{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,28]],"date-time":"2026-08-28T14:17:17Z","timestamp":1787926637129,"version":"build-2784847793"},"reference-count":105,"publisher":"MDPI AG","issue":"6","license":[{"start":{"date-parts":[[2022,6,4]],"date-time":"2022-06-04T00:00:00Z","timestamp":1654300800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100003970","name":"Isfahan University of Medical Sciences","doi-asserted-by":"publisher","award":["199306"],"award-info":[{"award-number":["199306"]}],"id":[{"id":"10.13039\/501100003970","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Biology"],"abstract":"<jats:p>Beta (\u03b2)-thalassemia is a group of human inherited abnormalities caused by various molecular defects, which involves a decrease or cessation in the balanced synthesis of the \u03b2-globin chains in hemoglobin structure. Traditional treatment for \u03b2-thalassemia major is allogeneic bone marrow transplantation (BMT) from a completely matched donor. The limited number of human leukocyte antigen (HLA)-matched donors, long-term use of immunosuppressive regimen and higher risk of immunological complications have limited the application of this therapeutic approach. Furthermore, despite improvements in transfusion practices and chelation treatment, many lingering challenges have encouraged researchers to develop newer therapeutic strategies such as nanomedicine and gene editing. One of the most powerful arms of genetic manipulation is gene editing tools, including transcription activator-like effector nucleases, zinc-finger nucleases, and clustered regularly interspaced short palindromic repeat\u2013Cas-associated nucleases. These tools have concentrated on \u03b3- or \u03b2-globin addition, regulating the transcription factors involved in expression of endogenous \u03b3-globin such as KLF1, silencing of \u03b3-globin inhibitors including BCL11A, SOX6, and LRF\/ZBTB7A, and gene repair strategies. In this review article, we present a systematic overview of the appliances of gene editing tools for \u03b2-thalassemia treatment and paving the way for patients\u2019 therapy.<\/jats:p>","DOI":"10.3390\/biology11060862","type":"journal-article","created":{"date-parts":[[2022,6,4]],"date-time":"2022-06-04T09:42:32Z","timestamp":1654335752000},"page":"862","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["Gene Editing-Based Technologies for Beta-hemoglobinopathies Treatment"],"prefix":"10.3390","volume":"11","author":[{"given":"Ilnaz","family":"Rahimmanesh","sequence":"first","affiliation":[{"name":"Applied Physiology Research Center, Cardiovascular Research Institute, Isfahan University of Medical Sciences, Isfahan 73461-81746, Iran"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maryam","family":"Boshtam","sequence":"additional","affiliation":[{"name":"Isfahan Cardiovascular Research Center, Cardiovascular Research Institute, Isfahan University of Medical Sciences, Isfahan 81583-88994, Iran"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shirin","family":"Kouhpayeh","sequence":"additional","affiliation":[{"name":"Erythron Genetics and Pathobiology Laboratory, Department of Immunology, Isfahan 76351-81647, Iran"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hossein","family":"Khanahmad","sequence":"additional","affiliation":[{"name":"Department of Genetics and Molecular Biology, School of Medicine, Isfahan University of Medical Sciences, Isfahan 73461-81746, Iran"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9696-5135","authenticated-orcid":false,"given":"Arezou","family":"Dabiri","sequence":"additional","affiliation":[{"name":"Applied Physiology Research Center, Cardiovascular Research Institute, Isfahan University of Medical Sciences, Isfahan 73461-81746, Iran"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2377-3319","authenticated-orcid":false,"given":"Shahrzad","family":"Ahangarzadeh","sequence":"additional","affiliation":[{"name":"Infectious Diseases and Tropical Medicine Research Center, Isfahan University of Medical Sciences, Isfahan 73461-81746, Iran"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yasaman","family":"Esmaeili","sequence":"additional","affiliation":[{"name":"Biosensor Research Center, School of Advanced Technologies in Medicine, Isfahan University of Medical Sciences, Isfahan 73461-81746, Iran"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6319-3204","authenticated-orcid":false,"given":"Elham","family":"Bidram","sequence":"additional","affiliation":[{"name":"Biosensor Research Center, School of Advanced Technologies in Medicine, Isfahan University of Medical Sciences, Isfahan 73461-81746, Iran"},{"name":"Department of Biomaterials, Nanotechnology and Tissue Engineering, School of Advanced Technologies in Medicine, Isfahan University of Medical Sciences, Isfahan 73461-81746, Iran"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Golnaz","family":"Vaseghi","sequence":"additional","affiliation":[{"name":"Isfahan Cardiovascular Research Center, Cardiovascular Research Institute, Isfahan University of Medical Sciences, Isfahan 81583-88994, Iran"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shaghayegh","family":"Haghjooy Javanmard","sequence":"additional","affiliation":[{"name":"Applied Physiology Research Center, Cardiovascular Research Institute, Isfahan University of Medical Sciences, Isfahan 73461-81746, Iran"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laleh","family":"Shariati","sequence":"additional","affiliation":[{"name":"Department of Biomaterials, Nanotechnology and Tissue Engineering, School of Advanced Technologies in Medicine, Isfahan University of Medical Sciences, Isfahan 73461-81746, Iran"},{"name":"Cancer Prevention Research, Isfahan University of Medical Sciences, Isfahan 73461-81746, Iran"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0391-1769","authenticated-orcid":false,"given":"Ali","family":"Zarrabi","sequence":"additional","affiliation":[{"name":"Department of Biomedical Engineering, Faculty of Engineering and Natural Sciences, Istinye University, Sariyer, Istanbul 34396, Turkey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9731-6228","authenticated-orcid":false,"given":"Rajender S.","family":"Varma","sequence":"additional","affiliation":[{"name":"Regional Centre of Advanced Technologies and Materials, Department of Physical Chemistry, Faculty of Science, Palacky University, \u0160lechtitel\u016f 27, 783 71 Olomouc, Czech Republic"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2022,6,4]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"295","DOI":"10.1089\/hum.2016.037","article-title":"Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects","volume":"27","author":"Riviere","year":"2016","journal-title":"Hum. 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