{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T03:15:13Z","timestamp":1791342913389,"version":"4.3.1"},"reference-count":73,"publisher":"American Society of Hematology","issue":"20","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2025,11,13]]},"abstract":"<jats:sec>\n                    <jats:title>Abstract<\/jats:title>\n                    <jats:p>Two gene therapy products have been approved by the US Food and Drug Administration for sickle cell disease. Nearly all patients in the clinical trials that led to approval either were sickle hemoglobin (HbS) gene homozygotes (sickle cell anemia) or had HbS-\u03b20 thalassemia. HbSC disease, caused by compound heterozygosity for HbS and hemoglobin C genes, is the second most common genotype of sickle cell disease. Gene therapy has not been tested in patients with HbSC disease who are severely symptomatic. We discuss the pathophysiology and clinical features of HbSC disease and how gene therapy is likely to provide a curative option for some individuals. We also discuss the mechanism through which fetal hemoglobin (HbF) and HbF-like HbA (HbAT87Q) might mitigate adverse clinical outcomes and end-organ damage in patients with HbSC disease and other compound heterozygous sickle hemoglobinopathies.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1182\/blood.2025029964","type":"journal-article","created":{"date-parts":[[2025,8,20]],"date-time":"2025-08-20T22:56:07Z","timestamp":1755730567000},"page":"2385-2391","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":3,"title":["Gene therapy for HbSC disease and other compound heterozygous sickle hemoglobinopathies: a time for inclusion"],"prefix":"10.1182","volume":"146","author":[{"given":"Andrew","family":"Wilks","sequence":"first","affiliation":[{"name":"1Division of Hematology and Medical Oncology, Department of Medicine, Center of Excellence for Sickle Cell Disease, Boston University Chobanian & Avedisian School of Medicine and Boston Medical Center, Boston, MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8800-8020","authenticated-orcid":false,"given":"Martin H.","family":"Steinberg","sequence":"additional","affiliation":[{"name":"1Division of Hematology and Medical Oncology, Department of Medicine, Center of Excellence for Sickle Cell Disease, Boston University Chobanian & Avedisian School of Medicine and Boston Medical Center, Boston, MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Haydar","family":"Frangoul","sequence":"additional","affiliation":[{"name":"2Sarah Cannon Cancer Insitute, Pediatric Hematology\/Oncology and Cellular Therapy, TriStar Centennial Medical Center, Nashville, TN"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"234","reference":[{"issue":"18","key":"2025111311213002900_bib1","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"},{"issue":"7","key":"2025111311213002900_bib2","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 Engl J Med"},{"key":"2025111311213002900_bib3","doi-asserted-by":"crossref","DOI":"10.1017\/CBO9780511596582","volume-title":"Disorders of hemoglobin: Genetics, Pathophysiology, and Clinical Management","author":"Steinberg","year":"2009","edition":"10th ed"},{"issue":"5","key":"2025111311213002900_bib4","doi-asserted-by":"crossref","first-page":"356","DOI":"10.1080\/03630269.2016.1222295","article-title":"Phenotypic diversity of sickle cell disease in patients with a double heterozygosity for Hb S and HbD-Punjab","volume":"40","author":"Torres","year":"2016","journal-title":"Hemoglobin"},{"issue":"26","key":"2025111311213002900_bib5","doi-asserted-by":"crossref","first-page":"1417","DOI":"10.1056\/NEJM197012242832601","article-title":"Hemoglobin