Combination therapy with a T mprss6 RNA i‐therapeutic and the oral iron chelator deferiprone additively diminishes secondary iron overload in a mouse model of β‐thalassemia intermedia
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β-thalassemias result from diminished β-globin synthesis and are associated with ineffective erythropoiesis and secondary iron overload caused by inappropriately low levels of the iron regulatory hormone hepcidin. The serine protease TMPRSS6 attenuates hepcidin production in response to iron stores. Hepcidin induction reduces iron overload and mitigates anemia in murine models of β-thalassemia intermedia. To further interrogate the efficacy of an RNAi-therapeutic downregulating Tmprss6, β-thalassemic Hbb(th3/+) animals on an iron replete, an iron deficient, or an iron replete diet also containing the iron chelator deferiprone were treated with Tmprss6 siRNA. We demonstrate that the total body iron burden is markedly improved in Hbb(th3/+) animals treated with siRNA and chelated with oral deferiprone, representing a significant improvement compared to either compound alone. These data indicate that siRNA suppression of Tmprss6, in conjunction with oral iron chelation therapy, may prove superior for treatment of anemia and secondary iron loading seen in β-thalassemia intermedia.
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Combination therapy of Tmprss6 siRNA LNPs with Deferiprone further mitigated anemia and secondary iron overload in murine β-thalassemia intermedia.
“In 2015, they employed the combination therapy of the Tmprss6 siRNA LNPs with Deferiprone, an oral iron chelator, for further mitigating anemia and secondary iron overload in murine β-thalassemia intermedia.[70]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [70]
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Cites
Mutations in TMPRSS6 cause iron-refractory iron deficiency anemia (IRIDA)
Finberg, K. E., Heeney, M. M., Campagna, D. R., Aydınok, Y., Pearson, H. A., Hartman, K. R., Mayo, M. M., Samuel, S. M., Strouse, J. J., Markianos, K., et al. (2008) · Nature Genetics · doi:10.1038/ng.130 · Cited by 675 · see citing works
Self-citationSilvestri, L., Pagani, A., Nai, A., De Domenico, I., Kaplan, J., & Camaschella, C. (2008) · Cell Metabolism · doi:10.1016/j.cmet.2008.09.012 · Cited by 555 · see citing works
Hepcidin in iron overload disorders
Papanikolaou, G., Tzilianos, M., Christakis, J. I., Bogdanos, D., Tsimirika, K., MacFarlane, J., Goldberg, Y. P., Sakellaropoulos, N., Ganz, T., & Nemeth, E. (2005) · Blood · doi:10.1182/blood-2004-12-4844 · Cited by 445 · see citing works
Liver iron concentrations and urinary hepcidin in -thalassemia
Origa, R., Galanello, R., Ganz, T., Giagu, N., Maccioni, L., Faa, G., & Nemeth, E. (2007) · Haematologica · doi:10.3324/haematol.10842 · Cited by 365 · see citing works
Gardenghi, S., Marongiu, M. F., Ramos, P., Guy, E. C., Breda, L., Chadburn, A., Liu, Y.-F., Amariglio, N., Rechavi, G., Rachmilewitz, E. A., et al. (2007) · Blood · doi:10.1182/blood-2006-09-048868 · Cited by 307 · see citing works
A mouse model for beta 0-thalassemia.
