Lack of Does Not Improve Anemia or Prevent the Activity of RAP-536 in a Mouse Model of β-thalassemia

Overview

Journal Blood

Publisher Elsevier

Specialty Hematology

Date 2019 Jun 2

PMID 31151988

Citations 27

Authors

Amaliris Guerra

Paraskevi Rea Oikonomidou

Sayantani Sinha

Jianbing Zhang

Vania Lo Presti

Callum R Hamilton

Laura Breda

Carla Casu

Ping La

Ana C Martins

Anoop K Sendamarai

Mark Fleming

Stefano Rivella

Affiliations

Soon will be listed here.

Citing Articles

Combination of a TGF-β ligand trap (RAP-GRL) and TMPRSS6-ASO is superior for correcting β-thalassemia.

Guerra A, Hamilton N, Rivera A, Demsko P, Guo S, Rivella S Am J Hematol. 2024; 99(7):1300-1312.

PMID: 38659383 PMC: 11166515. DOI: 10.1002/ajh.27332.

Anemia in patients receiving anticancer treatments: focus on novel therapeutic approaches.

Bozzini C, Busti F, Marchi G, Vianello A, Cerchione C, Martinelli G Front Oncol. 2024; 14:1380358.

PMID: 38628673 PMC: 11018927. DOI: 10.3389/fonc.2024.1380358.

Tissue Expression of Growth Differentiation Factor 11 in Patients with Breast Cancer.

Chen C, Lee T, Tsai I, Hsuan C, Hsu C, Wang C Diagnostics (Basel). 2024; 14(7).

PMID: 38611614 PMC: 11011301. DOI: 10.3390/diagnostics14070701.

Novel potential therapeutics to modify iron metabolism and red cell synthesis in diseases associated with defective erythropoiesis.

Guerra A, Parhiz H, Rivella S Haematologica. 2023; 108(10):2582-2593.

PMID: 37345473 PMC: 10542825. DOI: 10.3324/haematol.2023.283057.

Profile of Luspatercept in the Treatment of Anemia in Adults with Non-Transfusion-Dependent β-Thalassemia (NTDT): Design, Development and Potential Place in Therapy.

Musallam K, Taher A, Kattamis A, Kuo K, Sheth S, Cappellini M Drug Des Devel Ther. 2023; 17:1583-1591.

PMID: 37255740 PMC: 10226484. DOI: 10.2147/DDDT.S368584.

References

Ogilvy S, Metcalf D, Gibson L, Bath M, Harris A, Adams J . Promoter elements of vav drive transgene expression in vivo throughout the hematopoietic compartment. Blood. 1999; 94(6):1855-63. View

Heinrich A, Pelanda R, Klingmuller U . A mouse model for visualization and conditional mutations in the erythroid lineage. Blood. 2004; 104(3):659-66. DOI: 10.1182/blood-2003-05-1442. View

Ventura A, Kirsch D, McLaughlin M, Tuveson D, Grimm J, Lintault L . Restoration of p53 function leads to tumour regression in vivo. Nature. 2007; 445(7128):661-5. DOI: 10.1038/nature05541. View

Sinha M, Jang Y, Oh J, Khong D, Wu E, Manohar R . Restoring systemic GDF11 levels reverses age-related dysfunction in mouse skeletal muscle. Science. 2014; 344(6184):649-52. PMC: 4104429. DOI: 10.1126/science.1251152. View

Loffredo F, Steinhauser M, Jay S, Gannon J, Pancoast J, Yalamanchi P . Growth differentiation factor 11 is a circulating factor that reverses age-related cardiac hypertrophy. Cell. 2013; 153(4):828-39. PMC: 3677132. DOI: 10.1016/j.cell.2013.04.015. View

Attie K, Allison M, McClure T, Boyd I, Wilson D, Pearsall A . A phase 1 study of ACE-536, a regulator of erythroid differentiation, in healthy volunteers. Am J Hematol. 2014; 89(7):766-70. PMC: 4173124. DOI: 10.1002/ajh.23732. View