O Arab in four negro families and its interaction with hemoglobin S and hemoglobin C","volume":"283","author":"Milner","year":"1970","journal-title":"N Engl J Med"},{"issue":"8","key":"2025111311213002900_bib6","doi-asserted-by":"crossref","first-page":"e775","DOI":"10.1097\/MPH.0000000000001882","article-title":"Hemoglobin S\/Hemoglobin Quebec-Chori presenting as sickle cell disease: a case report","volume":"42","author":"Goode","year":"2020","journal-title":"J Pediatr Hematol Oncol"},{"issue":"3","key":"2025111311213002900_bib7","doi-asserted-by":"crossref","first-page":"344","DOI":"10.18295\/2075-0528.2746","article-title":"Compound heterozygosity for Hb S and Hb S-Oman: case report","volume":"8","author":"Venugopal","year":"2008","journal-title":"Sultan Qaboos Univ Med J"},{"issue":"11","key":"2025111311213002900_bib8","doi-asserted-by":"crossref","first-page":"773","DOI":"10.1097\/00043426-200411000-00019","article-title":"A novel sickle hemoglobin: hemoglobin S-South end","volume":"26","author":"Luo","year":"2004","journal-title":"J Pediatr Hematol Oncol"},{"issue":"3","key":"2025111311213002900_bib9","doi-asserted-by":"crossref","first-page":"363","DOI":"10.1182\/blood.V46.3.363.363","article-title":"The presence of hemoglobin S and C Harlem in an individual in the United States","volume":"46","author":"Moo-Penn","year":"1975","journal-title":"Blood"},{"issue":"24","key":"2025111311213002900_bib10","doi-asserted-by":"crossref","first-page":"9363","DOI":"10.1073\/pnas.83.24.9363","article-title":"Hemoglobin S Antilles: a variant with lower solubility than hemoglobin S and producing sickle cell disease in heterozygotes","volume":"83","author":"Monplaisir","year":"1986","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"15","key":"2025111311213002900_bib11","doi-asserted-by":"crossref","first-page":"1532","DOI":"10.1056\/NEJMoa040771","article-title":"Hemoglobin Jamaica plain-a sickling hemoglobin with reduced oxygen affinity","volume":"351","author":"Geva","year":"2004","journal-title":"N Engl J Med"},{"key":"2025111311213002900_bib12","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1016\/0014-5793(75)80246-8","article-title":"Hemoglobin C Ziguinchor \u03b12A\u03b226 (A3) glu-&gt;val \u03b258 (E2) pro-&gt;arg: the second sickling variant with amino acid substitutions in 2 residues of the \u03b2 polypeptide chain","volume":"58","author":"Goosens","year":"1975","journal-title":"FEBS Let"},{"key":"2025111311213002900_bib13","first-page":"33","article-title":"A new major sickle cell syndrome: HbS (\u03b26Val)\/HbC Ndjamena (\u03b26Val-\u03b237Gly)","volume":"30","author":"Ducrocq","year":"2006","journal-title":"Hemoglobin"},{"issue":"1","key":"2025111311213002900_bib14","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1081\/HEM-120002936","article-title":"Hb O-Tibesti [beta121(GH4)Glu-&gt;Lys; beta11(A8)Val-&gt;Ile], a hemoglobin variant carrying in the same beta chain the substitutions of Hb O-Arab and Hb Hamilton, found in combination with Hb S [beta6(A3)Glu--&gt;Val]","volume":"26","author":"Pr\u00e9hu","year":"2002","journal-title":"Hemoglobin"},{"issue":"3","key":"2025111311213002900_bib15","first-page":"403","article-title":"Hemoglobin S\/hemoglobin City of Hope compound heterozygote with a Sub Saharan genetic background and severe bone marrow hypoplasia","volume":"51","author":"Paradisi","year":"2010","journal-title":"Invest Clin"},{"issue":"2","key":"2025111311213002900_bib16","doi-asserted-by":"crossref","first-page":"120","DOI":"10.1159\/000318737","article-title":"A new sickling variant 'Hb S-Wake \u03b2[(Glu6Val-Asn139 Ser)]' found in a compound heterozygote