Yang, B., Kirby, S. L., Lewis, J., Detloff, P. J., Maeda, N., & Smithies, O. (1995) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.92.25.11608 · Cited by 272 · see citing works
Reducing TMPRSS6 ameliorates hemochromatosis and β-thalassemia in mice
Guo, S., Casu, C., Gardenghi, S., Booten, S. L., Aghajan, M., Peralta, R. Q., Watt, A., Freier, S., Monia, B. P., & Rivella, S. (2013) · Journal of Clinical Investigation · doi:10.1172/jci66969 · Cited by 224 · see citing works
Hepcidin as a therapeutic tool to limit iron overload and improve anemia in β-thalassemic mice
Gardenghi, S., Ramos, P., Marongiu, M. F., Melchiori, L., Breda, L., Guy, E., Muirhead, K., Rao, N., Roy, C. N., Andrews, N. C., et al. (2010) · Journal of Clinical Investigation · doi:10.1172/jci41717 · Cited by 220 · see citing works
Schmidt, P. J., Toudjarska, I., Sendamarai, A. K., Racie, T., Milstein, S., Bettencourt, B. R., Hettinger, J., Bumcrot, D., & Fleming, M. D. (2013) · Blood · doi:10.1182/blood-2012-09-453977 · Cited by 204 · see citing works
Self-citationTransferrin therapy ameliorates disease in β-thalassemic mice
Li, H., Rybicki, A. C., Suzuka, S. M., von Bonsdorff, L., Breuer, W., Hall, C. B., Cabantchik, Z. I., Bouhassira, E. E., Fabry, M. E., & Ginzburg, Y. Z. (2010) · Nature Medicine · doi:10.1038/nm.2073 · Cited by 192 · see citing works
Deletion of TMPRSS6 attenuates the phenotype in a mouse model of β-thalassemia
Nai, A., Pagani, A., Mandelli, G., Lidonnici, M. R., Silvestri, L., Ferrari, G., & Camaschella, C. (2012) · Blood · doi:10.1182/blood-2012-01-401885 · Cited by 161 · see citing works
Pasricha, S., Frazer, D. M., Bowden, D. K., & Anderson, G. J. (2013) · Blood · doi:10.1182/blood-2012-12-471441 · Cited by 154 · see citing works
Decreased hepcidin mRNA expression in thalassemic mice
Adamsky, K., Weizer, O., Amariglio, N., Breda, L., Harmelin, A., Rivella, S., Rachmilewitz, E., & Rechavi, G. (2004) · British Journal of Haematology · doi:10.1046/j.1365-2141.2003.04734.x · Cited by 108 · cited 1× · see citing works
“Similar to their human counterparts, these animals have moderately severe, transfusion-independent hypochromic, microcytic anemia associated and ineffective erythropoeisis resulting in splenomegaly, hepcidin suppression, and secondary iron overload [4–7].”
BackgroundOral deferiprone for iron chelation in people with thalassaemia
Fisher, S. A., Brunskill, S. J., Doree, C. J., Chowdhury, O., Gooding, S., & Roberts, D. J. (2013) · Cochrane Database of Systematic Reviews · doi:10.1002/14651858.cd004839.pub3 · Cited by 88 · see citing works
Tmprss6 is a genetic modifier of the Hfe-hemochromatosis phenotype in mice
Finberg, K. E., Whittlesey, R. L., & Andrews, N. C. (2011) · Blood · doi:10.1182/blood-2010-10-315507 · Cited by 81 · see citing works
Weizer-Stern, O., Adamsky, K., Amariglio, N., Rachmilewitz, E., Breda, L., Rivella, S., & Rechavi, G. (2006) · American Journal of Hematology · doi:10.1002/ajh.20549 · Cited by 71 · see citing works
Liver expression of hepcidin and other iron genes in two mouse models of beta-thalassemia.
Franceschi, L. D., Daraio, F., Filippini, A., Carturan, S., Muchitsch, E. M., Roetto, A., & Camaschella, C. (2006) · PubMed · Cited by 57 · see citing works
Gutschow, P., Schmidt, P. J., Han, H., Ostland, V., Bartnikas, T. B., Pettiglio, M. A., Herrera, C., Butler, J. S., Nemeth, E., Ganz, T., et al. (2014) · Haematologica · doi:10.3324/haematol.2014.116723 · Cited by 31 · see citing works
