele Telethon Institute for Gene Therapy, IRCSS San Raffaele Scientific Institute, Milan, Italy

Paolo Uva

CRS4, Science and Technology Park Polaris, Pula, Italy

Roberto Cusano

CRS4, Science and Technology Park Polaris, Pula, Italy

Alessandro Aiuti

San Raffaele Telethon Institute for Gene Therapy, IRCSS San Raffaele Scientific Institute, Milan, Italy

Francesca Ficara

CNR-IRGB, Milan Unit, Milan, Italy; Humanitas Clinical and Research Center - IRCCS, Rozzano, Italy

Cristina Sobacchi

CNR-IRGB, Milan Unit, Milan, Italy; Humanitas Clinical and Research Center - IRCCS, Rozzano, Italy

Bernhard Gentner

San Raffaele Telethon Institute for Gene Therapy, IRCSS San Raffaele Scientific Institute, Milan, Italy

Anna Villa

San Raffaele Telethon Institute for Gene Therapy, IRCSS San Raffaele Scientific Institute, Milan, Italy; CNR-IRGB, Milan Unit, Milan, Italy