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Journal Articles

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

Bernhard Gentner, MD, Maria Ester Bernardo, MD PhD, Francesca Tucci, MD, Erika Zonari, PhD, Francesca Fumagalli, MD, Silvia Pontesilli, MD, Serena Acquati, PhD, Paolo Silvani, MD, Fabio Ciceri, MD, Attilio Rovelli, MD, Giancarlo La Marca, PhD, Rossella Parini, MD, Silvia Gregori, PhD, Eugenio Montini, PhD, Luigi Naldini, MD PhD, Alessandro Aiuti, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 607.
https://doi.org/10.1182/blood-2019-128805
Published: 2019
Journal Articles

Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study

Mieke Aldenhoven, Robert F. Wynn, Paul J. Orchard, Anne O’Meara, Paul Veys, Alain Fischer, Vassili Valayannopoulos, Benedicte Neven, Attilio Rovelli, Vinod K. Prasad, Jakub Tolar, Heather Allewelt, Simon A. Jones, Rossella Parini, Marleen Renard, Victoria Bordon, Nico M. Wulffraat, Tom J. de Koning, Elsa G. Shapiro, Joanne Kurtzberg, Jaap Jan Boelens
Journal: Blood
Blood (2015) 125 (13): 2164–2172.
https://doi.org/10.1182/blood-2014-11-608075
Published: 2015
Includes: Supplemental data