Self-citation
Flagged first, then by citations
Cited by
Sorted by citations
Molecular basis of β thalassemia and potential therapeutic targets
Thein, S. L. (2017) · Blood Cells Molecules and Diseases · doi:10.1016/j.bcmd.2017.06.001 · Cited by 208 · see citing works
Ineffective erythropoiesis and regulation of iron status in iron loading anaemias
Camaschella, C., & Nai, A. (2016) · British Journal of Haematology · doi:10.1111/bjh.13820 · Cited by 172 · see citing works
Erythropoiesis: insights into pathophysiology and treatments in 2017
Zivot, A., Lipton, J. M. M., Narla, A., & Blanc, L. (2018) · Molecular Medicine · doi:10.1186/s10020-018-0011-z · Cited by 172 · see citing works
Minihepcidin peptides as disease modifiers in mice affected by β-thalassemia and polycythemia vera
Casu, C., Oikonomidou, P. R., Chen, H., Nandi, V., Ginzburg, Y., Prasad, P., Fleming, R. E., Shah, Y. M., Valore, E. V., Nemeth, E., et al. (2016) · Blood · doi:10.1182/blood-2015-10-676742 · Cited by 148 · see citing works
Hepcidin agonists as therapeutic tools
Casu, C., Nemeth, E., & Rivella, S. (2018) · Blood · doi:10.1182/blood-2017-11-737411 · Cited by 131 · see citing works
Liu, J. L., Sun, B., Yin, H.-J., & Liu, S. (2016) · Medicine · doi:10.1097/md.0000000000003150 · Cited by 138 · see citing works
Rivella, S. (2015) · Haematologica · doi:10.3324/haematol.2014.114827 · Cited by 122 · see citing works
Katsarou, A., & Pantopoulos, K. (2018) · Pharmaceuticals · doi:10.3390/ph11040127 · Cited by 115 · see citing works
Ineffective Erythropoiesis: Anemia and Iron Overload
Gupta, R., Musallam, K. M., Taher, A. T., & Rivella, S. (2017) · Hematology/Oncology Clinics of North America · doi:10.1016/j.hoc.2017.11.009 · Cited by 92 · see citing works
RNA delivery biomaterials for the treatment of genetic and rare diseases
Zhao, W., Hou, X., Vick, O. G., & Dong, Y. (2019) · Biomaterials · doi:10.1016/j.biomaterials.2019.119291 · Cited by 82 · see citing works
Casu, C., Aghajan, M., Oikonomidou, P. R., Guo, S., Monia, B. P., & Rivella, S. (2015) · Haematologica · doi:10.3324/haematol.2015.133348 · Cited by 80 · see citing works
Therapeutic potential of hepcidin − the master regulator of iron metabolism
Vyoral, D., & Petrák, J. (2016) · Pharmacological Research · doi:10.1016/j.phrs.2016.11.010 · Cited by 79 · see citing works
Hepcidin: Homeostasis and Diseases Related to Iron Metabolism
Reichert, C. O., da Cunha, J., Levy, D., Maselli, L. M. F., Bydlowski, S. P., & Spada, C. (2017) · Acta Haematologica · doi:10.1159/000471838 · Cited by 74 · see citing works
Decreasing TfR1 expression reverses anemia and hepcidin suppression in β-thalassemic mice
Li, H., Choesang, T., Bao, W., Chen, H., Feola, M., Garcia‐Santos, D., Li, J., Sun, S., Follenzi, A., Pham, P., et al. (2017) · Blood · doi:10.1182/blood-2016-09-742387 · Cited by 71 · see citing works
What can we learn from ineffective erythropoiesis in thalassemia?
Oikonomidou, P. R., & Rivella, S. (2017) · Blood Reviews · doi:10.1016/j.blre.2017.10.001 · Cited by 64 · see citing works
Vela, D. (2018) · Molecular Medicine · doi:10.1186/s10020-018-0008-7 · Cited by 63 · see citing works
Therapeutic Advances in Regulating the Hepcidin/Ferroportin Axis
Hawula, Z. J., Wallace, D. F., Subramaniam, V. N., & Rishi, G. (2019) · Pharmaceuticals · doi:10.3390/ph12040170 · Cited by 62 · see citing works
2021 update on clinical trials in β‐thalassemia
