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International Journal of Hematology-Oncology and Stem Cell Research، جلد ۱۹، شماره ۴، صفحات ۳۹۹-۴۲۳
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عنوان انگلیسی Genetic Engineering in Hematopoietic Stem Cells for β-Hemoglobinopathies Treatment: Advances, Challenges, and Clinical Translation
چکیده انگلیسی مقاله β-hemoglobinopathies rank among the most prevalent inherited blood disorders globally. Traditional management strategies are primarily palliative and often associated with significant challenges, including iron overload and limited long-term efficacy. Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative option for transfusion-dependent patients, but its broader applicability is constrained by factors that limit its use. Utilizing viral vectors and gene-editing tools, particularly CRISPR-Cas9 technology, researchers have developed therapies that target the root causes of these disorders. These innovative approaches have demonstrated substantial therapeutic potential, accompanied by favorable safety profiles, in clinical settings. Since the initial investigations, the genome editing tool has rapidly advanced for genetic abnormalities, particularly monogenic blood diseases, including β-hemoglobinopathies. This method suggests an approach with lower concerns in viral gene integration and insertional mutagenesis issues. This review comprehensively surveys the therapeutic strategies for β-thalassemia and sickle cell disease (SCD) currently in preclinical and clinical development, with a focus on the evolving treatment paradigm. Looking forward, critical research priorities include optimizing the efficiency and specificity of gene-editing platforms and pioneering novel delivery systems to guarantee both therapeutic efficacy and clinical safety.  
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نویسندگان مقاله | Sahar Taebi
Department of Medical Biotechnology, National Institute of Genetic Engineering and Biotechnology, Tehran, Iran


| Fatemeh Eskandari
1) Department of Tissue Engineering and Regenerative Medicine, School of Advanced Technologies in Medicine, Iran University of Medical Sciences (IUMS), Tehran, Iran 2) Cellular and Molecular Research Center, Iran University of Medical Sciences (IUMS), Tehran, Iran


| Masoumeh Kohandani
Department of Cellular and Molecular Biology, Faculty of Biological Sciences, Kharazmi University, Tehran, Iran


| Tahereh Manoochehrabadi
Tissue Engineering Research Group, Department of Anatomy and Cell Biology, School of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran


| Hajar Nasiri
1) Medical Biotechnology Research Center, School of Paramedicine, Guilan University of Medical Sciences, Rasht, Iran 2) Department of Tissue Engineering and Applied Cell Sciences, School of Paramedicine, Guilan University of Medical Sciences, Rasht, Iran
نشانی اینترنتی https://ijhoscr.tums.ac.ir/index.php/ijhoscr/article/view/2344
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