Breda L, Ghiaccio V, Tanaka N, Jarocha D, Ikawa Y, Abdulmalik O, Dong A, Casu C, Raabe TD, Shan X, Danet-Desnoyers GA, Doto AM, Everett J, Bushman FD, Radaelli E, Assenmacher CA, Tarrant JC, Hoepp N, Guzikowski V, Smith-Whitley K, Janet L. Kwiatkowski JL, Stefano Rivella S. Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies. Molecular Therapy, in press

Bou-Fakhredin R, Dia B, Ghadieh HE, Rivella S, Cappellini MD, Eid AA, Taher AT. CYP450 Mediates Reactive Oxygen Species Production in a Mouse Model of β-Thalassemia through an Increase in 20-HETE Activity. Int J Mol Sci. 2021 Jan 23;22(3):1106. PMID: 33498614

Sinha S, Pereira-Reis J, Guerra A, Rivella S, Duarte D. The Role of Iron in Benign and Malignant Hematopoiesis. Antioxid Redox Signal. 2021 Jan 7. PMID: 33231101

Musallam KM, Rivella S, Taher AT. Management of non-transfusion-dependent β-thalassemia (NTDT): The next 5 years. Am J Hematol. 2020 Nov 21. PMID: 33219704

Musallam KM, Cappellini MD, Viprakasit V, Kattamis A, Rivella S, Taher AT. Revisiting the non-transfusion-dependent (NTDT) vs. transfusion-dependent (TDT) thalassemia classification 10 years later. Am J Hematol. 2020 Nov 21. PMID: 33219703 No abstract available.

Ghoti H, Ackerman S, Rivella S, Casu C, Nadir Y. Heparanase Level and Procoagulant Activity Are Increased in Thalassemia and Attenuated by Janus Kinase 2 Inhibition. Am J Pathol. 2020 Oct;190(10):2146-2154. PMID: 32745462

Patino E, Doty SB, Bhatia D, Meza K, Zhu YS, Rivella S, Choi ME, Akchurin O. Carbonyl iron and iron dextran therapies cause adverse effects on bone health in juveniles with chronic kidney disease. Kidney Int. 2020 Nov;98(5):1210-1224. PMID: 32574618

Casu C, Pettinato M, Liu A, Aghajan M, Lo Presti V, Lidonnici MR, Munoz KA, O'Hara E, Olivari V, Di Modica SM, Booten S, Guo S, Neil G, Miari R, Shapir N, Zafir-Lavie I, Domev H, Ferrari G, Sitara D, Nai A, Rivella S. Correcting β-thalassemia by combined therapies that restrict iron and modulate erythropoietin activity.Blood. 2020 Oct 22;136(17):1968-1979. PMID: 32556142

Dong A, Ghiaccio V, Motta I, Guo S, Peralta R, Freier SM, Watt A, Damle S, Ikawa Y, Jarocha D, Chappell M, Stephanou C, Delbini P, Chen C, Christou S, Kleanthous M, Smith-Whitley K, Manwani D, Casu C, Abdulmalik O, Cappellini MD, Rivella S, Breda L. 2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring HbA production and chain rebalance. Haematologica. 2020 May 21. PMID: 32439726 Free article.

La P, Oved JH, Ghiaccio V, Rivella S. Mitochondria Biogenesis Modulates Iron-Sulfur Cluster Synthesis to Increase Cellular Iron Uptake. DNA Cell Biol. 2020 May;39(5):756-765. PMID: 32282232

Iolascon A, De Franceschi L, Muckenthaler M, Taher A, Rees D, de Montalembert M, Rivella S, Eleftheriou A, Cappellini MD. EHA Research Roadmap on Hemoglobinopathies and Thalassemia: An Update. Hemasphere. 2019 Jun 4;3(3):e208. PMID: 31723835 

Bystrom LM, Bezerra DP, Hsu HT, Zong H, Lara-Martínez LA, De Leon JP, Emmanuel M, Méry D, Gardenghi S, Hassane D, Neto CC, Cunningham-Rundles S, Becker MW, Rivella S, Guzman ML. Cranberry A-type proanthocyanidins selectively target acute myeloid leukemia cells. Blood Adv. 2019 Nov 12;3(21):3261-3265. PMID: 31698457 

Casu C, Chessa R, Liu A, Gupta R, Drakesmith H, Fleming R, Ginzburg YZ, MacDonald B, Rivella S. Minihepcidins improve ineffective erythropoiesis and splenomegaly in a new mouse model of adult β-thalassemia major. Haematologica. 2020 Jul;105(7):1835-1844. PMID: 31582543 

Chappell M, Rivella S. New potential players in hepcidin regulation. Haematologica. 2019 Sep;104(9):1691-1693. PMID: 31473605 

Parrow NL, Li Y, Feola M, Guerra A, Casu C, Prasad P, Mammen L, Ali F, Vaicikauskas E, Rivella S, Ginzburg YZ, Fleming RE. Lobe specificity of iron binding to transferrin modulates murine erythropoiesis and iron homeostasis. Blood. 2019 Oct 24;134(17):1373-1384. PMID: 31434707 