Musallam, K. M., Bou‐Fakhredin, R., Cappellini, M. D., & Taher, A. T. (2021) · American Journal of Hematology · doi:10.1002/ajh.26316 · Cited by 61 · see citing works
Iron, Copper, and Zinc Homeostasis: Physiology, Physiopathology, and Nanomediated Applications
Szabó, R., Bodolea, C., & Mocan, T. (2021) · Nanomaterials · doi:10.3390/nano11112958 · Cited by 59 · see citing works
Modulation of hepcidin to treat iron deregulation: potential clinical applications
Blanchette, N. L., Manz, D. H., Torti, F. M., & Torti, S. V. (2015) · Expert Review of Hematology · doi:10.1586/17474086.2016.1124757 · Cited by 58 · see citing works
Optimizing the diagnosis and the treatment of iron overload diseases
Brissot, P. (2015) · Expert Review of Gastroenterology & Hepatology · doi:10.1586/17474124.2016.1119043 · Cited by 55 · see citing works
Iron dysregulation in beta-thalassemia
Leecharoenkiat, K., Lithanatudom, P., Sornjai, W., & Smith, D. R. (2016) · Asian Pacific Journal of Tropical Medicine · doi:10.1016/j.apjtm.2016.07.035 · Cited by 51 · see citing works
Casu, C., Chessa, R., Liu, A., Gupta, R., Drakesmith, H., Fleming, R., Ginzburg, Y. Z., MacDonald, B., & Rivella, S. (2020) · Haematologica · doi:10.3324/haematol.2018.212589 · Cited by 50 · see citing works
Iron overload cardiomyopathy: Using the latest evidence to inform future applications
Kumfu, S., Chattipakorn, S. C., & Chattipakorn, N. (2022) · Experimental Biology and Medicine · doi:10.1177/15353702221076397 · Cited by 50 · see citing works
Czaja, A. J. (2019) · Alimentary Pharmacology & Therapeutics · doi:10.1111/apt.15173 · Cited by 47 · see citing works
Casu, C., Pettinato, M., Liu, A., Aghajan, M., Presti, V. L., Lidonnici, M. R., Munoz, K. A., O’Hara, E., Olivari, V., Modica, S. M. D., et al. (2020) · Blood · doi:10.1182/blood.2019004719 · Cited by 43 · see citing works
Garcia‐Santos, D., Hamdi, A., Saxova, Z., Fillebeen, C., Pantopoulos, K., Horváthová, M., & Ponka, P. (2017) · Blood · doi:10.1182/blood-2017-07-798728 · Cited by 40 · see citing works
Artuso, I., Lidonnici, M. R., Altamura, S., Mandelli, G., Pettinato, M., Muckenthaler, M. U., Silvestri, L., Ferrari, G., Camaschella, C., & Nai, A. (2018) · Blood · doi:10.1182/blood-2018-05-852277 · Cited by 38 · see citing works
Novel Therapeutic Advances in β-Thalassemia
Makis, A. C., Voskaridou, E., Papassotiriou, I., & Hatzimichael, E. C. (2021) · Biology · doi:10.3390/biology10060546 · Cited by 38 · see citing works
Iron as a therapeutic target in chronic liver disease
Kouroumalis, E., Tsomidis, I., & Voumvouraki, A. (2023) · World Journal of Gastroenterology · doi:10.3748/wjg.v29.i4.616 · Cited by 40 · see citing works
Retinal abnormalities in β-thalassemia major
Bhoiwala, D. L., & Dunaief, J. L. (2015) · Survey of Ophthalmology · doi:10.1016/j.survophthal.2015.08.005 · Cited by 37 · see citing works
Normal and dysregulated crosstalk between iron metabolism and erythropoiesis
Ginzburg, Y. Z., An, X., Rivella, S., & Goldfarb, A. N. (2023) · eLife · doi:10.7554/elife.90189 · Cited by 40 · see citing works
Vadolas, J., Ng, G. Z., Kysenius, K., Crouch, P. J., Dames, S., Eisermann, M., Nualkaew, T., Vilcassim, S., Schaeper, U., & Grigoriadis, G. (2021) · British Journal of Haematology · doi:10.1111/bjh.17428 · Cited by 32 · see citing works
Schmidt, P. J., Liu, K., Visner, G. A., Fitzgerald, K. J., Fishman, S., Racie, T., Hettinger, J. L., Butler, J. S., & Fleming, M. D. (2018) · American Journal of Hematology · doi:10.1002/ajh.25079 · Cited by 29 · see citing works
