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Corrigendum to STAT1 promotes megakaryopoiesis downstream of GATA-1 in mice
Corrigendum Open Access | 10.1172/JCI195974 Published in Volume 135, Issue 14 on July 15, 2025 J Clin Invest. 2025;135(14):e195974. https://doi.org/10.1172/JCI195974. © 2025 Huang et al. This work is licensed under the Creative Commons Attribution 4.0 International License. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/.
Lentivirus-mediated gene therapy for Fabry disease
Abstract Enzyme and chaperone therapies are used to treat Fabry disease. Such treatments are expensive and require intrusive biweekly infusions; they are also not particularly efficacious. In this pilot, single-arm study (NCT02800070), five adult males with Type 1 (classical) phenotype Fabry disease were infused with autologous lentivirus-transduced, CD34+-selected, hematopoietic stem/progenitor cells engineered to express alpha-galactosidase A (α-gal A).
STAT1 promotes megakaryopoiesis downstream of GATA-1 in mice
Acknowledgments We thank for Ross Levine and Gary Gilliland for the JAK2 V617F and c-MPL W515L constructs and Sandeep Gurbuxani and members of the Crispino lab for helpful discussions. This study is supported by a grant from the NIH (CA101774) and the Canadian Institute of Health Research (D.L. Barber). J.D. Crispino is a scholar of the Leukemia and Lymphoma Society. Address correspondence to: John D.
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