{"id":45098,"date":"2026-04-08T21:07:14","date_gmt":"2026-04-08T21:07:14","guid":{"rendered":"https:\/\/foreignnewstoday.com\/?p=45098"},"modified":"2026-04-08T21:07:15","modified_gmt":"2026-04-08T21:07:15","slug":"clinical-application-of-base-editing-for-treating-%ce%b2-thalassaemia","status":"publish","type":"post","link":"https:\/\/foreignnewstoday.com\/?p=45098","title":{"rendered":"Clinical application of base editing for treating \u03b2-thalassaemia"},"content":{"rendered":"


\n<\/p>\n

\n
  • \n

    Cao, A. & Galanello, R. Beta-thalassemia. Genet. Med.<\/i> 12<\/b>, 61\u201376 (2010).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Origa, R. Beta-thalassemia. Genet. Med.<\/i> 19<\/b>, 609\u2013619 (2017).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Taher, A. T., Musallam, K. M. & Cappellini, M. D. \u03b2-Thalassemias. N. Engl. J. Med.<\/i> 384<\/b>, 727\u2013743 (2021).<\/p>\n

    Article<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Hardouin, G., Miccio, A. & Brusson, M. Gene therapy for \u03b2-thalassemia: current and future options. Trends Mol. Med.<\/i> https:\/\/doi.org\/10.1016\/j.molmed.2024.12.001<\/a> (2025).<\/p>\n<\/li>\n

  • \n

    Wang, L. et al. Eliminating base-editor-induced genome-wide and transcriptome-wide off-target mutations. Nat. Cell Biol.<\/i> 23<\/b>, 552\u2013563 (2021).<\/p>\n

    Article<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Han, W. et al. Design and application of the transformer base editor in mammalian cells and mice. Nat. Protoc.<\/i> 18<\/b>, 3194\u20133228 (2023).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Han, W. et al. Base editing of the HBG<\/i> promoter induces potent fetal hemoglobin expression with no detectable off-target mutations in human HSCs. Cell Stem Cell<\/i> 30<\/b>, 1624\u20131639 (2023).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Galanello, R. & Origa, R. Beta-thalassemia. Orphanet J. Rare Dis.<\/i> 5<\/b>, 11 (2010).<\/p>\n

    Article<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Thein, S. L. The molecular basis of \u03b2-thalassemia. Cold Spring Harb. Perspect. Med.<\/i> 3<\/b>, a011700 (2013).<\/p>\n

    Article<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Cavazzana-Calvo, M. et al. Transfusion independence and HMGA2 activation after gene therapy of human \u03b2-thalassaemia. Nature<\/i> 467<\/b>, 318\u2013322 (2010).<\/p>\n

    Article<\/a>\u00a0
    \n     ADS<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Thompson, A. A. et al. Gene therapy in patients with transfusion-dependent \u03b2-thalassemia. N. Engl. J. Med.<\/i> 378<\/b>, 1479\u20131493 (2018).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Locatelli, F. et al. Betibeglogene autotemcel gene therapy for non-\u03b20<\/sup>\/\u03b20<\/sup> genotype \u03b2-thalassemia. N. Engl. J. Med.<\/i> 386<\/b>, 415\u2013427 (2022).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Li, S. et al. Modified lentiviral globin gene therapy for pediatric \u03b20<\/sup>\/\u03b20<\/sup> transfusion-dependent \u03b2-thalassemia: a single-center, single-arm pilot trial. Cell Stem Cell<\/i> 31<\/b>, 961\u2013973 (2024).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Kwiatkowski, J. L. et al. Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype \u03b2-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial. Lancet<\/i> 404<\/b>, 2175\u20132186 (2024).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Bauer, D. E. et al. An erythroid enhancer of BCL11A subject to genetic variation determines fetal hemoglobin level. Science<\/i> 342<\/b>, 253\u2013257 (2013).<\/p>\n

    Article<\/a>\u00a0
    \n     ADS<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Canver, M. C. et al. BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis. Nature<\/i> 527<\/b>, 192\u2013197 (2015).<\/p>\n

