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Haematologica|September 18, 2012
The murine growth differentiation factor 15 is not essential for systemic iron homeostasis in phlebotomized miceGuillem Casanovas, Maja Vujić Spasic, Carla Casu, et al.Haematologica|May 23, 2020
2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring HbA production and chain rebalanceAlisa Dong, Valentina Ghiaccio, Irene Motta, et al.Journal of Blood Medicine|March 5, 2015
Recent trends in the gene therapy of β-thalassemiaAlessia Finotti, Laura Breda, Carsten W Lederer, et al.Cancer Research|March 26, 2014
Identification and characterization of small molecules that inhibit nonsense-mediated RNA decay and suppress nonsense p53 mutationsLeenus Martin, Arsen Grigoryan, Ding Wang, et al.Molecular Therapy. Nucleic Acids|April 2, 2025
Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrationsLucas Tricoli, Sunetra Sase, Julia L Hacker, et al.Blood Advances|September 6, 2023
Elevated CDKN1A (P21) mediates β-thalassemia erythroid apoptosis, but its loss does not improve β-thalassemic erythropoiesisRaymond Liang, Miao Lin, Vijay Menon, et al.International Journal of Molecular Sciences|January 27, 2021
CYP450 Mediates Reactive Oxygen Species Production in a Mouse Model of β-Thalassemia through an Increase in 20-HETE ActivityRayan Bou-Fakhredin, Batoul Dia, Hilda E Ghadieh, et al.Annals of the New York Academy of Sciences|August 18, 2010
Hepcidin and Hfe in iron overload in beta-thalassemiaSara Gardenghi, Pedro Ramos, Antonia Follenzi, et al.Kidney International|June 24, 2020
Carbonyl iron and iron dextran therapies cause adverse effects on bone health in juveniles with chronic kidney diseaseEdwin Patino, Stephen B Doty, Divya Bhatia, et al.Drug Discovery Today|September 4, 2022
Emergent treatments for β-thalassemia and orphan drug legislationsEnrico Costa, Maria Domenica Cappellini, Stefano Rivella, et al.Pageof 13