{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,21]],"date-time":"2026-04-21T04:18:52Z","timestamp":1776745132951,"version":"3.51.2"},"reference-count":17,"publisher":"American Society of Hematology","issue":"11","license":[{"start":{"date-parts":[[2024,1,30]],"date-time":"2024-01-30T00:00:00Z","timestamp":1706572800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2024,3,14]]},"abstract":"<jats:sec>\n                  <jats:title>Abstract<\/jats:title>\n                  <jats:p>The root cause of sickle cell anemia has been known for 7 decades, yet no curative therapies have been available other than allogeneic bone marrow transplantation, for which applicability is limited. Two potentially curative therapies based on gene therapy and gene editing strategies have recently received US Food and Drug Administration approval. This review surveys the nature of these therapies and the opportunities and issues raised by the prospect of definitive genetically based therapies being available in clinical practice.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1182\/blood.2023021598","type":"journal-article","created":{"date-parts":[[2024,1,30]],"date-time":"2024-01-30T15:01:35Z","timestamp":1706626895000},"page":"967-970","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":9,"title":["\u201cTreatment with curative intent\u201d: the emergence of genetic therapies for sickle cell anemia"],"prefix":"10.1182","volume":"143","author":[{"suffix":"Jr.","given":"Edward J.","family":"Benz","sequence":"first","affiliation":[{"name":"1Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA"},{"name":"4Department of Medicine, Brigham and Women\u2019s Hospital, Harvard Medical School, Boston, MA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Leslie E.","family":"Silberstein","sequence":"additional","affiliation":[{"name":"2Department of Pediatrics, Center for Joint Transfusion Medicine, Boston Children\u2019s Hospital, Brigham and Women\u2019s Hospital, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Julie","family":"Panepinto","sequence":"additional","affiliation":[{"name":"3Division of Blood Diseases and Blood Resources, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"234","reference":[{"issue":"2","key":"2024031415212752700_bib1","doi-asserted-by":"crossref","first-page":"83","DOI":"10.1002\/ajmg.10542","article-title":"Linus Pauling's \"molecular diseases\": between history and memory","volume":"115","author":"Strasser","year":"2002","journal-title":"Am J Med Genet"},{"issue":"4581","key":"2024031415212752700_bib2","doi-asserted-by":"crossref","first-page":"326","DOI":"10.1038\/180326a0","article-title":"Gene mutation in human haemoglobin: the chemical difference between normal and sickle cell haemoglobin","volume":"180","author":"Ingram","year":"1957","journal-title":"Nature"},{"key":"2024031415212752700_bib3","first-page":"585","volume-title":"Hematology: Principles and Practice","author":"Hebbel","year":"2023","edition":"8th ed."},{"key":"2024031415212752700_bib4","first-page":"599","volume-title":"Hematology: Principles and Practice","author":"Menapace","year":"2023","edition":"8th ed."},{"issue":"7","key":"2024031415212752700_bib5","doi-asserted-by":"crossref","first-page":"617","DOI":"10.1056\/NEJMoa2117175","article-title":"Biologic and clinical efficacy of LentiGLobin for sickle cell disease","volume":"386","author":"Kanter","year":"2022","journal-title":"N\u00a0Engl J Med"},{"issue":"7","key":"2024031415212752700_bib6","doi-asserted-by":"crossref","first-page":"689","DOI":"10.1056\/NEJMe2119760","article-title":"Fetal-like hemoglobin in sickle cell anemia","volume":"386","author":"Steinberg","year":"2022","journal-title":"N\u00a0Engl J Med"},{"issue":"6058","key":"2024031415212752700_bib7","doi-asserted-by":"crossref","first-page":"993","DOI":"10.1126\/science.1211053","article-title":"Correction of sickle cell disease in adult mice by interference with fetal hemoglobin silencing","volume":"334","author":"Xu","year":"2011","journal-title":"Science"},{"issue":"6155","key":"2024031415212752700_bib8","doi-asserted-by":"crossref","first-page":"253","DOI":"10.1126\/science.1242088","article-title":"An erythroid enhancer of BCL11A subject to genetic variation determines fetal hemoglobin level","volume":"342","author":"Bauer","year":"2013","journal-title":"Science"},{"issue":"3","key":"2024031415212752700_bib9","doi-asserted-by":"crossref","first-page":"252","DOI":"10.1056\/NEJMoa2031054","article-title":"CRISPR_Cas9 gene editing for sickle cell anemia and \u03b2-thalassemia","volume":"384","author":"Frangoul","year":"2021","journal-title":"N\u00a0Engl J Med"},{"key":"2024031415212752700_bib10","unstructured":"Stanton\n              D\n            \n          . 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