Self-citationPorter, J. B., Taher, A. T., Viprakasit, V., Kattamis, A., Coates, T. D., Garbowski, M., Dürrenberger, F., Manolova, V., Richard, F., & Cappellini, M. D. (2021) · Expert Review of Hematology · doi:10.1080/17474086.2021.1935854 · Cited by 28 · see citing works
Nyffenegger, N., Flace, A., Doucerain, C., Dürrenberger, F., & Manolova, V. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22020873 · Cited by 27 · see citing works
New strategies to target iron metabolism for the treatment of beta thalassemia
Oikonomidou, P. R., Casu, C., & Rivella, S. (2016) · Annals of the New York Academy of Sciences · doi:10.1111/nyas.13018 · Cited by 26 · see citing works
Beta‐thalassemia and the advent of new interventions beyond transfusion and iron chelation
Chauhan, W., Shoaib, S., Fatma, R., Zaka‐ur‐Rab, Z., & Afzal, M. (2022) · British Journal of Clinical Pharmacology · doi:10.1111/bcp.15343 · Cited by 25 · see citing works
Wang, C., Fang, Z., Zhu, Z., Liu, J., & Chen, H. (2017) · Experimental Hematology · doi:10.1016/j.exphem.2017.05.002 · Cited by 25 · see citing works
Investigational drugs in phase I and phase II clinical trials for thalassemia
Motta, I., Scaramellini, N., & Cappellini, M. D. (2017) · Expert Opinion on Investigational Drugs · doi:10.1080/13543784.2017.1335709 · Cited by 24 · see citing works
Hepcidin as a therapeutic target in iron overload
Sandnes, M., & Reikvam, H. (2024) · Expert Opinion on Therapeutic Targets · doi:10.1080/14728222.2024.2443081 · Cited by 22 · see citing works
Fang, Z., Zhu, Z., Zhang, H., Peng, Y., Liu, J., Lu, H., Jiang, L., Liang, L., Xia, S., Wang, Q., et al. (2019) · British Journal of Haematology · doi:10.1111/bjh.16156 · Cited by 22 · see citing works
Rana, S., & Prabhakar, N. (2021) · Clinica Chimica Acta · doi:10.1016/j.cca.2021.10.032 · Cited by 21 · see citing works
Saad, H. K. M., Rahman, A. A. A., Ghani, A. S. A., Taib, W. R. W., Ismail, I., Johan, M. F., Al-wajeeh, A. S., & Al‐Jamal, H. A. N. (2022) · Biomedicines · doi:10.3390/biomedicines10010189 · Cited by 22 · see citing works
An, W., Feola, M., Aluri, S., Ruíz-Martínez, M., Shridharan, A., Levy, M., Fibach, E., Zhu, X., Verma, A., & Ginzburg, Y. Z. (2023) · eLife · doi:10.7554/elife.83103 · Cited by 16 · see citing works
Duality of Nrf2 in iron-overload cardiomyopathy
Federti, E., Vinchi, F., Iatcenko, I., Ghigo, A., Mattè, A., Mbiandjeu, S. C. T., Siciliano, A., Chiabrando, D., Tolosano, E., Vance, S. Z., et al. (2023) · Haematologica · doi:10.3324/haematol.2022.281995 · Cited by 13 · see citing works
Matriptase-2: Monitoring and Inhibiting Its Proteolytic Activity
Gitlin‐Domagalska, A., Mangold, M., Dębowski, D., Ptaszyńska, N., Łęgowska, A., Gütschow, M., & Rolka, K. (2018) · Future Medicinal Chemistry · doi:10.4155/fmc-2018-0346 · Cited by 13 · see citing works
Roydeva, P., Beckmann, A.-M., Stirnberg, M., Cesar, J., Kikelj, D., Ilaš, J., & Gütschow, M. (2016) · Pharmaceuticals · doi:10.3390/ph9010002 · Cited by 12 · see citing works
Current Landscape of Hepcidin Therapeutics
Nai, A., Silvestri, L., Asperti, M., & Vinchi, F. (2025) · Advances in experimental medicine and biology · doi:10.1007/978-3-031-92033-2_26 · Cited by 11 · see citing works
Çam, H., & Yılmaz, N. (2020) · Arab Journal of Gastroenterology · doi:10.1016/j.ajg.2020.04.013 · Cited by 10 · see citing works
Ducamp, S., Sendamarai, A. K., Campagna, D. R., Chin, D. W. L., Fujiwara, Y., Schmidt, P. J., & Fleming, M. D. (2024) · Blood · doi:10.1182/blood.2023023078 · Cited by 10 · see citing works
