Repository of Research and Investigative Information

Repository of Research and Investigative Information

Ilam University of Medical Sciences

An Expert Overview on Therapies in Non-Transfusion-Dependent Thalassemia: Classical to Cutting Edge in Treatment

Thu Feb 22 02:00:22 2024

(2023) An Expert Overview on Therapies in Non-Transfusion-Dependent Thalassemia: Classical to Cutting Edge in Treatment. Hemoglobin. pp. 56-70. ISSN 0363-0269

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Abstract

The thalassemia issue is a growing worldwide health concern that anticipates the number of patients suffering from the disease will soon increase significantly. Patients with b-thalassemia intermedia (b-TI) manifest mild to intermediate levels of anemia, which is a reason for it to be clinically located between thalassemia minor and b-thalassemia major (b-TM). Notably, the determination of the actual rate of b-TI is more complicated than b-TM. The leading cause of this illness could be partial repression of b-globin protein production; accordingly, the rate of b-globin gene repression is different in patients, and the gene repression intensity creates a different clinical status. This review article provides an overview of functional mechanisms, advantages, and disadvantages of the classic to latest new treatments for this group of patients, depending on the disease severity divided into the typical management strategies for patients with b-TI such as fetal hemoglobin (Hb) induction, splenectomy, bone marrow transplantation (BMT), transfusion therapy, and herbal and chemical iron chelators. Recently, novel erythropoiesis-stimulating agents have been added. Novel strategies are subclassified into molecular and cellular interventions. Genome editing is one of the efficient molecular therapies for improving hemoglobinopathies, especially b-TI. It encompasses high-fidelity DNA repair (HDR), base and prime editing, clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 procedure, nucleasefree strategies, and epigenetic modulation. In cellular interventions, we mentioned the approach pattern to improve erythropoiesis impairments in translational models and patients with b-TI that involve activin II receptor traps, Janus-associated kinase 2 (JAK2) inhibitors, and iron metabolism regulation.

Item Type: Article
Creators:
CreatorsEmail
Saeidnia, M.UNSPECIFIED
Fazeli, P.UNSPECIFIED
Farzi, A.UNSPECIFIED
Nezhad, M. A.UNSPECIFIED
Shabani-Borujeni, M.UNSPECIFIED
Erfani, M.UNSPECIFIED
Tamaddon, G.UNSPECIFIED
Karimi, M.UNSPECIFIED
Keywords: beta-Thalassemia intermedia (beta-TI) novel pharmacological drugs gene editing clustered regularly interspaced short palindromic repeats (CRISPR)/cas9 liver iron concentration beta-thalassemia extramedullary hematopoiesis ineffective erythropoiesis ligand trap molecular characterization cell transplantation hemoglobin-d d-punjab intermedia Biochemistry & Molecular Biology Hematology
Divisions:
Page Range: pp. 56-70
Journal or Publication Title: Hemoglobin
Journal Index: ISI
Volume: 47
Number: 2
Identification Number: https://doi.org/10.1080/03630269.2022.2158099
ISSN: 0363-0269
Depositing User: مهندس مهدی شریفی
URI: http://eprints.medilam.ac.ir/id/eprint/4446

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