    Article<\/a>\u00a0
    \n     ADS<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Traxler, E. A. et al. A genome-editing strategy to treat \u03b2-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition. Nat. Med.<\/i> 22<\/b>, 987\u2013990 (2016).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Musallam, K. M. et al. Fetal hemoglobin levels and morbidity in untransfused patients with \u03b2-thalassemia intermedia. Blood<\/i> 119<\/b>, 364\u2013367 (2012).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Bauer, D. E. & Orkin, S. H. Hemoglobin switching\u2019s surprise: the versatile transcription factor BCL11A is a master repressor of fetal hemoglobin. Curr. Opin. Genet. Dev.<\/i> 33<\/b>, 62\u201370 (2015).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Canver, M. C. & Orkin, S. H. Customizing the genome as therapy for the \u03b2-hemoglobinopathies. Blood<\/i> 127<\/b>, 2536\u20132545 (2016).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Wienert, B., Martyn, G. E., Funnell, A. P. W., Quinlan, K. G. R. & Crossley, M. Wake-up sleepy gene: reactivating fetal globin for \u03b2-hemoglobinopathies. Trends Genet.<\/i> 34<\/b>, 927\u2013940 (2018).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Sankaran, V. G. et al. Human fetal hemoglobin expression is regulated by the developmental stage-specific repressor BCL11A<\/i>. Science<\/i> 322<\/b>, 1839\u20131842 (2008).<\/p>\n

    Article<\/a>\u00a0
    \n     ADS<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Wu, Y. et al. Highly efficient therapeutic gene editing of human hematopoietic stem cells. Nat. Med.<\/i> 25<\/b>, 776\u2013783 (2019).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Zeng, J. et al. Therapeutic base editing of human hematopoietic stem cells. Nat. Med.<\/i> 26<\/b>, 535\u2013541 (2020).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Frangoul, H. et al. CRISPR\u2013Cas9 gene editing for sickle cell disease and \u03b2-thalassemia. N. Engl. J. Med.<\/i> 384<\/b>, 252\u2013260 (2021).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Fu, B. et al. CRISPR\u2013Cas9-mediated gene editing of the BCL11A<\/i> enhancer for pediatric \u03b20<\/sup>\/\u03b20<\/sup> transfusion-dependent \u03b2-thalassemia. Nat. Med.<\/i> 28<\/b>, 1573\u20131580 (2022).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Locatelli, F. et al. Exagamglogene autotemcel for transfusion-dependent \u03b2-thalassemia. N. Engl. J. Med.<\/i> 390<\/b>, 1663\u20131676 (2024).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Haapaniemi, E., Botla, S., Persson, J., Schmierer, B. & Taipale, J. CRISPR\u2013Cas9 genome editing induces a p53-mediated DNA damage response. Nat. Med.<\/i> 24<\/b>, 927\u2013930 (2018).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Schiroli, G. et al. Precise gene editing preserves hematopoietic stem cell function following transient p53-mediated DNA damage response. Cell Stem Cell<\/i> 24<\/b>, 551\u2013565 (2019).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Kosicki, M., Tomberg, K. & Bradley, A. Repair of double-strand breaks induced by CRISPR\u2013Cas9 leads to large deletions and complex rearrangements. Nat. Biotechnol.<\/i> 36<\/b>, 765\u2013771 (2018).<\/p>\n

    Article<\/a>\u00a0
    \n     ADS<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Cullot, G. et al. CRISPR\u2013Cas9 genome editing induces megabase-scale chromosomal truncations. Nat. Commun.<\/i> 10<\/b>, 1136 (2019).<\/p>\n

    Article<\/a>\u00a0
    \n     ADS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Tsuchida, C. A. et al. Mitigation of chromosome loss in clinical CRISPR\u2013Cas9-engineered T cells. Cell<\/i> 186<\/b>, 4567\u20134582 (2023).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Komor, A. C., Kim, Y. B., Packer, M. S., Zuris, J. A. & Liu, D. R. Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nature<\/i> 533<\/b>, 420\u2013424 (2016).<\/p>\n