Ikawa Y, Miccio A, Magrin E, Kwiatkowski JL, Rivella S, Cavazzana M. Gene therapy of hemoglobinopathies: progress and future challenges. Hum Mol Genet. 2019 Oct 1;28. PMID: 31322165 

Breveglieri G, Salvatori F, Finotti A, Cosenza LC, Zuccato C, Bianchi N, Breda L, Rivella S, Bresciani A, Bisbocci M, Borgatti M, Gambari R. Development and characterization of cellular biosensors for HTS of erythroid differentiation inducers targeting the transcriptional activity of γ-globin and β-globin gene promoters. Anal Bioanal Chem. 2019 Nov;411(29):7669-7680. PMID: 31273412

Guerra A, Oikonomidou PR, Sinha S, Zhang J, Lo Presti V, Hamilton CR, Breda L, Casu C, La P, Martins AC, Sendamarai AK, Fleming M, Rivella S. Lack of Gdf11 does not improve anemia or prevent the activity of RAP-536 in a mouse model of β-thalassemia. Blood. 2019 Aug 8;134(6):568-572. PMID: 31151988 

Akchurin O, Patino E, Dalal V, Meza K, Bhatia D, Brovender S, Zhu YS, Cunningham-Rundles S, Perelstein E, Kumar J, Rivella S, Choi ME. Interleukin-6 Contributes to the Development of Anemia in Juvenile CKD. Kidney Int Rep. 2018 Dec 19;4(3):470-483. PMID: 30899874 

Stagg DB, Whittlesey RL, Li X, Lozovatsky L, Gardenghi S, Rivella S, Finberg KE. Genetic loss of Tmprss6 alters terminal erythroid differentiation in a mouse model of β-thalassemia intermedia. Haematologica. 2019 Oct;104(10):e442-e446. PMID: 30819909 

Ghiaccio V, Chappell M, Rivella S, Breda L. Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges. Mol Diagn Ther. 2019 Apr;23(2):173-186. PMID: 30701409 

Rivella S. Iron metabolism under conditions of ineffective erythropoiesis in β-thalassemia. Blood. 2019 Jan 3;133(1):51-58. PMID: 30401707 

Casu C, Nemeth E, Rivella S. Hepcidin agonists as therapeutic tools. Blood. 2018 Mar 9. PMID: 29523504

Agoro R, Montagna A, Goetz R, Aligbe O, Singh G, Coe LM, Mohammadi M, Rivella S, Sitara D. Inhibition of fibroblast growth factor 23 (FGF23) signaling rescues renal anemia. FASEB J. 2018 Feb 26. PMID: 29481308

Guerra A, Musallam KM, Taher AT, Rivella S. Emerging Therapies. Hematol Oncol Clin North Am. 2018 Apr;32(2):343-352. PMID: 29458736

Gupta R, Musallam KM, Taher AT, Rivella S. Ineffective Erythropoiesis: Anemia and Iron Overload. Hematol Oncol Clin North Am. 2018 Apr;32(2):213-221. PMID: 29458727

Aschemeyer S, Qiao B, Stefanova D, Valore EV, Sek AC, Ruwe TA, Vieth KR, Jung G, Casu C, Rivella S, Jormakka M, Mackenzie B, Ganz T, Nemeth E. Structure-function analysis of ferroportin defines the binding site and an alternative mechanism of action of hepcidin. Blood. 2018 Feb 22;131(8). PMID: 29237594

Dong AC, Rivella S. Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia. Adv Exp Med Biol. 2017;1013:155-176. doi: 10.1007/978-1-4939-7299-9_6. PMID: 29127680

Casu C, Presti VL, Oikonomidou PR, Melchiori L, Abdulmalik O, Ramos P, Rivella S. Short-term administration of JAK2 inhibitors reduces splenomegaly in mouse models of β-thalassemia intermedia and major. Haematologica. 2018 Feb;103(2). PMID: 29097498

Taher AT, Karakas Z, Cassinerio E, Siritanaratkul N, Kattamis A, Maggio A, Rivella S, Hollaender N, Mahuzier B, Gadbaw B, Aydinok Y. Efficacy and safety of ruxolitinib in regularly transfused patients with thalassemia: results from a phase 2a study. Blood. 2018 Jan 11;131(2). PMID: 29097381

Oikonomidou PR, Rivella S. What can we learn from ineffective erythropoiesis in thalassemia? Blood Rev. 2017 Oct 3. PMID: 29054350

Hotz MJ, Qing D, Shashaty MGS, Zhang P, Faust H, Sondheimer N, Rivella S, Worthen GS, Mangalmurti NS. Red Blood Cells Homeostatically Bind Mitochondrial DNA through TLR9 to Maintain Quiescence and to Prevent Lung Injury.
Am J Respir Crit Care Med. 2018 Feb 15. PMID: 29053005