with Hb S \u03b2(Glu6Val) coinherited with homozygous \u03b1-thalassemia-2: phenotype and molecular characteristics","volume":"124","author":"Kutlar","year":"2010","journal-title":"Acta Haematol"},{"issue":"7","key":"2025111311213002900_bib17","doi-asserted-by":"crossref","first-page":"643","DOI":"10.1002\/ajh.20847","article-title":"Hemoglobin SE disease: a concise review","volume":"82","author":"Masiello","year":"2007","journal-title":"Am J Hematol"},{"issue":"7","key":"2025111311213002900_bib18","doi-asserted-by":"crossref","first-page":"2819","DOI":"10.1182\/blood.V90.7.2819","article-title":"Molecular interactions between Hb alpha-G Philadelphia, HbC, and HbS: phenotypic implications for SC alpha-G Philadelphia disease","volume":"90","author":"Lawrence","year":"1997","journal-title":"Blood"},{"issue":"4","key":"2025111311213002900_bib19","doi-asserted-by":"crossref","first-page":"295","DOI":"10.3109\/03630269308997483","article-title":"Three sickle cell anemia patients each with a different alpha chain variant. Diagnostic complications","volume":"17","author":"Gu","year":"1993","journal-title":"Hemoglobin"},{"issue":"3","key":"2025111311213002900_bib20","doi-asserted-by":"crossref","first-page":"192","DOI":"10.1002\/ajh.2830440310","article-title":"Hemoglobin sickle-Lepore: report of two siblings and review of the literature","volume":"44","author":"Seward","year":"1993","journal-title":"Am J Hematol"},{"issue":"3","key":"2025111311213002900_bib21","doi-asserted-by":"crossref","first-page":"e261","DOI":"10.5001\/omj.2021.102","article-title":"Non-S sickling hemoglobin variants: historical genetic diagnostic and clinical perspectives","volume":"36","author":"Ahmed","year":"2021","journal-title":"Oman Med J"},{"issue":"11","key":"2025111311213002900_bib22","doi-asserted-by":"crossref","first-page":"1390","DOI":"10.1002\/ajh.26702","article-title":"HbSC disease: a time for progress","volume":"97","author":"Minniti","year":"2022","journal-title":"Am J Hematol"},{"issue":"1","key":"2025111311213002900_bib23","doi-asserted-by":"crossref","first-page":"31","DOI":"10.1056\/NEJM197301042880108","article-title":"Frequency of sickle disorders in US blacks","volume":"288","author":"Motulsky","year":"1973","journal-title":"N Engl J Med"},{"key":"2025111311213002900_bib24","doi-asserted-by":"crossref","first-page":"1671","DOI":"10.1038\/srep01671","article-title":"The distribution of haemoglobin C and its prevalence in newborns in Africa","volume":"3","author":"Piel","year":"2013","journal-title":"Sci Rep"},{"issue":"1","key":"2025111311213002900_bib25","doi-asserted-by":"crossref","first-page":"6","DOI":"10.1002\/ajh.21035","article-title":"Mutations and polymorphisms in hemoglobin genes and the risk of pulmonary hypertension and death in sickle cell disease","volume":"83","author":"Taylor","year":"2008","journal-title":"Am J Hematol"},{"key":"2025111311213002900_bib26","doi-asserted-by":"crossref","DOI":"10.1017\/CBO9780511596582","volume-title":"Disorders of Hemoglobin: Genetics, Pathophysiology, and Clinical Management","author":"Steinberg","year":"2009","edition":"2nd ed"},{"issue":"23","key":"2025111311213002900_bib27","doi-asserted-by":"crossref","first-page":"1639","DOI":"10.1056\/NEJM199406093302303","article-title":"Mortality in sickle-cell disease. Life expectancy and risk-factors for early death","volume":"330","author":"Platt","year":"1994","journal-title":"N Engl J Med"},{"issue":"1","key":"2025111311213002900_bib28","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1056\/NEJM199107043250103","article-title":"Pain in sickle cell disease. Rates and risk-factors","volume":"325","author":"Platt","year":"1991","journal-title":"N