{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T19:57:59Z","timestamp":1792699079691,"version":"4.5.0"},"reference-count":47,"publisher":"Elsevier BV","issue":"10","license":[{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-017"},{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-037"},{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-012"},{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-029"},{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-004"}],"funder":[{"DOI":"10.13039\/501100003399","name":"Science and Technology Commission of Shanghai Municipality","doi-asserted-by":"publisher","award":["23XD1422500"],"award-info":[{"award-number":["23XD1422500"]}],"id":[{"id":"10.13039\/501100003399","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003399","name":"Science and Technology Commission of Shanghai Municipality","doi-asserted-by":"publisher","award":["23YF1407400"],"award-info":[{"award-number":["23YF1407400"]}],"id":[{"id":"10.13039\/501100003399","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003399","name":"Science and Technology Commission of Shanghai Municipality","doi-asserted-by":"publisher","award":["23ZR1442500"],"award-info":[{"award-number":["23ZR1442500"]}],"id":[{"id":"10.13039\/501100003399","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003399","name":"Science and Technology Commission of Shanghai Municipality","doi-asserted-by":"publisher","award":["23DX1900102"],"award-info":[{"award-number":["23DX1900102"]}],"id":[{"id":"10.13039\/501100003399","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003399","name":"Science and Technology Commission of Shanghai Municipality","doi-asserted-by":"publisher","award":["26J32800400"],"award-info":[{"award-number":["26J32800400"]}],"id":[{"id":"10.13039\/501100003399","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003399","name":"Science and Technology Commission of Shanghai Municipality","doi-asserted-by":"publisher","award":["23JS1400300"],"award-info":[{"award-number":["23JS1400300"]}],"id":[{"id":"10.13039\/501100003399","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100002855","name":"Ministry of Science and Technology of the People's Republic of China","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100002855","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003395","name":"Shanghai Municipal Education Commission","doi-asserted-by":"publisher","award":["SMEC"],"award-info":[{"award-number":["SMEC"]}],"id":[{"id":"10.13039\/501100003395","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100008750","name":"Shanghai Municipal Hospital Development Center","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100008750","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100017950","name":"Shanghai Municipal Health Commission","doi-asserted-by":"publisher","award":["SHDC12023109"],"award-info":[{"award-number":["SHDC12023109"]}],"id":[{"id":"10.13039\/100017950","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["32430018"],"award-info":[{"award-number":["32430018"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["32530057"],"award-info":[{"award-number":["32530057"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["32371514"],"award-info":[{"award-number":["32371514"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["32070170"],"award-info":[{"award-number":["32070170"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100012166","name":"National Key Research and Development Program of China","doi-asserted-by":"publisher","award":["2024YFC3405902"],"award-info":[{"award-number":["2024YFC3405902"]}],"id":[{"id":"10.13039\/501100012166","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100012166","name":"National Key Research and Development Program of China","doi-asserted-by":"publisher","award":["2023ZD0500501"],"award-info":[{"award-number":["2023ZD0500501"]}],"id":[{"id":"10.13039\/501100012166","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100012166","name":"National Key Research and Development Program of China","doi-asserted-by":"publisher","award":["2023YFC3403402"],"award-info":[{"award-number":["2023YFC3403402"]}],"id":[{"id":"10.13039\/501100012166","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100012166","name":"National Key Research and Development Program of China","doi-asserted-by":"publisher","award":["2023YFC2706301"],"award-info":[{"award-number":["2023YFC2706301"]}],"id":[{"id":"10.13039\/501100012166","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100012166","name":"National Key Research and Development Program of China","doi-asserted-by":"publisher","award":["2024YFA1803301"],"award-info":[{"award-number":["2024YFA1803301"]}],"id":[{"id":"10.13039\/501100012166","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100013105","name":"Shanghai Institute of Materia Medica Rising-Star Program","doi-asserted-by":"publisher","award":["24QB2707400"],"award-info":[{"award-number":["24QB2707400"]}],"id":[{"id":"10.13039\/501100013105","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Cell Stem Cell"],"published-print":{"date-parts":[[2026,10]]},"DOI":"10.1016\/j.stem.2026.08.009","type":"journal-article","created":{"date-parts":[[2026,9,7]],"date-time":"2026-09-07T15:07:22Z","timestamp":1788793642000},"page":"1597-1609.e4","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":2,"title":["Clinical