Pasricha SR, Lim PJ, Duarte TL, Casu C, Oosterhuis D, Mleczko-Sanecka K, Suciu M, Da Silva AR, Al-Hourani K, Arezes J, McHugh K, Gooding S, Frost JN, Wray K, Santos A, Porto G, Repapi E, Gray N, Draper SJ, Ashley N, Soilleux E, Olinga P, Muckenthaler MU, Hughes JR, Rivella S, Milne TA, Armitage AE, Drakesmith H. Hepcidin is regulated by promoter-associated histone acetylation and HDAC3. Nat Commun. 2017 Sep 1. PMID: 28864822

Nowak RB, Papoin J, Gokhin DS, Casu C, Rivella S, Lipton JM, Blanc L, Fowler VM. Tropomodulin 1 controls erythroblast enucleation via regulation of F-actin in the enucleosome. Blood. 2017 Aug 31. PMID: 28729432

Iolascon A, Andolfo I, Barcellini W, Corcione F, Garçon L, De Franceschi L, Pignata C, Graziadei G, Pospisilova D, Rees DC, de Montalembert M, Rivella S, Gambale A, Russo R, Ribeiro L, Vives-Corrons J, Martinez PA, Kattamis A, Gulbis B, Cappellini MD, Roberts I, Tamary H; Working Study Group on Red Cells and Iron of the EHA. Recommendations regarding splenectomy in hereditary hemolytic anemias. Haematologica. 2017 Aug. PMID: 28550188

Crielaard BJ, Lammers T, Rivella S. Targeting iron metabolism in drug discovery and delivery. Nat Rev Drug Discov. 2017 Feb 3. PMID: 28154410

Li H, Choesang T, Bao W, Chen H, Feola M, Garcia-Santos D, Li J, Sun S, Follenzi A, Pham P, Liu J, Zhang J, Ponka P, An X, Mohandas N, Fleming E, Rivella S, Li G, Ginzburg Y. Decreasing TfR1 expression reverses anemia and hepcidin suppression in β-thalassemic mice. Blood. 2017 Feb 1. PMID: 28151426
Muckenthaler MU, Rivella S, Hentze MW, Galy B. A Red Carpet for Iron Metabolism. Cell. 2017 Jan 26. PMID: 28129536

Han CY, Koo JH, Kim SH, Gardenghi S, Rivella S, Strnad P, Hwang SJ, Kim SG. Hepcidin inhibits Smad3 phosphorylation in hepatic stellate cells by impeding ferroportin-mediated regulation of Akt. Nat Commun. 2016 Dec 22;7:13817. PMID: 28004654 

Bystrom LM, Zong H, Martinez L, Neto C, Roboz GJ, Rivella S, Guzman ML. A-type proanthocyanidins selectively target acute myeloid leukemia cells in vitro and in vivo. Planta Med. 2016 Dec;81. PMID: 27976147

Akchurin O, Sureshbabu A, Doty SB, Zhu YS, Patino E, Cunningham-Rundles S, Choi ME, Boskey AL, Rivella S. Lack of Hepcidin Ameliorates Anemia and Improves Growth in an Adenine-induced Mouse Model of Chronic Kidney Disease. Am J Physiol Renal Physiol. 2016 Nov 1;311(5) PMID:27440777

Casu C, Oikonomidou PR, Chen H, Nandi V, Ginzburg Y, Prasad P, Fleming RE, Shah YM, Valore EV, Nemeth E, Ganz T, MacDonald B, Rivella S. Minihepcidin peptides as disease modifiers in mice affected by β-thalassemia and polycythemia vera. Blood. 2016 Jul 14;128(2):265-76. PMID:27154187

Breda L, Motta I, Lourenco S, Gemmo C, Deng W, Rupon JW, Abdulmalik OY, Manwani D, Blobel GA, Rivella S. Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers. Blood. 2016 Jul 12. PMID:27405777

Oikonomidou PR, Casu C, Yang Z, Crielaard B, Shim JH, Rivella S, Vogiatzi MG. Polycythemia is associated with bone loss and reduced osteoblast activity in mice. Osteoporos Int. 2016 Apr;27(4):1559-68. PMID:26650379

Oikonomidou PR, Casu C, Rivella S. New strategies to target iron metabolism for the treatment of beta thalassemia. Ann N Y Acad Sci. 2016 Mar;1368(1):162-8. PMID:26919168

Chen H, Choesang T, Li H, Sun S, Pham P, Bao W, Feola M, Westerman M, Li G, Follenzi A, Blanc L,Rivella S, Fleming RE, Ginzburg YZ. Increased hepcidin in transferrin-treated thalassemic mice correlates with increased liver BMP2 expression and decreased hepatocyte ERK activation. Haematologica. 2016 Mar;101(3):297-308. PMID:26635037