Modell B, Darlison M . Global epidemiology of haemoglobin disorders and derived service indicators. Bull World Health Organ. 2008; 86(6):480-7. PMC: 2647473. DOI: 10.2471/blt.06.036673. View

Lin Y, Slape C, Zhang Z, Aplan P . NUP98-HOXD13 transgenic mice develop a highly penetrant, severe myelodysplastic syndrome that progresses to acute leukemia. Blood. 2005; 106(1):287-95. PMC: 1201424. DOI: 10.1182/blood-2004-12-4794. View

Paulson R . Targeting a new regulator of erythropoiesis to alleviate anemia. Nat Med. 2014; 20(4):334-5. DOI: 10.1038/nm.3524. View

10.

McPherron A, Huynh T, Lee S . Redundancy of myostatin and growth/differentiation factor 11 function. BMC Dev Biol. 2009; 9:24. PMC: 2666675. DOI: 10.1186/1471-213X-9-24. View

11.

Dussiot M, Maciel T, Fricot A, Chartier C, Negre O, Veiga J . An activin receptor IIA ligand trap corrects ineffective erythropoiesis in β-thalassemia. Nat Med. 2014; 20(4):398-407. PMC: 7730561. DOI: 10.1038/nm.3468. View

12.

Sherman M, Borgstein N, Mook L, Wilson D, Yang Y, Chen N . Multiple-dose, safety, pharmacokinetic, and pharmacodynamic study of sotatercept (ActRIIA-IgG1), a novel erythropoietic agent, in healthy postmenopausal women. J Clin Pharmacol. 2013; 53(11):1121-30. DOI: 10.1002/jcph.160. View

13.

Breda L, Motta I, Lourenco S, Gemmo C, Deng W, Rupon J . Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers. Blood. 2016; 128(8):1139-43. PMC: 5000846. DOI: 10.1182/blood-2016-01-691089. View

14.

Sako D, Grinberg A, Liu J, Davies M, Castonguay R, Maniatis S . Characterization of the ligand binding functionality of the extracellular domain of activin receptor type IIb. J Biol Chem. 2010; 285(27):21037-48. PMC: 2898293. DOI: 10.1074/jbc.M110.114959. View

15.

Suragani R, Cadena S, Cawley S, Sako D, Mitchell D, Li R . Transforming growth factor-β superfamily ligand trap ACE-536 corrects anemia by promoting late-stage erythropoiesis. Nat Med. 2014; 20(4):408-14. DOI: 10.1038/nm.3512. View

16.

Suragani R, Cawley S, Li R, Wallner S, Alexander M, Mulivor A . Modified activin receptor IIB ligand trap mitigates ineffective erythropoiesis and disease complications in murine β-thalassemia. Blood. 2014; 123(25):3864-72. PMC: 4064330. DOI: 10.1182/blood-2013-06-511238. View

17.

Katsimpardi L, Rubin L . Young systemic factors as a medicine for age-related neurodegenerative diseases. Neurogenesis (Austin). 2016; 2(1):e1004971. PMC: 4973601. DOI: 10.1080/23262133.2015.1004971. View

18.

Weatherall D . Thalassemia in the next millennium. Keynote address. Ann N Y Acad Sci. 1998; 850:1-9. DOI: 10.1111/j.1749-6632.1998.tb10456.x. View

19.

Egerman M, Cadena S, Gilbert J, Meyer A, Nelson H, Swalley S . GDF11 Increases with Age and Inhibits Skeletal Muscle Regeneration. Cell Metab. 2015; 22(1):164-74. PMC: 4497834. DOI: 10.1016/j.cmet.2015.05.010. View

20.

Katsimpardi L, Litterman N, Schein P, Miller C, Loffredo F, Wojtkiewicz G . Vascular and neurogenic rejuvenation of the aging mouse brain by young systemic factors. Science. 2014; 344(6184):630-4. PMC: 4123747. DOI: 10.1126/science.1251141. View