Engl J Med"},{"issue":"8","key":"2025111311213002900_bib29","doi-asserted-by":"crossref","first-page":"1136","DOI":"10.3324\/haematol.2011.055202","article-title":"Hemoglobin sickle cell disease complications: a clinical study of 179 cases","volume":"97","author":"Lionnet","year":"2012","journal-title":"Haematol"},{"issue":"10","key":"2025111311213002900_bib30","doi-asserted-by":"crossref","first-page":"1275","DOI":"10.1002\/ajh.26652","article-title":"The pleiotropic effects of alpha thalassemia on HbSS and HbSC sickle cell disease: reduced erythrocyte cation co-transport activity, serum erythropoietin, and transfusion burden, do not translate into increased survival","volume":"97","author":"Brewin","year":"2022","journal-title":"Am J Hematol"},{"issue":"2","key":"2025111311213002900_bib31","doi-asserted-by":"crossref","first-page":"EVIDoa2400402","DOI":"10.1056\/EVIDoa2400402","article-title":"Hydroxyurea for children and adults with hemoglobin SC disease","volume":"4","author":"Dei-Adomakoh","year":"2025","journal-title":"NEJM Evid"},{"issue":"5","key":"2025111311213002900_bib32","doi-asserted-by":"crossref","first-page":"985","DOI":"10.1182\/blood.V68.5.985.985","article-title":"Alpha-thalassemia in blacks: genetic and clinical aspects and interactions with the sickle hemoglobin gene","volume":"68","author":"Steinberg","year":"1986","journal-title":"Blood"},{"issue":"5","key":"2025111311213002900_bib33","doi-asserted-by":"crossref","first-page":"805","DOI":"10.1083\/jcb.128.5.805","article-title":"A posttranslational modification of beta-actin contributes to the slow dissociation of the spectrin-protein 4.1-actin complex of irreversibly sickled cells","volume":"128","author":"Shartava","year":"1995","journal-title":"J Cell Biol"},{"issue":"1","key":"2025111311213002900_bib34","first-page":"53","article-title":"The irreversibly sickled cell: a perspective","volume":"50","author":"Goodman","year":"2004","journal-title":"Cell Mol Biol"},{"issue":"1","key":"2025111311213002900_bib35","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1002\/ajh.23344","article-title":"Changes in the properties of normal human red blood cells during in vivo aging","volume":"88","author":"Franco","year":"2013","journal-title":"Am J Hematol"},{"issue":"21","key":"2025111311213002900_bib36","doi-asserted-by":"crossref","first-page":"3750","DOI":"10.1016\/j.bpj.2024.09.024","article-title":"Novel single-cell measurements suggest irreversibly sickled cells are neither dense nor dehydrated","volume":"123","author":"Reese","year":"2024","journal-title":"Biophys J"},{"key":"2025111311213002900_bib37","doi-asserted-by":"crossref","DOI":"10.1017\/CBO9780511596582","volume-title":"Disorders of hemoglobin: Genetics, Pathophysiology, and Clinical Management","author":"Steinberg","year":"2009","edition":"2nd ed"},{"issue":"3","key":"2025111311213002900_bib38","doi-asserted-by":"crossref","first-page":"167","DOI":"10.1016\/S0268-960X(03)00003-1","article-title":"The paradox of hemoglobin SC disease","volume":"17","author":"Nagel","year":"2003","journal-title":"Blood Rev"},{"key":"2025111311213002900_bib39","doi-asserted-by":"crossref","first-page":"100971","DOI":"10.1016\/j.mam.2021.100971","article-title":"Impact of hemoglobin biophysical studies on molecular pathogenesis and drug therapy for sickle cell disease","volume":"84","author":"Eaton","year":"2022","journal-title":"Mol Aspects Med"},{"issue":"1","key":"2025111311213002900_bib40","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1002\/1097-0134(20010101)42:1<99::AID-PROT100>3.0.CO;2-R","article-title":"Differential pathways in oxy and