Engert A, Balduini C, Brand A, Coiffier B, Cordonnier C, Döhner H, de Wit TD, Eichinger S, Fibbe W, Green T, de Haas F, Iolascon A, Jaffredo T, Rodeghiero F, Salles G, Schuringa JJ; EHA Roadmap for European Hematology Research. The European Hematology Association Roadmap for European Hematology Research: a consensus document. Haematologica. 2016 Feb;101(2):115-208. PMID:26819058

Casu C, Aghajan M, Oikonomidou PR, Guo S, Monia BP, Rivella S. Combination of Tmprss6- ASO and the iron chelator deferiprone improves erythropoiesis and reduces iron overload in a mouse model of beta-thalassemia intermedia. Haematologica. 2016 Jan;101(1). PMID:26405152

Valdés-Ferrer SI, Papoin J, Dancho ME, Olofsson P, Li J, Lipton JM, Avancena P, Yang H, Zou YR, Chavan SS, Volpe BT, Gardenghi S, Rivella S, Diamond B, Andersson U, Steinberg BM, Blanc L, Tracey KJ. HMGB1 mediates anemia of inflammation in murine sepsis survivors. Mol Med. 2015 Dec 29. PMID:26736178

Das N, Xie L, Ramakrishnan SK, Campbell A, Rivella S, Shah YM. Intestine-specific Disruption of Hypoxia-inducible Factor (HIF)-2α Improves Anemia in Sickle Cell Disease. J Biol Chem. 2015 Sep 25;290(39):23523-7. PMID:26296885

Guimarães JS, Cominal JG, Silva-Pinto AC, Olbina G, Ginzburg YZ, Nandi V, Westerman M, Rivella S, de Souza AM. Altered erythropoiesis and iron metabolism in carriers of thalassemia. Eur J Haematol. 2015 Jun;94(6):511-8. PMID:25307880

Rivella S. β-thalassemias: paradigmatic diseases for scientific discoveries and development of innovative therapies. Haematologica. 2015 Apr;100(4):418-30. PMID:25828088

Bystrom LM, Rivella S. Cancer cells with irons in the fire. Free Radic Biol Med. 2015 Feb;79:337-42. PMID:24835768

Breveglieri G, Mancini I, Bianchi N, Lampronti I, Salvatori F, Fabbri E, Zuccato C, Cosenza LC, Montagner G, Borgatti M, Altruda F, Fagoonee S, Carandina G, Rubini M, Aiello V, Breda L, Rivella S, Gambari R, Finotti A. Generation and Characterization of a Transgenic Mouse Carrying a Functional Human β -Globin Gene with the IVSI-6 Thalassemia Mutation. Biomed Res Int. 2015;2015:687635. PMID:26097845

Yien YY, Gnanapragasam MN, Gupta R, Rivella S, Bieker JJ. Alternative splicing of EKLF/KLF1 in murine primary erythroid tissues. Exp Hematol. 2015 Jan;43(1):65-70. PMID:25283745

Finotti A, Breda L, Lederer CW, Bianchi N, Zuccato C, Kleanthous M, Rivella S, Gambari R. Recent trends in the gene therapy of β-thalassemia. J Blood Med. 2015 Feb 19;6:69-85. PMID:25737641

Casu C, Rivella S. Iron age: novel targets for iron overload. Hematology Am Soc Hematol Educ Program. 2014 Dec 5;2014(1):216-21. PMID:25696858

Zhang X, Campreciós G, Rimmelé P, Liang R, Yalcin S, Mungamuri SK, Barminko J, D'Escamard V, Baron MH, Brugnara C, Papatsenko D, Rivella S, Ghaffari S. FOXO3-mTOR metabolic cooperation in the regulation of erythroid cell maturation and homeostasis. Am J Hematol. 2014 Oct;89(10):954-63. PMID:24966026

Deng W, Rupon JW, Krivega I, Breda L, Motta I, Jahn KS, Reik A, Gregory PD, Rivella S, Dean A, Blobel GA. Reactivation of developmentally silenced globin genes by forced chromatin looping. Cell. 2014 Aug 14;158(4):849-60. PMID:25126789

Kautz L, Jung G, Valore EV, Rivella S, Nemeth E, Ganz T. Identification of erythroferrone as an erythroid regulator of iron metabolism. Nat Genet. 2014 Jul;46(7):678-84. PMID:24880340

Suragani RN, Cawley SM, Li R, Wallner S, Alexander MJ, Mulivor AW, Gardenghi S, Rivella S, Grinberg AV, Pearsall RS, Kumar R. Modified activin receptor IIB ligand trap mitigates ineffective erythropoiesis and disease complications in murine β-thalassemia. Blood. 2014 Jun 19;123(25):3864-72. PMID:24795345

Martin L, Grigoryan A, Wang D, Wang J, Breda L, Rivella S, Cardozo T, Gardner LB. Identification and characterization of small molecules that inhibit nonsense-mediated RNA decay and suppress nonsense p53 mutations. Cancer Res. 2014 Jun 1;74(11):3104-13. PMID:24662918