base editing for \u03b2-hemoglobinopathies across different genetic backgrounds"],"prefix":"10.1016","volume":"33","author":[{"given":"Rongrong","family":"Liu","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yongrong","family":"Lai","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lijie","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaowen","family":"Qian","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xu-Kai","family":"Ma","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yaliang","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gaohui","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lingling","family":"Shi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zifeng","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jia","family":"Wei","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhenbin","family":"Wei","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hongsheng","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xuemei","family":"Zhou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ling","family":"Yu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bei","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaowen","family":"Zhai","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaodun","family":"Mou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Li","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4242-2899","authenticated-orcid":false,"given":"Jia","family":"Chen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.stem.2026.08.009_bib1","doi-asserted-by":"crossref","first-page":"2157","DOI":"10.1182\/blood-2017-10-808550","article-title":"Subphenotypes of sickle cell disease in Africa","volume":"130","author":"Piel","year":"2017","journal-title":"Blood"},{"key":"10.1016\/j.stem.2026.08.009_bib2","doi-asserted-by":"crossref","DOI":"10.1038\/nrdp.2018.10","article-title":"Sickle cell disease","volume":"4","author":"Kato","year":"2018","journal-title":"Nat. Rev. Dis. Primers"},{"key":"10.1016\/j.stem.2026.08.009_bib3","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1001\/jama.2022.10233","article-title":"Sickle Cell Disease: A Review","volume":"328","author":"Kavanagh","year":"2022","journal-title":"JAMA"},{"key":"10.1016\/j.stem.2026.08.009_bib4","doi-asserted-by":"crossref","first-page":"61","DOI":"10.1097\/GIM.0b013e3181cd68ed","article-title":"Beta-thalassemia","volume":"12","author":"Cao","year":"2010","journal-title":"Genet. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib5","doi-asserted-by":"crossref","first-page":"609","DOI":"10.1038\/gim.2016.173","article-title":"beta-Thalassemia","volume":"19","author":"Origa","year":"2017","journal-title":"Genet. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib6","doi-asserted-by":"crossref","first-page":"727","DOI":"10.1056\/NEJMra2021838","article-title":"beta-Thalassemias","volume":"384","author":"Taher","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib7","doi-asserted-by":"crossref","first-page":"344","DOI":"10.1016\/j.molmed.2024.12.001","article-title":"Gene therapy for beta-thalassemia: current and future options","volume":"31","author":"Hardouin","year":"2025","journal-title":"Trends Mol. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib8","doi-asserted-by":"crossref","first-page":"543","DOI":"10.1126\/science.110.2865.543","article-title":"Sickle cell anemia a molecular disease","volume":"110","author":"Pauling","year":"1949","journal-title":"Science"},{"key":"10.1016\/j.stem.2026.08.009_bib9","doi-asserted-by":"crossref","first-page":"792","DOI":"10.1038\/178792a0","article-title":"A specific chemical difference between the globins of normal human and sickle-cell anaemia haemoglobin","volume":"178","author":"Ingram","year":"1956","journal-title":"Nature"},{"key":"10.1016\/j.stem.2026.08.009_bib10","doi-asserted-by":"crossref","first-page":"326","DOI":"10.1038\/180326a0","article-title":"Gene mutations in human haemoglobin: the chemical difference between normal and sickle cell haemoglobin","volume":"180","author":"Ingram","year":"1957","journal-title":"Nature"},{"key":"10.1016\/j.stem.2026.08.009_bib11","doi-asserted-by":"crossref","first-page":"545","DOI":"10.1001\/archinte.1974.00320160039005","article-title":"Ultrastructural features of erythrocyte and hemoglobin sickling","volume":"133","author":"White","year":"1974","journal-title":"Arch. Intern. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib12","doi-asserted-by":"crossref","first-page":"667","DOI":"10.1038\/256667a0","article-title":"Role of hybrid tetramer formation in gelation of haemoglobin S","volume":"256","author":"Bookchin","year":"1975","journal-title":"Nature"},{"key":"10.1016\/j.stem.2026.08.009_bib13","doi-asserted-by":"crossref","first-page":"194","DOI":"10.1038\/300194a0","article-title":"Kinetics of sickle haemoglobin polymerization in single red cells","volume":"300","author":"Coletta","year":"1982","journal-title":"Nature"},{"key":"10.1016\/j.stem.2026.08.009_bib14","doi-asserted-by":"crossref","first-page":"221","DOI":"10.1038\/nm.3814","article-title":"Anemia: progress in molecular mechanisms and therapies","volume":"21","author":"Sankaran","year":"2015","journal-title":"Nat. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib15","doi-asserted-by":"crossref","first-page":"807","DOI":"10.1056\/NEJMoa1103070","article-title":"A functional element necessary for fetal hemoglobin silencing","volume":"365","author":"Sankaran","year":"2011","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib16","doi-asserted-by":"crossref","first-page":"2136","DOI":"10.1182\/blood-2008-09-115238","article-title":"Chaperoning erythropoiesis","volume":"113","author":"Weiss","year":"2009","journal-title":"Blood"},{"key":"10.1016\/j.stem.2026.08.009_bib17","doi-asserted-by":"crossref","first-page":"252","DOI":"10.1056\/NEJMoa2031054","article-title":"CRISPR-Cas9 Gene Editing for Sickle Cell Disease and beta-Thalassemia","volume":"384","author":"Frangoul","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib18","doi-asserted-by":"crossref","first-page":"1649","DOI":"10.1056\/NEJMoa2309676","article-title":"Exagamglogene Autotemcel for Severe Sickle Cell Disease","volume":"390","author":"Frangoul","year":"2024","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib19","doi-asserted-by":"crossref","first-page":"1573","DOI":"10.1038\/s41591-022-01906-z","article-title":"CRISPR-Cas9-mediated gene editing of the BCL11A enhancer for pediatric beta(0)\/beta(0) transfusion-dependent beta-thalassemia","volume":"28","author":"Fu","year":"2022","journal-title":"Nat. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib20","doi-asserted-by":"crossref","first-page":"1663","DOI":"10.1056\/NEJMoa2309673","article-title":"Exagamglogene Autotemcel for Transfusion-Dependent beta-Thalassemia","volume":"390","author":"Locatelli","year":"2024","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib21","doi-asserted-by":"crossref","first-page":"1292","DOI":"10.1056\/NEJMoa2501277","article-title":"CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat beta-Thalassemia","volume":"394","author":"Frangoul","year":"2026","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib22","doi-asserted-by":"crossref","first-page":"1281","DOI":"10.1056\/NEJMoa2415550","article-title":"CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat Sickle Cell Disease","volume":"394","author":"Hanna","year":"2026","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib23","doi-asserted-by":"crossref","first-page":"1338","DOI":"10.1056\/NEJMe2602194","article-title":"More Options for Gene Editing in Hemoglobinopathies","volume":"394","author":"Locatelli","year":"2026","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib24","doi-asserted-by":"crossref","first-page":"927","DOI":"10.1038\/s41591-018-0049-z","article-title":"CRISPR-Cas9 genome editing induces a p53-mediated DNA damage response","volume":"24","author":"Haapaniemi","year":"2018","journal-title":"Nat. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib25","doi-asserted-by":"crossref","first-page":"551","DOI":"10.1016\/j.stem.2019.02.019","article-title":"Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response","volume":"24","author":"Schiroli","year":"2019","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.stem.2026.08.009_bib26","doi-asserted-by":"crossref","first-page":"765","DOI":"10.1038\/nbt.4192","article-title":"Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements","volume":"36","author":"Kosicki","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.stem.2026.08.009_bib27","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-019-09006-2","article-title":"CRISPR-Cas9 genome editing induces megabase-scale chromosomal truncations","volume":"10","author":"Cullot","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.stem.2026.08.009_bib28","doi-asserted-by":"crossref","first-page":"820","DOI":"10.1056\/NEJMoa2215643","article-title":"CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease","volume":"389","author":"Sharma","year":"2023","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib29","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1038\/nature17946","article-title":"Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage","volume":"533","author":"Komor","year":"2016","journal-title":"Nature"},{"key":"10.1016\/j.stem.2026.08.009_bib30","doi-asserted-by":"crossref","first-page":"324","DOI":"10.1038\/nbt.4102","article-title":"Base editing with a Cpf1-cytidine deaminase fusion","volume":"36","author":"Li","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.stem.2026.08.009_bib31","doi-asserted-by":"crossref","first-page":"946","DOI":"10.1038\/nbt.4198","article-title":"Efficient base editing in methylated regions with a human APOBEC3A-Cas9 fusion","volume":"36","author":"Wang","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.stem.2026.08.009_bib32","doi-asserted-by":"crossref","first-page":"874","DOI":"10.1016\/j.tibs.2020.06.003","article-title":"A Tale of Two Moieties: Rapidly Evolving CRISPR\/Cas-Based Genome Editing","volume":"45","author":"Yang","year":"2020","journal-title":"Trends Biochem. Sci."