Self-citationWake, M., Palin, A., Belot, A., Berger, M., Lorgouilloux, M., Bichon, M., Papworth, J., Bayliss, L., Grimshaw, B., Rynkiewicz, N. K., et al. (2024) · Blood Advances · doi:10.1182/bloodadvances.2023012010 · Cited by 9 · see citing works
Guerra, A., Hamilton, N., Rivera, A., Demsko, P., Guo, S., & Rivella, S. (2024) · American Journal of Hematology · doi:10.1002/ajh.27332 · Cited by 9 · see citing works
Generation of an in vitro model of β‐thalassemia using the CRISPR/Cas9 genome editing system
Ajami, M., Atashi, A., Kaviani, S., Kiani, J., & Soleimani, M. (2019) · Journal of Cellular Biochemistry · doi:10.1002/jcb.29377 · Cited by 9 · see citing works
Emerging Approaches for Enabling RNAi Therapeutics
Mallick, A. M., Tripathi, A., Mishra, S., Mukherjee, A., Dutta, C., Chatterjee, A., & Roy, R. S. (2022) · Chemistry - An Asian Journal · doi:10.1002/asia.202200451 · Cited by 8 · see citing works
Schmidt, P. J., Hollowell, M. L., Fitzgerald, K. J., Butler, J. S., & Fleming, M. D. (2020) · American Journal of Hematology · doi:10.1002/ajh.25743 · Cited by 8 · see citing works
Self-citationChisté, R. C., Ribeiro, D., Freitas, M., Leite, A., Moniz, T., Rangel, M., & Fernandes, E. G. R. (2015) · Journal of Inorganic Biochemistry · doi:10.1016/j.jinorgbio.2015.11.009 · Cited by 7 · see citing works
Does Hepcidin Tuning Have a Role among Emerging Treatments for Thalassemia?
Longo, F., & Piga, A. G. (2022) · Journal of Clinical Medicine · doi:10.3390/jcm11175119 · Cited by 7 · see citing works
Cellular and animal models for the investigation of β-thalassemia
Nai, A., Cordero-Sánchez, C., Tanzi, E., Pagani, A., Silvestri, L., & Modica, S. M. D. (2023) · Blood Cells Molecules and Diseases · doi:10.1016/j.bcmd.2023.102761 · Cited by 6 · see citing works
Schmidt, P. J., Fitzgerald, K. J., Butler, J. S., & Fleming, M. D. (2020) · American Journal of Hematology · doi:10.1002/ajh.26048 · Cited by 6 · see citing works
Self-citationСаприна, Т. В., Zima, A. P., Мусина, Н. Н., Prokhorenko, T. S., Latypova, A. V., Shakhmanova, N. S., & Budeeva, S. V. (2019) · Diabetes Mellitus · doi:10.14341/dm9378 · Cited by 5 · see citing works
Functionally impaired isoforms regulate TMPRSS6 proteolytic activity
Dion, S. P., Désilets, A., Lemieux, G., & Leduc, R. (2022) · PLoS ONE · doi:10.1371/journal.pone.0273825 · Cited by 5 · see citing works
RNAi-based Gene Therapy for Blood Genetic Diseases
Hu, M., Ni, Q., Yang, Y., & Luo, J. (2016) · InTech eBooks · doi:10.5772/61644 · Cited by 4 · see citing works
Reichert, C. O., da Cunha, J., Levy, D., Maselli, L. M. F., Bydlowski, S. P., & Spada, C. (2017) · InTech eBooks · doi:10.5772/intechopen.69507 · Cited by 4 · see citing works
Iron Deficiency, Iron Overload and Therapy
Crichton, R. R. (2016) · Wiley eBooks · doi:10.1002/9781118925645.ch11 · Cited by 4 · see citing works
Karwasra, R., Singh, S., Raza, K., Sharma, N., & Varma, S. (2020) · Journal of Drug Delivery Science and Technology · doi:10.1016/j.jddst.2020.102159 · Cited by 3 · see citing works
Zhu, R., Zhang, J., Zhang, Q., Dong, W., Meng, Y., Wang, Y., Wang, X., & Han, S. (2024) · Nurse Education in Practice · doi:10.1016/j.nepr.2024.103974 · Cited by 2 · see citing works
Casu, C., Aghajan, M., Oikonomidau, R., Guo, S., Monia, B. P., & Rivella, S. (2014) · Blood · doi:10.1182/blood.v124.21.4024.4024 · Cited by 2 · see citing works
Cutting not the key to TMPRSS6 activity?
