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The Role of miRNAs as Therapeutic Tools in Sickle Cell Disease
1Department of Biochemistry, College of Medicine, Imam Abdulrahman Bin Faisal University, Dammam 31141, Saudi Arabia.
Insights
MicroRNAs (miRNAs) show potential in treating sickle cell disorder (SCD) by regulating gene expression and enhancing fetal hemoglobin (HbF) production. These molecular tools offer promising therapeutic strategies for managing SCD severity.
Area of Science:
- Molecular Biology
- Genetics
- Hematology
Background:
- Sickle cell disorder (SCD) is a complex monogenic condition.
- Abnormal hemoglobin polymerization causes erythrocyte susceptibility under deoxygenation.
- MicroRNA (miRNA) dysregulation impacts SCD clinical severity.
Purpose of the Study:
- To review the role of key miRNAs in hemoglobinopathies.
- To explore miRNAs as therapeutic targets for SCD.
- To highlight miRNA-based strategies for enhancing fetal hemoglobin (HbF) synthesis.
Main Methods:
- Literature review focusing on miRNA functions in SCD.
- Analysis of miRNA roles in hematopoiesis and erythroid differentiation.
- Evaluation of miRNA impact on clinical severity and anemia.
Main Results:
- Specific miRNAs influence hematopoiesis, erythroid differentiation, and anemia severity in SCD.
- miRNAs can modulate cell cycle, iron levels, hemolysis, and oxidative stress.
- Dysregulated miRNAs can either worsen or ameliorate SCD phenotypes.
Conclusions:
- miRNAs are attractive molecular tools for innovative SCD therapeutics.
- miRNA mimics and antagomirs show promise for inducing HbF synthesis.
- Targeted miRNA therapies may ameliorate the clinical severity of sickle cell disorder.
Abstract:
Background and Objectives: Sickle cell disorder (SCD) is a paradigmatic example of a complex monogenic disorder. SCD is characterized by the production of abnormal hemoglobin, primarily in the deoxygenated state, which makes erythrocytes susceptible to intracellular hemoglobin polymerization. Functional studies have affirmed that the dysregulation of miRNAs enhances clinical severity or has an ameliorating effect in SCD. miRNAs can be effectively regulated to reduce the pace of cell cycle progression, to reduce iron levels, to influence hemolysis and oxidative stress, and most importantly, to increase γ-globin gene expression and enhance the effectiveness of hydroxyurea. Results: This review highlights the roles played by some key miRNAs in hemoglobinopathies, especially in hematopoiesis, erythroid differentiation, and severity of anemia, which make miRNAs attractive molecular tools for innovative therapeutic approaches. Conclusions: In this era of targeted medicine, miRNAs mimics and antagomirs may be promising inducers of HbF synthesis which could ameliorate the clinical severity of SCD.
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