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As the human body has no mechanisms to eliminate the excess of iron, its metabolism needs to be tightly controlled in order to avoid all the sequelae associated with high iron levels. Iron overload is the main cause of morbidity and mortality in beta thalassemia. The master regulator of iron homeostasis, hepcidin, is chronically repressed in this disorder, leading to increased intestinal iron absorption and consequent iron overload. Many groups have focused on obtaining a better understanding of the pathways involved in iron regulation. New molecules have recently been synthesized and used in animal models of dysregulated iron metabolism, demonstrating their ability to target and reduce iron load. Antisense oligonucleotides, as well as lipid nanoparticle\u2013formulated small interfering RNAs and minihepcidins peptides, are novel agents that have already proved to be efficient in modulating iron metabolism in mouse models and are therefore promising candidates for the treatment of patients affected by iron disorders.<\/jats:p>","DOI":"10.1111\/nyas.13018","type":"journal-article","created":{"date-parts":[[2016,2,26]],"date-time":"2016-02-26T10:31:59Z","timestamp":1456482719000},"page":"162-168","source":"Crossref","is-referenced-by-count":19,"title":["New strategies to target iron metabolism for the treatment of beta thalassemia"],"prefix":"10.1111","volume":"1368","author":[{"given":"Paraskevi Rea","family":"Oikonomidou","sequence":"first","affiliation":[{"name":"Department of Pediatrics, Division of Hematology Children's Hospital of Philadelphia  Philadelphia Pennsylvania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carla","family":"Casu","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Hematology Children's Hospital of Philadelphia  Philadelphia Pennsylvania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stefano","family":"Rivella","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Hematology Children's Hospital of Philadelphia  Philadelphia Pennsylvania"},{"name":"Cell and Molecular Biology Graduate Group University of Pennsylvania  Philadelphia Pennsylvania"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2016,2,25]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1097\/GIM.0b013e3181cd68ed"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1002\/9780470696705.refs"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(79)92175-5"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.10842"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.cell.2008.01.025"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V51.3.527.527"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/iub.226"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1152\/physrev.1992.72.2.449"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1043-2760(98)00101-5"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2014-11-575357"},{"key":"e_1_2_8_12_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2006-12-063503"},{"key":"e_1_2_8_13_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V98.12.3261"},{"key":"e_1_2_8_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0092-8674(00)81013-2"},{"key":"e_1_2_8_15_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.88.13.5680"},{"key":"e_1_2_8_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.blre.2012.12.003"},{"key":"e_1_2_8_17_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI30117"},{"key":"e_1_2_8_18_1","doi-asserted-by":"publisher","DOI":"10.1038\/sj.leu.2403383"},{"key":"e_1_2_8_19_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2011-07-356006"},{"key":"e_1_2_8_20_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI102335"},{"key":"e_1_2_8_21_1","doi-asserted-by":"crossref","first-page":"64","DOI":"10.1182\/blood.V12.1.64.64","article-title":"Erythrokinetics in Cooley's anemia","volume":"12","author":"Finch C.A.","year":"1957","journal-title":"Blood"},{"key":"e_1_2_8_22_1","doi-asserted-by":"publisher","DOI":"10.1097\/00005792-197001000-00002"},{"key":"e_1_2_8_23_1","doi-asserted-by":"crossref","first-page":"374","DOI":"10.1182\/blood.V82.2.374.374","article-title":"Accelerated programmed cell death (apoptosis) in erythroid precursors of patients with severe beta\u2010thalassemia (Cooley's anemia)","volume":"82","author":"Yuan J.","year":"1993","journal-title":"Blood"},{"key":"e_1_2_8_24_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0301-472X(00)00555-5"},{"key":"e_1_2_8_25_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2007-12-126938"},{"key":"e_1_2_8_26_1","doi-asserted-by":"crossref","first-page":"794","DOI":"10.1182\/blood.V39.6.794.794","article-title":"Hemichromes in single inclusion bodies in red cells of beta thalassemia","volume":"39","author":"Rachmilewitz E.A.","year":"1972","journal-title":"Blood"},{"key":"e_1_2_8_27_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.redox.2015.07.018"},{"key":"e_1_2_8_28_1","first-page":"9","article-title":"Of macrophages and red blood cells; a complex love story","volume":"5","author":"Back D.Z.","year":"2014","journal-title":"Front. 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