deoxy HbC aggregation\/crystallization","volume":"42","author":"Hirsch","year":"2001","journal-title":"Proteins"},{"issue":"4","key":"2025111311213002900_bib41","doi-asserted-by":"crossref","first-page":"775","DOI":"10.1182\/blood.V66.4.775.775","article-title":"Ligand state of intraerythrocytic circulating HbC crystals in homozygous CC patients","volume":"66","author":"Hirsch","year":"1985","journal-title":"Blood"},{"issue":"12","key":"2025111311213002900_bib42","doi-asserted-by":"crossref","first-page":"5936","DOI":"10.1016\/S0021-9258(18)60656-8","article-title":"The inhibition of hemoglobin C crystallization by hemoglobin F","volume":"263","author":"Hirsch","year":"1988","journal-title":"J Biol Chem"},{"issue":"5","key":"2025111311213002900_bib43","doi-asserted-by":"crossref","first-page":"1823","DOI":"10.1182\/blood.V74.5.1823.1823","article-title":"Acceleration of hemoglobin C crystallization by hemoglobin S","volume":"74","author":"Lin","year":"1989","journal-title":"Blood"},{"issue":"11","key":"2025111311213002900_bib44","doi-asserted-by":"crossref","first-page":"1548","DOI":"10.1182\/blood-2016-10-745711","article-title":"Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation","volume":"129","author":"Gluckman","year":"2017","journal-title":"Blood"},{"key":"2025111311213002900_bib45","doi-asserted-by":"crossref","first-page":"9636897241246351","DOI":"10.1177\/09636897241246351","article-title":"Hematopoietic stem cell transplantation in sickle cell disease: a multidimensional review","volume":"33","author":"Rostami","year":"2024","journal-title":"Cell Transpl"},{"issue":"24","key":"2025111311213002900_bib46","doi-asserted-by":"crossref","first-page":"2309","DOI":"10.1056\/NEJMoa0904971","article-title":"Allogeneic hematopoietic stem-cell transplantation for sickle cell disease","volume":"361","author":"Hsieh","year":"2009","journal-title":"N Engl J Med"},{"issue":"21","key":"2025111311213002900_bib47","doi-asserted-by":"crossref","first-page":"2561","DOI":"10.1182\/blood-2016-05-715870","article-title":"A trial of unrelated donor marrow transplantation for children with severe sickle cell disease","volume":"128","author":"Shenoy","year":"2016","journal-title":"Blood"},{"issue":"25","key":"2025111311213002900_bib48","doi-asserted-by":"crossref","first-page":"2654","DOI":"10.1182\/blood.2023023301","article-title":"An international learning collaborative phase 2 trial for haploidentical bone marrow transplant in sickle cell disease","volume":"143","author":"Kassim","year":"2024","journal-title":"Blood"},{"issue":"3","key":"2025111311213002900_bib49","doi-asserted-by":"crossref","first-page":"EVIDoa2400192","DOI":"10.1056\/EVIDoa2400192","article-title":"Haploidentical bone marrow transplantation for sickle cell disease","volume":"4","author":"Kassim","year":"2025","journal-title":"NEJM Evid"},{"key":"2025111311213002900_bib50","doi-asserted-by":"crossref","first-page":"1403936","DOI":"10.3389\/fimmu.2024.1403936","article-title":"Graft-versus-tumor effect of post-transplant cyclophosphamide-based allogeneic hematopoietic cell transplantation","volume":"15","author":"Nakamae","year":"2024","journal-title":"Front Immunol"},{"issue":"5","key":"2025111311213002900_bib51","doi-asserted-by":"crossref","first-page":"306","DOI":"10.1097\/00043426-200106000-00014","article-title":"Hydroxyurea therapy for pediatric patients with hemoglobin SC disease","volume":"23","author":"Miller","year":"2001","journal-title":"J Pediatr Hematol Oncol"},{"issue":"4","key":"2025111311213002900_bib52","doi-asserted-by":"crossref","first-page":"838","DOI":"10.1046\/j.1365-2141.1997.3173132.x","article-title":"Cellular