Gabriela Pereira Dos Santos, Larissa Teodoro Rabi, André Alves Bezerra, Marcelo Rodrigues da Cunha, Amilton Iatecola, Victor Augusto Ramos Fernandes
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Two search strategies were used in the PubMed, SciELO, and LILACS databases between July and August 2023 to conduct this review. Manual searches were also conducted by checking references of potentially eligible studies. Eligibility criteria consisted of clinical trials and cohort studies from the last five years that investigated transcription factors associated with Hb F. The transcription factors investigated in at least four eligible studies were included in this review. As a result, 56 eligible studies provided data on the BCL11A, LRF, NF-Y, GATA1, KLF1, HRI, ATF4, and MYB factors. The studies demonstrated that Hb F is cooperatively regulated by transcription factors with the BCL11A factor appearing to be the most specific target gene for γ-globin induction. Although these data are promising, there are still significant gaps and intervention limitations due to the adverse functions of the target genes. New studies that clarify the aspects and functionalities of Hb F regulators may enable new clinical approaches for sickle cell anemia patients.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":null,"pages":null},"PeriodicalIF":0.0000,"publicationDate":"2024-08-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Transcriptional regulators of fetal hemoglobin.\",\"authors\":\"Gabriela Pereira Dos Santos, Larissa Teodoro Rabi, André Alves Bezerra, Marcelo Rodrigues da Cunha, Amilton Iatecola, Victor Augusto Ramos Fernandes\",\"doi\":\"10.1016/j.htct.2024.06.001\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Sickle cell anemia is a hereditary disease caused by sickle-shaped red blood cells that can lead to vaso-occlusive crises. Treatment options are currently limited, highlighting the need to develop new clinical approaches. 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引用次数: 0
摘要
镰状细胞性贫血是一种遗传性疾病,由镰状红细胞引起,可导致血管闭塞性危象。目前的治疗方案有限,因此需要开发新的临床方法。研究表明,胎儿血红蛋白(Hb F)水平的升高与镰状细胞性贫血患者死亡率和发病率的降低有关。有鉴于此,研究人员一直在努力阐明 Hb F 的转录调控,以开发新的治疗干预措施。本研究旨在介绍 Hb F 的主要转录因子,并讨论这些分子靶点的临床可行性。在 2023 年 7 月至 8 月期间,本研究采用两种检索策略在 PubMed、SciELO 和 LILACS 数据库中进行了综述。此外,还通过查阅可能符合条件的研究的参考文献进行了人工检索。符合条件的研究包括过去五年中调查与 Hb F 相关的转录因子的临床试验和队列研究。因此,56 项符合条件的研究提供了有关 BCL11A、LRF、NF-Y、GATA1、KLF1、HRI、ATF4 和 MYB 因子的数据。这些研究表明,Hb F 受转录因子的协同调控,而 BCL11A 因子似乎是诱导γ-球蛋白的最特异的靶基因。尽管这些数据很有希望,但由于靶基因的不良功能,仍存在很大的差距和干预限制。阐明 Hb F 调节因子的各个方面和功能的新研究可能会为镰状细胞贫血症患者提供新的临床治疗方法。
Sickle cell anemia is a hereditary disease caused by sickle-shaped red blood cells that can lead to vaso-occlusive crises. Treatment options are currently limited, highlighting the need to develop new clinical approaches. Studies demonstrated that elevated levels of fetal hemoglobin (Hb F) are associated with a reduction of mortality and morbidity in sickle cell anemia patients. In light of this, researchers have been trying to elucidate the transcriptional regulation of Hb F to develop new therapeutic interventions. The present study aimed to present the main transcription factors of Hb F and discuss the clinical feasibility of these molecular targets. Two search strategies were used in the PubMed, SciELO, and LILACS databases between July and August 2023 to conduct this review. Manual searches were also conducted by checking references of potentially eligible studies. Eligibility criteria consisted of clinical trials and cohort studies from the last five years that investigated transcription factors associated with Hb F. The transcription factors investigated in at least four eligible studies were included in this review. As a result, 56 eligible studies provided data on the BCL11A, LRF, NF-Y, GATA1, KLF1, HRI, ATF4, and MYB factors. The studies demonstrated that Hb F is cooperatively regulated by transcription factors with the BCL11A factor appearing to be the most specific target gene for γ-globin induction. Although these data are promising, there are still significant gaps and intervention limitations due to the adverse functions of the target genes. New studies that clarify the aspects and functionalities of Hb F regulators may enable new clinical approaches for sickle cell anemia patients.