{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,1]],"date-time":"2026-08-01T13:17:48Z","timestamp":1785590268847,"version":"3.56.0"},"reference-count":157,"publisher":"Wiley","issue":"S1","license":[{"start":{"date-parts":[[2026,3,14]],"date-time":"2026-03-14T00:00:00Z","timestamp":1773446400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2026,3,14]],"date-time":"2026-03-14T00:00:00Z","timestamp":1773446400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2026,4]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>The practical aspects of developing curative treatments for sickle cell disease (SCD) in Africa, such as gene therapy and hematopoietic stem cell transplantation, involve strengthening healthcare infrastructure, training healthcare professionals, establishing regional treatment centers, and creating national SCD programs. The costs associated with gene therapy and stem cell transplants can be prohibitive, especially in low\u2010 and middle\u2010income countries. Strategies to address affordability, including local manufacturing, government funding, and partnerships with global health agencies, are essential to ensure equitable access. Establishing ethical and regulatory guidelines while raising awareness among patients and communities is critical. Early diagnosis through newborn screening and adequate clinical care programs are vital for identifying individuals with SCD who could benefit from curative therapies and other interventions. Addressing cultural beliefs and promoting positive attitudes toward SCD and its management is essential. While curative therapies offer hope, hydroxyurea and other disease\u2010modifying therapies with established clinical benefits are more accessible in many settings. Prioritizing these medications ensures that patients with SCD are medically prepared to receive curative therapies while simultaneously building capacity for advanced treatments, thus creating a pragmatic approach. Lastly, international collaboration and partnerships among researchers, global health agencies, and local organizations are vital for sharing knowledge, resources, and best practices in SCD management.<\/jats:p>","DOI":"10.1002\/ajh.70221","type":"journal-article","created":{"date-parts":[[2026,3,14]],"date-time":"2026-03-14T10:46:21Z","timestamp":1773485181000},"page":"56-74","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Exploring Affordable Curative Therapy for Sickle Cell Disease in Africa: A Comprehensive Overview"],"prefix":"10.1002","volume":"101","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0484-5903","authenticated-orcid":false,"given":"Adetola A.","family":"Kassim","sequence":"first","affiliation":[{"name":"Department of Medicine, Division of Hematology and Oncology Vanderbilt\u2010Meharry Center for Excellence in Sickle Cell Disease, Vanderbilt University Medical Center  Nashville Tennessee USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexis A.","family":"Thompson","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Cancer and Blood Disease Institute Cincinnati Children's Hospital Medical Center  Cincinnati Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Punam","family":"Malik","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Children's Hospital of Philadelphia University of Pennsylvania Perelman School of Medicine  Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,3,14]]},"reference":[{"issue":"8","key":"e_1_2_10_2_1","doi-asserted-by":"crossref","first-page":"e585","DOI":"10.1016\/S2352-3026(23)00118-7","article-title":"Global, Regional, and National Prevalence and Mortality Burden of Sickle Cell Disease, 2000\u20132021: A Systematic Analysis From the Global Burden of Disease Study 2021","volume":"10","author":"GBD 2021 Sickle Cell Disease Collaborators","year":"2023","journal-title":"Lancet. Haematology"},{"key":"e_1_2_10_3_1","doi-asserted-by":"crossref","first-page":"263","DOI":"10.1146\/annurev-pathmechdis-012418-012838","article-title":"Pathophysiology of Sickle Cell Disease","volume":"14","author":"Sundd P.","year":"2019","journal-title":"Annual Review of Pathology"},{"key":"e_1_2_10_4_1","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1080\/00034983.1979.11687243","article-title":"Abnormal Haemoglobins in The Sudan Savanna of Nigeria. I. Prevalence