effects of hydroxyurea in Hb SC disease","volume":"98","author":"Steinberg","year":"1997","journal-title":"Br J Haematol"},{"issue":"2","key":"2025111311213002900_bib53","doi-asserted-by":"crossref","first-page":"323","DOI":"10.1002\/pbc.24283","article-title":"Hydroxyurea treatment of children with hemoglobin SC disease","volume":"60","author":"Yates","year":"2013","journal-title":"Pediatr Blood Cancer"},{"issue":"2","key":"2025111311213002900_bib54","doi-asserted-by":"crossref","first-page":"238","DOI":"10.1002\/ajh.24255","article-title":"Effects of hydroxyurea treatment for patients with hemoglobin SC disease","volume":"91","author":"Luchtman-Jones","year":"2016","journal-title":"Am J Hematol"},{"issue":"6","key":"2025111311213002900_bib55","doi-asserted-by":"crossref","first-page":"771","DOI":"10.1111\/j.1365-2141.2010.08523.x","article-title":"The effects of hydroxycarbamide and magnesium on haemoglobin SC disease: results of the multi-centre CHAMPS trial","volume":"152","author":"Wang","year":"2011","journal-title":"Br J Haematol"},{"issue":"3","key":"2025111311213002900_bib56","doi-asserted-by":"crossref","first-page":"206","DOI":"10.1002\/ajh.10140","article-title":"Outcome in hemoglobin SC disease: a four-decade observational study of clinical, hematologic, and genetic factors","volume":"70","author":"Powars","year":"2002","journal-title":"Am J Hematol"},{"issue":"1","key":"2025111311213002900_bib57","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1182\/blood.2024028136","article-title":"A novel mouse model of hemoglobin SC disease reveals mechanisms underlying beneficial effects of hydroxyurea","volume":"146","author":"Setayesh","year":"2025","journal-title":"Blood"},{"issue":"6","key":"2025111311213002900_bib58","doi-asserted-by":"crossref","first-page":"1228","DOI":"10.1111\/bjh.17561","article-title":"Delta-globin gene expression improves sickle cell disease in a humanised mouse model","volume":"193","author":"Porcu","year":"2021","journal-title":"Br J Haematol"},{"issue":"20","key":"2025111311213002900_bib59","doi-asserted-by":"crossref","first-page":"2719","DOI":"10.1182\/blood-2017-02-765891","article-title":"Treating sickle cell disease by targeting HbS polymerization","volume":"129","author":"Eaton","year":"2017","journal-title":"Blood"},{"issue":"8","key":"2025111311213002900_bib60","doi-asserted-by":"crossref","first-page":"e26444","DOI":"10.1002\/pbc.26444","article-title":"Hemoglobin C trait accentuates erythrocyte dehydration in hereditary xerocytosis","volume":"64","author":"Yang","year":"2017","journal-title":"Pediatr Blood Cancer"},{"issue":"25","key":"2025111311213002900_bib61","doi-asserted-by":"crossref","first-page":"2699","DOI":"10.1182\/blood-2017-04-590810","article-title":"Disorders of erythrocyte hydration","volume":"130","author":"Gallagher","year":"2017","journal-title":"Blood"},{"issue":"4","key":"2025111311213002900_bib62","doi-asserted-by":"crossref","first-page":"245","DOI":"10.1111\/j.1365-2257.1996.tb01287.x","article-title":"Mean cell haemoglobin concentration in subjects with haemoglobin C, D, E and S traits","volume":"18","author":"Hinchliffe","year":"1996","journal-title":"Clin Lab Haematol"},{"issue":"1","key":"2025111311213002900_bib63","doi-asserted-by":"crossref","first-page":"126","DOI":"10.1172\/JCI105961","article-title":"Subunit dissociation of certain abnormal human hemoglobins","volume":"48","author":"Bunn","year":"1969","journal-title":"J Clin Invest"},{"issue":"20","key":"2025111311213002900_bib64","doi-asserted-by":"crossref","first-page":"5390","DOI":"10.1016\/S0021-9258(18)62767-X","article-title":"The dimerization of deoxyhemoglobin and of