Crielaard BJ, Rivella S. β-Thalassemia and Polycythemia vera: targeting chronic stress erythropoiesis. Int J Biochem Cell Biol. 2014 Jun;51:89-92. PMID:24718374

Bystrom LM, Guzman ML, Rivella S. Iron and reactive oxygen species: friends or foes of cancer cells? Antioxid Redox Signal. 2014 Apr 20;20(12):1917-24. PMID:23198911

Coe LM, Madathil SV, Casu C, Lanske B, Rivella S, Sitara D. FGF-23 is a negative regulator of prenatal and postnatal erythropoiesis. J Biol Chem. 2014 Apr 4;289(14):9795-810. PMID:24509850

Breda L, Rivella S. Modulators of erythropoiesis: emerging therapies for hemoglobinopathies and disorders of red cell production. Hematol Oncol Clin North Am. 2014 Apr;28(2):375-86. PMID:24589272

Gardenghi S, Renaud TM, Meloni A, Casu C, Crielaard BJ, Bystrom LM, Greenberg-Kushnir N, Sasu BJ, Cooke KS, Rivella S. Distinct roles for hepcidin and interleukin-6 in the recovery from anemia in mice injected with heat-killed Brucella abortus. Blood. 2014 Feb 20;123(8):1137-45. PMID:24357729

Rivella S. Enucleate or replicate? Ask the cytoskeleton. Blood. 2014 Jan 30;123(5):601-2. PMID:24482493

Anderson ER, Taylor M, Xue X, Ramakrishnan SK, Martin A, Xie L, Bredell BX, Gardenghi S, Rivella S, Shah YM. Intestinal HIF2α promotes tissue-iron accumulation in disorders of iron overload with anemia. Proc Natl Acad Sci U S A. 2013 Dec 10;110(50):E4922-30. PMID:24282296

Richardson CL, Delehanty LL, Bullock GC, Rival CM, Tung KS, Kimpel DL, Gardenghi S, Rivella S, Goldfarb AN. Isocitrate ameliorates anemia by suppressing the erythroid iron restriction response. J Clin Invest. 2013 Aug;123(8):3614-23. PMID:23863711

Breda L, Rivella S, Zuccato C, Gambari R. Combining gene therapy and fetal hemoglobin induction for treatment of β-thalassemia. Expert Rev Hematol. 2013 Jun;6(3):255-64. PMID:23782080

Musallam KM, Rivella S, Vichinsky E, Rachmilewitz EA. Non-transfusion-dependent thalassemias. Haematologica. 2013 Jun;98(6):833-44. PMID:23729725

Guo S, Casu C, Gardenghi S, Booten S, Aghajan M, Peralta R, Watt A, Freier S, Monia BP, Rivella S. Reducing TMPRSS6 ameliorates hemochromatosis and β-thalassemia in mice. J Clin Invest. 2013 Apr;123(4):1531-41. PMID:23524968

Ramos P, Casu C, Gardenghi S, Breda L, Crielaard BJ, Guy E, Marongiu MF, Gupta R, Levine RL, Abdel-Wahab O, Ebert BL, Van Rooijen N, Ghaffari S, Grady RW, Giardina PJ, Rivella S. Macrophages support pathological erythropoiesis in polycythemia vera and β-thalassemia. Nat Med. 2013 Apr;19(4):437-45. PMID:23502961

Dong A, Rivella S, Breda L.Gene therapy for hemoglobinopathies: progress and challenges. Transl Res. 2013 Apr;161(4):293-306. PMID: 23337292

Casanovas G, Vujić Spasic M, Casu C, Rivella S, Strelau J, Unsicker K, Muckenthaler MU. The murine growth differentiation factor 15 is not essential for systemic iron homeostasis in phlebotomized mice. Haematologica. 2013 Mar;98(3):444-7. PMID: 22983584

Zuccato C, Breda L, Salvatori F, Breveglieri G, Gardenghi S, Bianchi N, Brognara E, Lampronti I, Borgatti M, Rivella S, Gambari R. A combined approach for β-thalassemia based on gene therapy-mediated adult hemoglobin (HbA) production and fetal hemoglobin (HbF) induction. Ann Hematol. 2012 Aug;91(8):1201-13. PMID: 22460946

Wang L, Rosenberg JB, De BP, Ferris B, Wang R, Rivella S, Kaminsky SM, Crystal RG. In vivo gene transfer strategies to achieve partial correction of von Willebrand disease. Hum Gene Ther. 2012 Jun;23(6):576-88. PMID: 22482515

Rivella S. Do not super-excess me! Blood. 2012 May 31;119(22):5064-5. PMID: 22653953

Kobayashi H, Gilbert V, Liu Q, Kapitsinou PP, Unger TL, Rha J, Rivella S, Schlöndorff D, Haase VH. Myeloid cell-derived hypoxia-inducible factor attenuates inflammation in unilateral ureteral obstruction-induced kidney injury. J Immunol. 2012 May 15;188(10):5106-15. PMID: 22490864

