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Gene correction of HAX1 reversed Kostmann disease phenotype in patient-specific induced pluripotent stem cells
Authors: Pittermann, E.; Lachmann, N.; MacLean, G.; Emmrich, S.; Ackermann, M.; Gohring, G.; Schlegelberger, B.; Welte, K.; Schambach, A.; Heckl, D.; Orkin, S. H.; Cantz, T.; Klusmann, J. H.
Date of Publication (YYYY-MM-DD): 2017-06-13
Title of Journal: Blood Adv
Volume: 1
Issue / Number: 14
Start Page: 903
End Page: 914
Document Type: Article
ID: 744114.0

MicroRNA-150-regulated vectors allow lymphocyte-sparing transgene expression in hematopoietic gene therapy
Authors: Lachmann, N.; Jagielska, J.; Heckl, D.; Brennig, S.; Pfaff, N.; Maetzig, T.; Modlich, U.; Cantz, T.; Gentner, B.; Schambach, A.; Moritz, T.
Date of Publication (YYYY-MM-DD): 2012-10-06
Title of Journal: Gene Ther
Volume: 19
Issue / Number: 9
Start Page: 915
End Page: 924
Document Type: Article
ID: 648349.0