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congenital neutropenia
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Journal Articles

PML-controlled responses in severe congenital neutropenia with ELANE -misfolding mutations

Clinical Trials & Observations
Patricia A. Olofsen, Dennis A. Bosch, Onno Roovers, Paulina M. H. van Strien, Hans W. J. de Looper, Remco M. Hoogenboezem, Sander Barnhoorn, Pier G. Mastroberardino, Mehrnaz Ghazvini, Vincent H. J. van der Velden, Eric M. J. Bindels, Emma M. de Pater, Ivo P. Touw
Journal: Blood Advances
Blood Adv (2021) 5 (3): 775–786.
https://doi.org/10.1182/bloodadvances.2020003214
Published: 2021
Includes: Supplemental data
Journal Articles

Gene correction of HAX1 reversed Kostmann disease phenotype in patient-specific induced pluripotent stem cells

Erik Pittermann, Nico Lachmann, Glenn MacLean, Stephan Emmrich, Mania Ackermann, Gudrun Göhring, Brigitte Schlegelberger, Karl Welte, Axel Schambach, Dirk Heckl, Stuart H. Orkin, Tobias Cantz, Jan-Henning Klusmann
Journal: Blood Advances
Blood Adv (2017) 1 (14): 903–914.
https://doi.org/10.1182/bloodadvances.2016003798
Published: 2017
Includes: Supplemental data