    Article<\/a>\u00a0
    \n     ADS<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Gaudelli, N. M. et al. Programmable base editing of A*T to G*C in genomic DNA without DNA cleavage. Nature<\/i> 551<\/b>, 464\u2013471 (2017).<\/p>\n

    Article<\/a>\u00a0
    \n     ADS<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Li, X. et al. Base editing with a Cpf1\u2013cytidine deaminase fusion. Nat. Biotechnol.<\/i> 36<\/b>, 324\u2013327 (2018).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Lei, L. et al. APOBEC3 induces mutations during repair of CRISPR\u2013Cas9-generated DNA breaks. Nat. Struct. Mol. Biol.<\/i> 25<\/b>, 45\u201352 (2018).<\/p>\n

    Article<\/a>\u00a0
    \n     ADS<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Anzalone, A. V., Koblan, L. W. & Liu, D. R. Genome editing with CRISPR\u2013Cas nucleases, base editors, transposases and prime editors. Nat. Biotechnol.<\/i> 38<\/b>, 824\u2013844 (2020).<\/p>\n

    Article<\/a>\u00a0
    \n     ADS<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Yang, L. & Chen, J. A tale of two moieties: rapidly evolving CRISPR\/Cas-based genome editing. Trends Biochem. Sci.<\/i> 45<\/b>, 874\u2013888 (2020).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Kim, J. S. & Chen, J. Base editing of organellar DNA with programmable deaminases. Nat. Rev. Mol. Cell Biol.<\/i> 25<\/b>, 34\u201345 (2024).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Karimian, A., Ahmadi, Y. & Yousefi, B. Multiple functions of p21 in cell cycle, apoptosis and transcriptional regulation after DNA damage. DNA Repair<\/i> 42<\/b>, 63\u201371 (2016).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Bae, S., Park, J. & Kim, J. S. Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases. Bioinformatics<\/i> 30<\/b>, 1473\u20131475 (2014).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Kim, D. et al. Genome-wide target specificities of CRISPR RNA-guided programmable deaminases. Nat. Biotechnol.<\/i> 35<\/b>, 475\u2013480 (2017).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Wang, X. et al. Efficient base editing in methylated regions with a human APOBEC3A\u2013Cas9 fusion. Nat. Biotechnol.<\/i> 36<\/b>, 946\u2013949 (2018).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Mayuranathan, T. et al. Potent and uniform fetal hemoglobin induction via base editing. Nat. Genet.<\/i> 55<\/b>, 1210\u20131220 (2023).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Liao, J. et al. Therapeutic adenine base editing of human hematopoietic stem cells. Nat. Commun.<\/i> 14<\/b>, 207 (2023).<\/p>\n

    Article<\/a>\u00a0
    \n     ADS<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Cradick, T. J., Qiu, P., Lee, C. M., Fine, E. J. & Bao, G. COSMID: a web-based tool for identifying and validating CRISPR\/Cas off-target sites. Mol. Ther. Nucleic Acids<\/i> 3<\/b>, e214 (2014).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Cromer, M. K. et al. Comparative analysis of CRISPR off-target discovery tools following ex vivo editing of CD34+<\/sup> hematopoietic stem and progenitor cells. Mol. Ther.<\/i> 31<\/b>, 1074\u20131087 (2023).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Wang, L. et al. Enhanced base editing by co-expression of free uracil DNA glycosylase inhibitor. Cell Res.<\/i> 27<\/b>, 1289\u20131292 (2017).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Komor, A. C. et al. Improved base excision repair inhibition and bacteriophage Mu Gam protein yields C:G-to-T:A base editors with higher efficiency and product purity. Sci. Adv.<\/i> 3<\/b>, eaao4774 (2017).<\/p>\n

    Article<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Yang, B., Yang, L. & Chen, J. Development and application of base editors. CRISPR J.<\/i> 2<\/b>, 91\u2013104 (2019).<\/p>\n