},{"key":"10.1016\/j.stem.2026.08.009_bib33","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1038\/nature24644","article-title":"Programmable base editing of A\u00b7T to G\u00b7C in genomic DNA without DNA cleavage","volume":"551","author":"Gaudelli","year":"2017","journal-title":"Nature"},{"key":"10.1016\/j.stem.2026.08.009_bib34","doi-asserted-by":"crossref","first-page":"1824","DOI":"10.1056\/NEJMoa2504835","article-title":"Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease","volume":"394","author":"Gupta","year":"2026","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib35","doi-asserted-by":"crossref","first-page":"552","DOI":"10.1038\/s41556-021-00671-4","article-title":"Eliminating base-editor-induced genome-wide and transcriptome-wide off-target mutations","volume":"23","author":"Wang","year":"2021","journal-title":"Nat. Cell Biol."},{"key":"10.1016\/j.stem.2026.08.009_bib36","doi-asserted-by":"crossref","first-page":"3194","DOI":"10.1038\/s41596-023-00877-w","article-title":"Design and application of the transformer base editor in mammalian cells and mice","volume":"18","author":"Han","year":"2023","journal-title":"Nat. Protoc."},{"key":"10.1016\/j.stem.2026.08.009_bib37","doi-asserted-by":"crossref","first-page":"1624","DOI":"10.1016\/j.stem.2023.10.007","article-title":"Base editing of the HBG promoter induces potent fetal hemoglobin expression with no detectable off-target mutations in human HSCs","volume":"30","author":"Han","year":"2023","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.stem.2026.08.009_bib38","doi-asserted-by":"crossref","first-page":"923","DOI":"10.1038\/s41586-026-10342-9","article-title":"Clinical application of base editing for treating beta-thalassaemia","volume":"653","author":"Lai","year":"2026","journal-title":"Nature"},{"key":"10.1016\/j.stem.2026.08.009_bib39","doi-asserted-by":"crossref","first-page":"776","DOI":"10.1038\/s41591-019-0401-y","article-title":"Highly efficient therapeutic gene editing of human hematopoietic stem cells","volume":"25","author":"Wu","year":"2019","journal-title":"Nat. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib40","doi-asserted-by":"crossref","first-page":"1473","DOI":"10.1093\/bioinformatics\/btu048","article-title":"Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases","volume":"30","author":"Bae","year":"2014","journal-title":"Bioinformatics"},{"key":"10.1016\/j.stem.2026.08.009_bib41","doi-asserted-by":"crossref","first-page":"475","DOI":"10.1038\/nbt.3852","article-title":"Genome-wide target specificities of CRISPR RNA-guided programmable deaminases","volume":"35","author":"Kim","year":"2017","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.stem.2026.08.009_bib42","doi-asserted-by":"crossref","first-page":"1289","DOI":"10.1038\/cr.2017.111","article-title":"Enhanced base editing by co-expression of free uracil DNA glycosylase inhibitor","volume":"27","author":"Wang","year":"2017","journal-title":"Cell Res."},{"key":"10.1016\/j.stem.2026.08.009_bib43","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.aao4774","article-title":"Improved base excision repair inhibition and bacteriophage Mu Gam protein yields C:G-to-T:A base editors with higher efficiency and product purity","volume":"3","author":"Komor","year":"2017","journal-title":"Sci. Adv."},{"key":"10.1016\/j.stem.2026.08.009_bib44","doi-asserted-by":"crossref","first-page":"91","DOI":"10.1089\/crispr.2019.0001","article-title":"Development and Application of Base Editors","volume":"2","author":"Yang","year":"2019","journal-title":"CRISPR J."},{"key":"10.1016\/j.stem.2026.08.009_bib45","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0017327","article-title":"Homozygous deletion of six olfactory receptor genes in a subset of individuals with Beta-thalassemia","volume":"6","author":"Van Ziffle","year":"2011","journal-title":"PLoS One"},{"key":"10.1016\/j.stem.2026.08.009_bib46","doi-asserted-by":"crossref","DOI":"10.1101\/cshperspect.a011700","article-title":"The molecular basis of beta-thalassemia","volume":"3","author":"Thein","year":"2013","journal-title":"Cold Spring Harb. Perspect. Med."},{"key":"10.1016\/j.stem.2026.08.009_bib47","series-title":"Guidelines for the Management of Non Transfusion Dependent Thalassaemia (NTDT)","author":"Taher","year":"2013"}],"container-title":["Cell Stem Cell"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1934590926003103?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1934590926003103?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T15:14:38Z","timestamp":1790867678000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1934590926003103"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,10]]},"references-count":47,"journal-issue":{"issue":"10","published-print":{"date-parts":[[2026,10]]}},"alternative-id":["S1934590926003103"],"URL":"https:\/\/doi.org\/10.1016\/j.stem.2026.08.009","relation":{},"ISSN":["1934-5909"],"issn-type":[{"value":"1934-5909","type":"print"}],"subject":[],"published":{"date-parts":[[2026,10]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Clinical base editing for \u03b2-hemoglobinopathies across different genetic backgrounds","name":"articletitle","label":"Article Title"},{"value":"Cell Stem Cell","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.stem.2026.08.009","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"https:\/\/doi.org\/10.1016\/j.stem.2026.09.003","name":"associatedlink","label":"CrossRef DOI link to the associated document"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 Elsevier Inc. All rights are reserved, including those for text and data mining, AI training, and similar technologies.","name":"copyright","label":"Copyright"}]}}