Gene editing-based technologies for beta-hemoglobinopathies treatment

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dc.authoridAli Zarrabi / 0000-0003-0391-1769en_US
dc.authorscopusidAli Zarrabi / 23483174100
dc.authorwosidAli Zarrabi / U-2602-2019en_US
dc.contributor.authorRahimmanesh, Ilnaz
dc.contributor.authorBoshtam, Maryam
dc.contributor.authorKouhpayeh, Shirin
dc.contributor.authorKhanahmad, Hossein
dc.contributor.authorDabiri, Arezou
dc.contributor.authorAhangarzadeh, Shahrzad
dc.contributor.authorEsmaeili, Yasaman
dc.contributor.authorBidram, Elham
dc.contributor.authorVaseghi, Golnaz
dc.contributor.authorHaghjooy, Shaghayegh
dc.contributor.authorShariati, Laleh
dc.contributor.authorZarrabi, Ali
dc.contributor.authorVarma, Rajender S.
dc.date.accessioned2022-06-28T07:45:38Z
dc.date.available2022-06-28T07:45:38Z
dc.date.issued2022en_US
dc.departmentİstinye Üniversitesi, Mühendislik ve Doğa Bilimleri Fakültesi, Biyomedikal Mühendisliği Bölümüen_US
dc.description.abstractSimple Summary: ?-thalassemia syndromes are clinically and genetically heterogeneous blood disorders presented by ?-chain deficiency in hemoglobin production. Despite improvements in transfusion practices and chelation treatment, many lingering challenges have encouraged researchers to develop newer therapeutic strategies such as gene editing. One of the most powerful arms of genetic manipulation is gene editing tools, which have been recently applied to improve ?-thalassemia symptoms. Nevertheless, several obstacles, such as off-target effects, protospaceradjacent motif requirement, efficient gene transfer and expression methods, DNA-damage toxicity, and immunotoxicity issues still need to be addressed in order to improve the safety and efficacy of the gene editing approaches. Hence, additional efforts are needed to address these problems, evaluate the safety of genome editing tools at the clinical level and follow the outcomes of gene editing tools-mediated therapeutic approaches in related patients. Abstract: Beta (?)-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 ?-globin chains in hemoglobin structure. Traditional treatment for ?-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–Cas-associated nucleases. These tools have concentrated on ?- or ?-globin addition, regulating the transcription factors involved in expression of endogenous ?-globin such as KLF1, silencing of ?-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 ?-thalassemia treatment and paving the way for patients’ therapy.en_US
dc.identifier.citationRahimmanesh I, Boshtam M, Kouhpayeh S, Khanahmad H, Dabiri A, Ahangarzadeh S, Esmaeili Y, Bidram E, Vaseghi G, Haghjooy Javanmard S, Shariati L, Zarrabi A, Varma RS. Gene Editing-Based Technologies for Beta-hemoglobinopathies Treatment. Biology (Basel). 2022 Jun 4;11(6):862. doi: 10.3390/biology11060862. PMID: 35741383; PMCID: PMC9219845.en_US
dc.identifier.doi10.3390/biology11060862en_US
dc.identifier.issn2079-7737en_US
dc.identifier.pmid9219845en_US
dc.identifier.scopus2-s2.0-85131857566en_US
dc.identifier.scopusqualityQ1en_US
dc.identifier.urihttp://doi.org/10.3390/biology11060862
dc.identifier.urihttps://hdl.handle.net/20.500.12713/2951
dc.identifier.wosWOS:000816280400001en_US
dc.identifier.wosqualityQ2en_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakScopusen_US
dc.indekslendigikaynakPubMeden_US
dc.institutionauthorZarrabi, Ali
dc.language.isoenen_US
dc.publisherMDPIen_US
dc.relation.ispartofBiologyen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/embargoedAccessen_US
dc.subjectBeta-Thalassemiaen_US
dc.subjectGene Therapyen_US
dc.subjectZFNen_US
dc.subjectTALENen_US
dc.subjectCRISPRen_US
dc.titleGene editing-based technologies for beta-hemoglobinopathies treatmenten_US
dc.typeReview Articleen_US

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