Rivella S. The role of ineffective erythropoiesis in non-transfusion-dependent thalassemia. Blood Rev. 2012 Apr;26 Suppl 1:S12-5. PMID: 22631035

Parrow NL, Gardenghi S, Ramos P, Casu C, Grady RW, Anderson ER, Shah YM, Li H, Ginzburg YZ, Fleming RE, Rivella S. Decreased hepcidin expression in murine β-thalassemia is associated with suppression of Bmp/Smad signaling. Blood. 2012 Mar 29;119(13):3187-9. PMID:22461476

Breda L, Casu C, Gardenghi S, Bianchi N, Cartegni L, Narla M, Yazdanbakhsh K, Musso M, Manwani D, Little J, Gardner LB, Kleinert DA, Prus E, Fibach E, Grady RW, Giardina PJ, Gambari R, Rivella S. Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients. PLoS One. 2012;7(3):e32345. PMID: 22479321

Ginzburg Y, Rivella S. β-thalassemia: a model for elucidating the dynamic regulation of ineffective erythropoiesis and iron metabolism. Blood. 2011 Oct 20;118(16):4321-30. PMID:21768301

Parrow NL, Gardenghi S, Rivella S. Prospects for a hepcidin mimic to treat β-thalassemia and hemochromatosis. Expert Rev Hematol. 2011 Jun;4(3):233-5. PMID: 21668388

Ramos P, Guy E, Chen N, Proenca CC, Gardenghi S, Casu C, Follenzi A, Van Rooijen N, Grady RW, de Sousa M, Rivella S. Enhanced erythropoiesis in Hfe-KO mice indicates a role for Hfe in the modulation of erythroid iron homeostasis. Blood. 2011 Jan 27;117(4):1379-89. PMID: 21059897

Gardenghi S, Grady RW, Rivella S. Anemia, ineffective erythropoiesis, and hepcidin: interacting factors in abnormal iron metabolism leading to iron overload in β-thalassemia. Hematol Oncol Clin North Am. 2010 Dec;24(6):1089-107. PMID: 21075282

Gardenghi S, Ramos P, Marongiu MF, Melchiori L, Breda L, Guy E, Muirhead K, Rao N, Roy CN, Andrews NC, Nemeth E, Follenzi A, An X, Mohandas N, Ginzburg Y, Rachmilewitz EA, Giardina PJ, Grady RW, Rivella S. Hepcidin as a therapeutic tool to limit iron overload and improve anemia in β-thalassemic mice. J Clin Invest. 2010 Dec;120(12):4466-77. PMID: 21099112

Nasimuzzaman M, Khandros E, Wang X, Kong Y, Zhao H, Weiss D, Rivella S, Weiss MJ, Persons DA. Analysis of alpha hemoglobin stabilizing protein overexpression in murine β-thalassemia. Am J Hematol. 2010 Oct;85(10):820-2. PMID: 20815047

Gardenghi S, Ramos P, Follenzi A, Rao N, Rachmilewitz EA, Giardina PJ, Grady RW, Rivella S. Hepcidin and Hfe in iron overload in beta-thalassemia. Ann N Y Acad Sci. 2010 Aug;1202:221-5. PMID: 20712796

Breda L, Kleinert DA, Casu C, Casula L, Cartegni L, Fibach E, Mancini I, Giardina PJ, Gambari R,Rivella S. A preclinical approach for gene therapy of beta-thalassemia. Ann N Y Acad Sci. 2010 Aug;1202:134-40. PMID: 20712784

Ramos P, Melchiori L, Gardenghi S, Van-Roijen N, Grady RW, Ginzburg Y, Rivella S. Iron metabolism and ineffective erythropoiesis in beta-thalassemia mouse models. Ann N Y Acad Sci. 2010 Aug;1202:24-30. PMID: 20712768

Vogiatzi MG, Tsay J, Verdelis K, Rivella S, Grady RW, Doty S, Giardina PJ, Boskey AL. Changes in bone microarchitecture and biomechanical properties in the th3 thalassemia mouse are associated with decreased bone turnover and occur during the period of bone accrual. Calcif Tissue Int. 2010 Jun;86(6):484-94. PMID: 20449578

Rivella S, Nemeth E, Miller JL. Crosstalk between Erythropoiesis and Iron Metabolism. Adv Hematol. 2010;2010. pii: 317095. PMID: 20862206

Melchiori L, Gardenghi S, Rivella S. beta-Thalassemia: HiJAKing Ineffective Erythropoiesis and Iron Overload. Adv Hematol. 2010;2010:938640. PMID:20508726

Rivella S, Rachmilewitz E. Future alternative therapies for β-thalassemia. Expert Rev Hematol. 2009 Dec 1;2(6):685. PMID: 20174612

Chan AS, Jensen KK, Skokos D, Doty S, Lederman HK, Kaplan RN, Rafii S, Rivella S, Lyden D. Id1 represses osteoclast-dependent transcription and affects bone formation and hematopoiesis. PLoS One. 2009 Nov 24;4(11):e7955. PMID:19956687