of Haemoglobins and Relationships Between Sickle Cell Trait, Malaria and Survival","volume":"73","author":"Fleming A. F.","year":"1979","journal-title":"Annals of Tropical Medicine and Parasitology"},{"key":"e_1_2_10_5_1","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0014699","article-title":"Mortality in Sickle Cell Anemia in Africa: A Prospective Cohort Study in Tanzania","volume":"6","author":"Makani J.","year":"2011","journal-title":"PLoS One"},{"issue":"11","key":"e_1_2_10_6_1","doi-asserted-by":"crossref","DOI":"10.1001\/jamanetworkopen.2023.44546","article-title":"Use of Disease\u2010Modifying Treatments in Patients With Sickle Cell Disease","volume":"6","author":"Newman T. V.","year":"2023","journal-title":"JAMA Network Open"},{"key":"e_1_2_10_7_1","doi-asserted-by":"crossref","first-page":"541","DOI":"10.1016\/j.jpeds.2008.09.052","article-title":"Trends in Pediatric Sickle Cell Disease\u2010Related Mortality in the United States, 1983\u20132002","volume":"154","author":"Yanni E.","year":"2009","journal-title":"Journal of Pediatrics"},{"key":"e_1_2_10_8_1","first-page":"663","volume-title":"Disease Control Priorities in Developing Countries","author":"Weatherall D.","year":"2006"},{"issue":"6","key":"e_1_2_10_9_1","doi-asserted-by":"crossref","first-page":"615","DOI":"10.1182\/blood-2018-10-880575","article-title":"Decreased Median Survival of Adults With Sickle Cell Disease After Adjusting for Left Truncation Bias: A Pooled Analysis","volume":"133","author":"DeBaun M. R.","year":"2019","journal-title":"Blood"},{"key":"e_1_2_10_10_1","doi-asserted-by":"crossref","first-page":"36","DOI":"10.1002\/ajh.21569","article-title":"Cardiopulmonary Complications Leading to Premature Deaths in Adult Patients With Sickle Cell Disease","volume":"85","author":"Fitzhugh C. D.","year":"2009","journal-title":"American Journal of Hematology"},{"issue":"2","key":"e_1_2_10_11_1","doi-asserted-by":"crossref","first-page":"203","DOI":"10.1002\/ajh.21905","article-title":"Health\u2010Related Quality of Life in Adults With Sickle Cell Disease (SCD): A Report From the Comprehensive Sickle Cell Centers Clinical Trial Consortium","volume":"86","author":"Dampier C.","year":"2011","journal-title":"American Journal of Hematology"},{"key":"e_1_2_10_12_1","doi-asserted-by":"crossref","DOI":"10.1186\/1477-7525-3-50","article-title":"Health Related Quality of Life in Sickle Cell Patients: The PiSCES Project","volume":"3","author":"McClish D. K.","year":"2005","journal-title":"Health and Quality of Life Outcomes"},{"issue":"6","key":"e_1_2_10_13_1","doi-asserted-by":"crossref","first-page":"661","DOI":"10.1080\/13803395.2016.1149153","article-title":"Cognitive Deficits Are Associated With Unemployment in Adults With Sickle Cell Anemia","volume":"38","author":"Sanger M.","year":"2016","journal-title":"Journal of Clinical and Experimental Neuropsychology"},{"issue":"1","key":"e_1_2_10_14_1","doi-asserted-by":"crossref","first-page":"519","DOI":"10.1182\/hematology.2023000485","article-title":"Using Disease\u2010Modifying Therapies in Sickle Cell Disease","volume":"2023","author":"Rai P.","year":"2023","journal-title":"Hematology. American Society of Hematology. Education Program"},{"issue":"9","key":"e_1_2_10_15_1","doi-asserted-by":"crossref","first-page":"1153","DOI":"10.1002\/ajh.25202","article-title":"Clustering of End\u2010Organ Disease and Earlier Mortality in Adults With Sickle Cell Disease: A Retrospective\u2010Prospective Cohort Study","volume":"93","author":"Chaturvedi S.","year":"2018","journal-title":"American Journal of Hematology"},{"issue":"1","key":"e_1_2_10_16_1","doi-asserted-by":"crossref","first-page":"435","DOI":"10.1182\/asheducation-2017.1.435","article-title":"Chronic Organ Failure in Adult Sickle Cell Disease","volume":"2017","author":"Vichinsky E.","year":"2017","journal-title":"Hematology. American Society of Hematology. Education Program"},{"issue":"5","key":"e_1_2_10_17_1","doi-asserted-by":"crossref","first-page":"530","DOI":"10.1002\/ajh.23683","article-title":"Factors Associated With Survival in a Contemporary Adult Sickle Cell Disease Cohort","volume":"89","author":"Elmariah H.","year":"2014","journal-title":"American Journal of Hematology"},{"issue":"9","key":"e_1_2_10_18_1","doi-asserted-by":"crossref","first-page":"886","DOI":"10.1056\/NEJMoa035477","article-title":"Pulmonary