    Article<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Taher, A., Vichinsky, E., Musallam, K., Cappellini, M. D. & Viprakasit, V. in Guidelines for the Management of Non Transfusion Dependent Thalassaemia (NTDT)<\/i> (ed. Weatherall, D.) (Thalassaemia International Federation, 2013).<\/p>\n<\/li>\n

  • \n

    Kirk, P. et al. International reproducibility of single breathhold T2* MR for cardiac and liver iron assessment among five thalassemia centers. J. Magn. Reson. Imaging<\/i> 32<\/b>, 315\u2013319 (2010).<\/p>\n

    Article<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Sharma, A. et al. CRISPR\u2013Cas9 editing of the HBG1<\/i> and HBG2<\/i> promoters to treat sickle cell disease. N. Engl. J. Med.<\/i> 389<\/b>, 820\u2013832 (2023).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Frangoul, H. et al. Exagamglogene autotemcel for severe sickle cell disease. N. Engl. J. Med.<\/i> 390<\/b>, 1649\u20131662 (2024).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Fucharoen, S., Shimizu, K. & Fukumaki, Y. A novel C-T transition within the distal CCAAT motif of the G\u03b3-globin gene in the Japanese HPFH: implication of factor binding in elevated fetal globin expression. Nucleic Acids Res.<\/i> 18<\/b>, 5245\u20135253 (1990).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Oner, R., Kutlar, F., Gu, L. H. & Huisman, T. H. The Georgia type of nondeletional hereditary persistence of fetal hemoglobin has a C\u2192T mutation at nucleotide-114 of the A<\/sup>\u03b3-globin gene. Blood<\/i> 77<\/b>, 1124\u20131125 (1991).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Motum, P. I., Deng, Z. M., Huong, L. & Trent, R. J. The Australian type of nondeletional G<\/sup>\u03b3-HPFH has a C\u2192G substitution at nucleotide -114 of the G<\/sup>\u03b3 gene. Br. J. Haematol.<\/i> 86<\/b>, 219\u2013221 (1994).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Zertal-Zidani, S. et al. A novel C\u2192A transversion within the distal CCAAT motif of the G<\/sup>\u03b3-globin gene in the Algerian G<\/sup>\u03b3\u03b2+<\/sup>-hereditary persistence of fetal hemoglobin. Hemoglobin<\/i> 23<\/b>, 159\u2013169 (1999).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Ribeil, J. A. et al. Ineffective erythropoiesis in \u03b2-thalassemia. Sci. World J.<\/i> 2013<\/b>, 394295 (2013).<\/p>\n

    Article<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Cancellieri, S. et al. Human genetic diversity alters off-target outcomes of therapeutic gene editing. Nat. Genet.<\/i> 55<\/b>, 34\u201343 (2023).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Breda, L. et al. In vivo hematopoietic stem cell modification by mRNA delivery. Science<\/i> 381<\/b>, 436\u2013443 (2023).<\/p>\n

    Article<\/a>\u00a0
    \n     ADS<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n     PubMed Central<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Wognum, B., Yuan, N., Lai, B. & Miller, C. L. Colony forming cell assays for human hematopoietic progenitor cells. Methods Mol. Biol.<\/i> 946<\/b>, 267\u2013283 (2013).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Mathias, L. A. et al. Ineffective erythropoiesis in \u03b2-thalassemia major is due to apoptosis at the polychromatophilic normoblast stage. Exp. Hematol.<\/i> 28<\/b>, 1343\u20131353 (2000).<\/p>\n

    Article<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n

  • \n

    Arlet, J. B. et al. HSP70 sequestration by free \u03b1-globin promotes ineffective erythropoiesis in \u03b2-thalassaemia. Nature<\/i> 514<\/b>, 242\u2013246 (2014).<\/p>\n

    Article<\/a>\u00a0
    \n     ADS<\/a>\u00a0
    \n     CAS<\/a>\u00a0
    \n     PubMed<\/a>\u00a0
    \n
    \n Google Scholar<\/a>\u00a0\n <\/p>\n<\/li>\n<\/div>\n


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