Bartnikas, T. B. (2020) · Blood · doi:10.1182/blood.2020006608 · Cited by 2 · see citing works
Levy, M., & Ginzburg, Y. Z. (2025) · Advances in experimental medicine and biology · doi:10.1007/978-3-031-92033-2_11 · Cited by 1 · see citing works
[Hepcidin for iron homeostasis and target therapy in ironrelated disorders].
Fan, S., Zheng, Y., & Shi, J. (2015) · PubMed Central · doi:10.3760/cma.j.issn.0253-2727.2015.11.020 · Cited by 1 · see citing works
Huang, L., & Fu, R. (2019) · PubMed Central · doi:10.3760/cma.j.issn.0253-2727.2019.08.021 · Cited by 1 · see citing works
Extracellular: Plasma Membrane Proteases – Serine Proteases
Antalis, T., Pawar, N. R., & Buzza, M. S. (2022) · Elsevier eBooks · doi:10.1016/b978-0-12-821618-7.00036-5 · Cited by 1 · see citing works
Loss of UBE2O Mitigates Beta Thalassemia Through Broad Proteomic Changes
Nguyen, A. T. (2018) · Digital Access to Scholarship at Harvard (DASH) (Harvard University)
Identification and analysis of genetic and chemical modulators of iron metabolism
Hawula, Z. J. (2021) · Queensland University of Technology · doi:10.5204/thesis.eprints.225904
Crichton, R. R. (2016) · doi:10.1002/9781118925645.fmatter
2026 Update on Clinical Trials in β‐Thalassemia
Musallam, K. M., Locatelli, F., Algeri, M., Cappellini, M. D., & Taher, A. T. (2026) · American Journal of Hematology · doi:10.1002/ajh.70472
- Authors
- Schmidt, P. J., Racie, T., Westerman, M., Fitzgerald, K., Butler, J. S., & Fleming, M. D.
- Affiliations
- Boston Children's Hospital (US)
- Alnylam Pharmaceuticals (United States) (US)
- Intrinsic LifeSciences (United States) (US)
- Corresponding author
- Schmidt, Paul J.
- Venue
- American Journal of Hematology
- Publisher
- Wiley
- Published
- 2015-03-23
- Volume
- 90
- Issue
- 4
- Pages
- 310-313
- ISSN
- 0361-8609 (ISSN-L)
- 1096-8652
- PMC
- PMC4403964
- OpenAlex
- W1832707526
- Cited by
- 77
- References
- 18
- Citations by year
- 2026: 1 · 2025: 2 · 2024: 5 · 2023: 5 · 2022: 7 · 2021: 8 · 2020: 6 · 2019: 8 · 2018: 8 · 2017: 9 · 2016: 9 · 2015: 8 · 2014: 1
- FWCI
- 8.91
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- American Journal of Hematology (publishedVersion, cc-by-nc)
- PubMed Central (publishedVersion, cc-by-nc)
- PubMed (publishedVersion)
- Digital Access to Scholarship at Harvard (DASH) (Harvard University) (submittedVersion, cc-by)
- Funders
- Alnylam Pharmaceuticals
- Topics
- Health Sciences › Medicine › Hematology › Iron Metabolism and Disorders
- Health Sciences › Medicine › Genetics › Hemoglobinopathies and Related Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Cell Biology › Hemoglobin structure and function
- Keywords
- beta-thalassemia intermedia, TMPRSS6, RNAi therapy, deferiprone, iron chelation, secondary iron overload, hepcidin, iron deficiency, anemia, combination therapy
- MeSH terms
- Deferiprone, Administration, Oral, Animals, Combined Modality Therapy, Disease Models, Animal, Drug Carriers, Female, Iron, Iron Chelating Agents, Membrane Proteins, Pyridones, Serine Endopeptidases, beta-Thalassemia, RNA Interference, RNA, Small Interfering, Mice, Nanoparticles, Hepcidins
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