Breda L, Gambari R, Rivella S. Gene therapy in thalassemia and hemoglobinopathies. Mediterr J Hematol Infect Dis. 2009 Nov 13;1(1):e2009008. PMID:21415990

Salvatori F, Breveglieri G, Zuccato C, Finotti A, Bianchi N, Borgatti M, Feriotto G, Destro F, Canella A, Brognara E, Lampronti I, Breda L, Rivella S, Gambari R. Production of beta-globin and adult hemoglobin following G418 treatment of erythroid precursor cells from homozygous beta(0)39 thalassemia patients. Am J Hematol. 2009 Nov;84(11):720-8. PMID:19810011

Biagioli M, Pinto M, Cesselli D, Zaninello M, Lazarevic D, Roncaglia P, Simone R, Vlachouli C, Plessy C, Bertin N, Beltrami A, Kobayashi K, Gallo V, Santoro C, Ferrer I, Rivella S, Beltrami CA, Carninci P, Raviola E, Gustincich S.

Unexpected expression of alpha- and beta-globin in mesencephalic dopaminergic neurons and glial cells. Proc Natl Acad Sci U S A. 2009 Sep 8;106(36):15454-9. PMID:19717439

Salvatori F, Cantale V, Breveglieri G, Zuccato C, Finotti A, Bianchi N, Borgatti M, Feriotto G, Destro F, Canella A, Breda L, Rivella S, Gambari R. Development of K562 cell clones expressing beta-globin mRNA carrying the beta039 thalassaemia mutation for the screening of correctors of stop-codon mutations. Biotechnol Appl Biochem. 2009 Jul 9;54(1):41-52. PMID: 19216718

Rivella S. Ineffective erythropoiesis and thalassemias. Curr Opin Hematol. 2009 May;16(3):187-94. PMID:19318943

Rechavi G, Rivella S. Regulation of iron absorption in hemoglobinopathies. Curr Mol Med. 2008 Nov;8(7):646-62. Review. PMID:18991651

Libani IV, Guy EC, Melchiori L, Schiro R, Ramos P, Breda L, Scholzen T, Chadburn A, Liu Y, Kernbach M, Baron-Lühr B, Porotto M, de Sousa M, Rachmilewitz EA, Hood JD, Cappellini MD, Giardina PJ, Grady RW, Gerdes J, Rivella S. Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in beta-thalassemia. Blood. 2008 Aug 1;112(3):875-85. PMID:18480424

Pinto JP, Ramos P, de Almeida SF, Oliveira S, Breda L, Michalak M, Porto G, Rivella S, de Sousa M. Protective role of calreticulin in HFE hemochromatosis. Free Radic Biol Med. 2008 Jan 1;44(1):99-108. PMID:18045552

Gardenghi S, Marongiu MF, Ramos P, Guy E, Breda L, Chadburn A, Liu Y, Amariglio N, Rechavi G, Rachmilewitz EA, Breuer W, Cabantchik ZI, Wrighting DM, Andrews NC, de Sousa M, Giardina PJ, Grady RW, Rivella S. Ineffective erythropoiesis in beta-thalassemia is characterized by increased iron absorption mediated by down-regulation of hepcidin and up-regulation of ferroportin. Blood. 2007 Jun 1;109(11):5027-35. PMID:17299088

Sadelain M, Boulad F, Galanello R, Giardina P, Locatelli F, Maggio A, Rivella S, Riviere I, Tisdale J. Therapeutic options for patients with severe beta-thalassemia: the need for globin gene therapy. Hum Gene Ther. 2007 Jan;18(1):1-9. PMID:17173507

Weizer-Stern O, Adamsky K, Amariglio N, Levin C, Koren A, Breuer W, Rachmilewitz E, Breda L,Rivella S, Cabantchik ZI, Rechavi G. Downregulation of hepcidin and haemojuvelin expression in the hepatocyte cell-line HepG2 induced by thalassaemic sera. Br J Haematol. 2006 Oct;135(1):129-38. PMID:16939499

Weizer-Stern O, Adamsky K, Amariglio N, Rachmilewitz E, Breda L, Rivella S, Rechavi G. mRNA expression of iron regulatory genes in beta-thalassemia intermedia and beta-thalassemia major mouse models. Am J Hematol. 2006 Jul;81(7):479-83. PMID:16755567

Breda L, Gardenghi S, Guy E, Rachmilewitz EA, Weizer-Stern O, Adamsky K, Amariglio N, Rechavi G, Giardina PJ, Grady RW, Rivella S. Exploring the role of hepcidin, an antimicrobial and iron regulatory peptide, in increased iron absorption in beta-thalassemia. Ann N Y Acad Sci. 2005;1054:417-22. PMID:16339690