oxyhemoglobin. Evidence for cleavage along the same plane","volume":"245","author":"Park","year":"1970","journal-title":"J Biol Chem"},{"issue":"5","key":"2025111311213002900_bib65","doi-asserted-by":"crossref","first-page":"988","DOI":"10.1021\/bi00702a024","article-title":"Asymmetrical hemoglobin hybrids. An approach to the study of subunit interactions","volume":"13","author":"Bunn","year":"1974","journal-title":"Biochemistry"},{"key":"2025111311213002900_bib66","author":"Gupta","year":"13  2025"},{"issue":"6","key":"2025111311213002900_bib67","doi-asserted-by":"crossref","first-page":"676","DOI":"10.1002\/ajh.26518","article-title":"Fetal hemoglobin in beta hemoglobinopathies: is enough too much?","volume":"97","author":"Steinberg","year":"2022","journal-title":"Am J Hematol"},{"issue":"17","key":"2025111311213002900_bib68","first-page":"2085","article-title":"In vivo HSC prime editing rescues sickle cell disease in a mouse model","volume":"141","author":"Li","year":"2023","journal-title":"Blood"},{"issue":"6656","key":"2025111311213002900_bib69","doi-asserted-by":"crossref","first-page":"436","DOI":"10.1126\/science.ade6967","article-title":"In vivo hematopoietic stem cell modification by mRNA delivery","volume":"381","author":"Breda","year":"2023","journal-title":"Science"},{"issue":"6","key":"2025111311213002900_bib70","first-page":"352.e1","article-title":"Best practices in gene therapy for sickle cell disease and transfusion-dependent \u03b2-thalassemia","volume":"31","author":"Frangoul","year":"2025","journal-title":"Transpl Cell Ther"},{"issue":"4","key":"2025111311213002900_bib71","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1080\/14653240902849788","article-title":"Granulocyte colony-stimulating factor (G-CSF) administration in individuals with sickle cell disease: time for a moratorium?","volume":"11","author":"Fitzhugh","year":"2009","journal-title":"Cytotherapy"},{"issue":"11","key":"2025111311213002900_bib72","doi-asserted-by":"crossref","first-page":"3782","DOI":"10.3390\/jcm9113782","article-title":"Fetal hemoglobin in sickle hemoglobinopathies: high HbF genotypes and phenotypes","volume":"9","author":"Steinberg","year":"2020","journal-title":"J Clin Med"},{"issue":"9","key":"2025111311213002900_bib73","doi-asserted-by":"crossref","first-page":"1647","DOI":"10.1002\/ajh.27729","article-title":"Mean corpuscular hemoglobin modulates HbF\/F-cell and clinical response to gene therapy and hydroxyurea in sickle cell disease","volume":"100","author":"Steinberg","year":"2025","journal-title":"Am J Hematol"}],"container-title":["Blood"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/ashpublications.org\/blood\/article-pdf\/doi\/10.1182\/blood.2025029964\/2420003\/blood_bld-2025-029964-main.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/ashpublications.org\/blood\/article-pdf\/doi\/10.1182\/blood.2025029964\/2420003\/blood_bld-2025-029964-main.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,11,13]],"date-time":"2025-11-13T16:21:48Z","timestamp":1763050908000},"score":1,"resource":{"primary":{"URL":"https:\/\/ashpublications.org\/blood\/article\/146\/20\/2385\/546809\/Gene-therapy-for-HbSC-disease-and-other-compound"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,11,13]]},"references-count":73,"journal-issue":{"issue":"20","published-print":{"date-parts":[[2025,11,13]]}},"URL":"https:\/\/doi.org\/10.1182\/blood.2025029964","relation":{},"ISSN":["0006-4971","1528-0020"],"issn-type":[{"value":"0006-4971","type":"print"},{"value":"1528-0020","type":"electronic"}],"subject":[],"published-other":{"date-parts":[[2025,11,13]]},"published":{"date-parts":[[2025,11,13]]}}}