Hypertension as a Risk Factor for Death in Patients With Sickle Cell Disease","volume":"350","author":"Gladwin M. T.","year":"2004","journal-title":"New England Journal of Medicine"},{"issue":"1","key":"e_1_2_10_19_1","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1056\/NEJMoa1005565","article-title":"A Hemodynamic Study of Pulmonary Hypertension in Sickle Cell Disease","volume":"365","author":"Parent F.","year":"2011","journal-title":"New England Journal of Medicine"},{"issue":"1","key":"e_1_2_10_20_1","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1183\/09031936.00134410","article-title":"Pulmonary Hypertension Diagnosed by Right Heart Catheterisation in Sickle Cell Disease","volume":"39","author":"Fonseca G. H. H.","year":"2012","journal-title":"European Respiratory Journal"},{"issue":"9","key":"e_1_2_10_21_1","doi-asserted-by":"crossref","first-page":"857","DOI":"10.1056\/NEJMp038250","article-title":"Pulmonary Hypertension in Sickle Cell Disease","volume":"350","author":"Vichinsky E. P.","year":"2004","journal-title":"New England Journal of Medicine"},{"issue":"13","key":"e_1_2_10_22_1","doi-asserted-by":"crossref","first-page":"1544","DOI":"10.1182\/blood-2015-05-644435","article-title":"Low Forced Expiratory Volume Is Associated With Earlier Death in Sickle Cell Anemia","volume":"126","author":"Kassim A. A.","year":"2015","journal-title":"Blood"},{"issue":"1","key":"e_1_2_10_23_1","first-page":"288","article-title":"Cerebrovascular Accidents in Sickle Cell Disease: Rates and Risk Factors","volume":"91","author":"Ohene\u2010Frempong K.","year":"1998","journal-title":"Blood"},{"key":"e_1_2_10_24_1","doi-asserted-by":"crossref","first-page":"461","DOI":"10.1016\/0002-9343(78)90772-6","article-title":"The Natural History of Stroke in Sickle Cell Disease","volume":"65","author":"Powars D.","year":"1978","journal-title":"American Journal of Medicine"},{"issue":"3","key":"e_1_2_10_25_1","doi-asserted-by":"crossref","first-page":"360","DOI":"10.1111\/bjh.12024","article-title":"High One Year Mortality in Adults With Sickle Cell Disease and End\u2010Stage Renal Disease","volume":"159","author":"McClellan A. C.","year":"2012","journal-title":"British Journal of Haematology"},{"issue":"16","key":"e_1_2_10_26_1","doi-asserted-by":"crossref","first-page":"2038","DOI":"10.1182\/blood-2016-01-694562","article-title":"Silent Cerebral Infarcts and Cerebral Aneurysms Are Prevalent in Adults With Sickle Cell Anemia","volume":"127","author":"Kassim A. A.","year":"2016","journal-title":"Blood"},{"issue":"2","key":"e_1_2_10_27_1","doi-asserted-by":"crossref","first-page":"162","DOI":"10.1002\/ajh.23604","article-title":"Parent Education and Biologic Factors Influence on Cognition in Sickle Cell Anemia","volume":"89","author":"King A. A.","year":"2014","journal-title":"American Journal of Hematology"},{"issue":"10","key":"e_1_2_10_28_1","first-page":"E188","article-title":"Silent Cerebral Infarction, Income, and Grade Retention Among Students With Sickle Cell Anemia","volume":"89","author":"King A. A.","year":"2014","journal-title":"American Journal of Hematology"},{"key":"e_1_2_10_29_1","volume-title":"Sickle\u2010Cell Anaemia: Report by the Secretariat","author":"World Health Assembly","year":"2006"},{"issue":"1","key":"e_1_2_10_30_1","doi-asserted-by":"crossref","first-page":"e26","DOI":"10.1016\/S2352-3026(21)00368-9","article-title":"Hydroxyurea for Primary Stroke Prevention in Children With Sickle Cell Anaemia in Nigeria (SPRING): A Double\u2010Blind, Multicentre, Randomised, Phase 3 Trial","volume":"9","author":"Abdullahi S. U.","year":"2022","journal-title":"Lancet. Haematology"},{"issue":"4","key":"e_1_2_10_31_1","doi-asserted-by":"crossref","first-page":"e261","DOI":"10.1016\/S2352-3026(22)00405-7","article-title":"Hydroxyurea With Dose Escalation for Primary Stroke Risk Reduction in Children With Sickle Cell Anaemia in Tanzania (SPHERE): An Open\u2010Label, Phase 2 Trial","volume":"10","author":"Ambrose E. E.","year":"2023","journal-title":"Lancet. Haematology"},{"issue":"12","key":"e_1_2_10_32_1","doi-asserted-by":"crossref","first-page":"1940","DOI":"10.1002\/pbc.24698","article-title":"Chronic Blood Transfusion for Primary and Secondary Stroke Prevention in Nigerian