Rachmilewitz EA, Weizer-Stern O, Adamsky K, Amariglio N, Rechavi G, Breda L, Rivella S, Cabantchik ZI. Role of iron in inducing oxidative stress in thalassemia: Can it be prevented by inhibition of absorption and by antioxidants? Ann N Y Acad Sci. 2005;1054:118-23. Review. PMID:16339657

Sadelain M, Lisowski L, Samakoglu S, Rivella S, May C, Riviere I. Progress toward the genetic treatment of the beta-thalassemias. Ann N Y Acad Sci. 2005;1054:78-91. PMID:16339654

Sadelain M, Rivella S, Lisowski L, Samakoglu S, Rivière I. Globin gene transfer for treatment of the beta-thalassemias and sickle cell disease. Best Pract Res Clin Haematol. 2004 Sep;17(3):517-34. PMID:15498721

Adamsky K, Weizer O, Amariglio N, Breda L, Harmelin A, Rivella S, Rachmilewitz E, Rechavi G. Decreased hepcidin mRNA expression in thalassemic mice. Br J Haematol. 2004 Jan;124(1):123-4. PMID:14675418

Rivella S, May C, Chadburn A, Rivière I, Sadelain M. A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human beta-globin gene transfer. Blood. 2003 Apr 15;101(8):2932-9. PMID: 12480689

Rivella S, Sadelain M. Therapeutic globin gene delivery using lentiviral vectors. Curr Opin Mol Ther. 2002 Oct;4(5):505-14. PMID: 12435053

May C, Rivella S, Chadburn A, Sadelain M. Successful treatment of murine beta-thalassemia intermedia by transfer of the human beta-globin gene. Blood. 2002 Mar 15;99(6):1902-8. PMID: 11877258

May C, Rivella S, Callegari J, Heller G, Gaensler KM, Luzzatto L, Sadelain M. Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin. Nature. 2000 Jul 6;406(6791):82-6. PMID: 10894546

Rivella S, Callegari JA, May C, Tan CW, Sadelain M. The cHS4 insulator increases the probability of retroviral expression at random chromosomal integration sites. J Virol. 2000 May;74(10):4679-87. PMID: 10775605

Rivella S, Palermo B, Pelizon C, Sala C, Arrigo G, Toniolo D. Selection and mapping of replication origins from a 500-kb region of the human X chromosome and their relationship to gene expression. Genomics. 1999 Nov 15;62(1):11-20. PMID: 10585763

Sadelain M, May C, Rivella S, Glade Bender J. Basic principles of gene transfer in hematopoietic stem cells. Prog Exp Tumor Res. 1999;36:1-19. PMID: 10386062

Rivella S, Sadelain M. Genetic treatment of severe hemoglobinopathies: the combat against transgene variegation and transgene silencing. Semin Hematol. 1998 Apr;35(2):112-25. PMID: 9565154

Mancini M, Sala C, Rivella S, Toniolo D. Selection and fine mapping of chromosome-specific cDNAs: application to human chromosome 1. Genomics. 1996 Dec 1;38(2):149-54. PMID: 8954796

Rivella S, Tamanini F, Bione S, Mancini M, Herman G, Chatterjee A, Maestrini E, Toniolo D. A comparative transcriptional map of a region of 250 kb on the human and mouse X chromosome between the G6PD and the FLN1 genes. Genomics. 1995 Aug 10;28(3):377-82. PMID: 7490070

Bione S, Maestrini E, Rivella S, Mancini M, Regis S, Romeo G, Toniolo D. Identification of a novel X-linked gene responsible for Emery-Dreifuss muscular dystrophy. Nat Genet. 1994 Dec;8(4):323-7. PMID: 7894480

Tribioli C, Mancini M, Plassart E, Bione S, Rivella S, Sala C, Torri G, Toniolo D. Isolation of new genes in distal Xq28: transcriptional map and identification of a human homologue of the ARD1 N-acetyl transferase of Saccharomyces cerevisiae. Hum Mol Genet. 1994 Jul;3(7):1061-7. PMID: 7981673

Gariboldi M, Maestrini E, Canzian F, Manenti G, De Gregorio L, Rivella S, Chatterjee A, Herman GE, Archidiacono N, Antonacci R, et al. Comparative mapping of the actin-binding protein 280 genes in human and mouse. Genomics. 1994 May 15;21(2):428-30. PMID: 8088838

Bione S, Tamanini F, Maestrini E, Tribioli C, Poustka A, Torri G, Rivella S, Toniolo D.  Transcriptional organization of a 450-kb region of the human X chromosome in Xq28. Proc Natl Acad Sci U S A. 1993 Dec 1;90(23):10977-81. PMID: 8248200

Maestrini E, Patrosso C, Mancini M, Rivella S, Rocchi M, Repetto M, Villa A, Frattini A, Zoppè M, Vezzoni P, et al. Mapping of two genes encoding isoforms of the actin binding protein ABP-280, a dystrophin like protein, to Xq28 and to chromosome 7. Hum Mol Genet. 1993 Jun;2(6):761-6. PMID: 7689010