Erythropoiesis: insights into pathophysiology and treatments in 2017
Abstract Erythropoiesis is a tightly-regulated and complex process originating in the bone marrow from a multipotent stem cell and terminating in a mature, enucleated erythrocyte. Altered red cell production can result from the direct impairment of medullary erythropoiesis, as seen in the thalassemi...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
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BMC
2018-03-01
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| Series: | Molecular Medicine |
| Subjects: | |
| Online Access: | http://link.springer.com/article/10.1186/s10020-018-0011-z |
| _version_ | 1811213201284530176 |
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| author | Andrea Zivot Jeffrey M. Lipton Anupama Narla Lionel Blanc |
| author_facet | Andrea Zivot Jeffrey M. Lipton Anupama Narla Lionel Blanc |
| author_sort | Andrea Zivot |
| collection | DOAJ |
| description | Abstract Erythropoiesis is a tightly-regulated and complex process originating in the bone marrow from a multipotent stem cell and terminating in a mature, enucleated erythrocyte. Altered red cell production can result from the direct impairment of medullary erythropoiesis, as seen in the thalassemia syndromes, inherited bone marrow failure as well as in the anemia of chronic disease. Alternatively, in disorders such as sickle cell disease (SCD) as well as enzymopathies and membrane defects, medullary erythropoiesis is not, or only minimally, directly impaired. Despite these differences in pathophysiology, therapies have traditionally been non-specific, limited to symptomatic control of anemia via packed red blood cell (pRBC) transfusion, resulting in iron overload and the eventual need for iron chelation or splenectomy to reduce defective red cell destruction. Likewise, in polycythemia vera overproduction of red cells has historically been dealt with by non-specific myelosuppression or phlebotomy. With a deeper understanding of the molecular mechanisms underlying disease pathophysiology, new therapeutic targets have been identified including induction of fetal hemoglobin, interference with aberrant signaling pathways and gene therapy for definitive cure. This review, utilizing some representative disorders of erythropoiesis, will highlight novel therapeutic modalities currently in development for treatment of red cell disorders. |
| first_indexed | 2024-04-12T05:42:55Z |
| format | Article |
| id | doaj.art-39bd3718db0848f48345348128bdc362 |
| institution | Directory Open Access Journal |
| issn | 1076-1551 1528-3658 |
| language | English |
| last_indexed | 2024-04-12T05:42:55Z |
| publishDate | 2018-03-01 |
| publisher | BMC |
| record_format | Article |
| series | Molecular Medicine |
| spelling | doaj.art-39bd3718db0848f48345348128bdc3622022-12-22T03:45:34ZengBMCMolecular Medicine1076-15511528-36582018-03-0124111510.1186/s10020-018-0011-zErythropoiesis: insights into pathophysiology and treatments in 2017Andrea Zivot0Jeffrey M. Lipton1Anupama Narla2Lionel Blanc3Laboratory of Developmental Erythropoiesis, Center for Autoimmune, Musculoskeletal, and Hematopoietic Diseases, The Feinstein Institute for Medical ResearchLaboratory of Developmental Erythropoiesis, Center for Autoimmune, Musculoskeletal, and Hematopoietic Diseases, The Feinstein Institute for Medical ResearchDepartment of Molecular Medicine and Pediatrics, Donald and Barbara Zucker School of Medicine at Hofstra NorthwellLaboratory of Developmental Erythropoiesis, Center for Autoimmune, Musculoskeletal, and Hematopoietic Diseases, The Feinstein Institute for Medical ResearchAbstract Erythropoiesis is a tightly-regulated and complex process originating in the bone marrow from a multipotent stem cell and terminating in a mature, enucleated erythrocyte. Altered red cell production can result from the direct impairment of medullary erythropoiesis, as seen in the thalassemia syndromes, inherited bone marrow failure as well as in the anemia of chronic disease. Alternatively, in disorders such as sickle cell disease (SCD) as well as enzymopathies and membrane defects, medullary erythropoiesis is not, or only minimally, directly impaired. Despite these differences in pathophysiology, therapies have traditionally been non-specific, limited to symptomatic control of anemia via packed red blood cell (pRBC) transfusion, resulting in iron overload and the eventual need for iron chelation or splenectomy to reduce defective red cell destruction. Likewise, in polycythemia vera overproduction of red cells has historically been dealt with by non-specific myelosuppression or phlebotomy. With a deeper understanding of the molecular mechanisms underlying disease pathophysiology, new therapeutic targets have been identified including induction of fetal hemoglobin, interference with aberrant signaling pathways and gene therapy for definitive cure. This review, utilizing some representative disorders of erythropoiesis, will highlight novel therapeutic modalities currently in development for treatment of red cell disorders.http://link.springer.com/article/10.1186/s10020-018-0011-zErythropoiesisTherapyRed cell disorders |
| spellingShingle | Andrea Zivot Jeffrey M. Lipton Anupama Narla Lionel Blanc Erythropoiesis: insights into pathophysiology and treatments in 2017 Molecular Medicine Erythropoiesis Therapy Red cell disorders |
| title | Erythropoiesis: insights into pathophysiology and treatments in 2017 |
| title_full | Erythropoiesis: insights into pathophysiology and treatments in 2017 |
| title_fullStr | Erythropoiesis: insights into pathophysiology and treatments in 2017 |
| title_full_unstemmed | Erythropoiesis: insights into pathophysiology and treatments in 2017 |
| title_short | Erythropoiesis: insights into pathophysiology and treatments in 2017 |
| title_sort | erythropoiesis insights into pathophysiology and treatments in 2017 |
| topic | Erythropoiesis Therapy Red cell disorders |
| url | http://link.springer.com/article/10.1186/s10020-018-0011-z |
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