Children With Sickle Cell Disease: A 5\u2010Year Appraisal","volume":"60","author":"Lagunju I. A.","year":"2013","journal-title":"Pediatric Blood & Cancer"},{"issue":"1","key":"e_1_2_10_33_1","article-title":"Blood Transfusion for Preventing Primary and Secondary Stroke in People With Sickle Cell Disease","volume":"1","author":"Estcourt L. J.","year":"2017","journal-title":"Cochrane Database of Systematic Reviews"},{"issue":"3","key":"e_1_2_10_34_1","doi-asserted-by":"crossref","first-page":"772","DOI":"10.1182\/blood-2010-01-261123","article-title":"Silent Cerebral Infarcts Occur Despite Regular Blood Transfusion Therapy After First Strokes in Children With Sickle Cell Disease","volume":"117","author":"Hulbert M. L.","year":"2011","journal-title":"Blood"},{"issue":"8","key":"e_1_2_10_35_1","doi-asserted-by":"crossref","first-page":"699","DOI":"10.1056\/NEJMoa1401731","article-title":"Controlled Trial of Transfusions for Silent Cerebral Infarcts in Sickle Cell Anemia","volume":"371","author":"DeBaun M. R.","year":"2014","journal-title":"New England Journal of Medicine"},{"issue":"3","key":"e_1_2_10_36_1","first-page":"243","article-title":"Cardiomyopathy With Restrictive Physiology in Sickle Cell Disease","volume":"9","author":"Niss O.","year":"2016","journal-title":"JACC: Cardiovascular Imaging"},{"issue":"2","key":"e_1_2_10_37_1","doi-asserted-by":"crossref","first-page":"205","DOI":"10.1182\/blood-2017-02-767624","article-title":"Association Between Diffuse Myocardial Fibrosis and Diastolic Dysfunction in Sickle Cell Anemia","volume":"130","author":"Niss O.","year":"2017","journal-title":"Blood"},{"issue":"9","key":"e_1_2_10_38_1","doi-asserted-by":"crossref","first-page":"1208","DOI":"10.1182\/blood.2020005944","article-title":"IL\u201018 Mediates Sickle Cell Cardiomyopathy and Ventricular Arrhythmias","volume":"137","author":"Gupta A.","year":"2021","journal-title":"Blood"},{"issue":"6","key":"e_1_2_10_39_1","doi-asserted-by":"crossref","first-page":"1217","DOI":"10.1016\/j.hoc.2022.07.014","article-title":"The Cardiopulmonary Complications of Sickle Cell Disease","volume":"36","author":"Desai A. A.","year":"2022","journal-title":"Hematology\/Oncology Clinics of North America"},{"issue":"11","key":"e_1_2_10_40_1","doi-asserted-by":"crossref","first-page":"1322","DOI":"10.1182\/blood.2021015303","article-title":"Early Initiation of Disease\u2010Modifying Therapy Can Impede or Prevent Diffuse Myocardial Fibrosis in Sickle Cell Anemia","volume":"140","author":"Niss O.","year":"2022","journal-title":"Blood"},{"issue":"18","key":"e_1_2_10_41_1","doi-asserted-by":"crossref","first-page":"3668","DOI":"10.1182\/bloodadvances.2021004394C","article-title":"American Society of Hematology 2021 Guidelines for Sickle Cell Disease: Stem Cell Transplantation","volume":"5","author":"Kanter J.","year":"2021","journal-title":"Blood Advances"},{"issue":"6","key":"e_1_2_10_42_1","doi-asserted-by":"crossref","first-page":"369","DOI":"10.1056\/NEJM199608083350601","article-title":"Bone Marrow Transplantation for Sickle Cell Disease","volume":"335","author":"Walters M. C.","year":"1996","journal-title":"New England Journal of Medicine"},{"issue":"1","key":"e_1_2_10_43_1","first-page":"22","article-title":"Bone Marrow Transplantation for Sickle Cell Disease. The United States Experience","volume":"16","author":"Johnson F. L.","year":"1994","journal-title":"American Journal of Pediatric Hematology\/Oncology"},{"issue":"4","key":"e_1_2_10_44_1","doi-asserted-by":"crossref","first-page":"446","DOI":"10.1002\/ajh.25401","article-title":"Bone Marrow Transplantation for Adolescents and Young Adults With Sickle Cell Disease: Results of a Prospective Multicenter Pilot Study","volume":"94","author":"Krishnamurti L.","year":"2019","journal-title":"American Journal of Hematology"},{"issue":"1","key":"e_1_2_10_45_1","doi-asserted-by":"crossref","first-page":"91","DOI":"10.3324\/haematol.2018.213207","article-title":"Long\u2010Term Event\u2010Free Survival, Chimerism and Fertility Outcomes in 234 Patients With Sickle\u2010Cell Anemia Younger Than 30\u2009Years After Myeloablative Conditioning and Matched\u2010Sibling Transplantation in France","volume":"105","author":"Bernaudin F.","year":"2020","journal-title":"Haematologica"},{"issue":"12","key":"e_1_2_10_46_1","doi-asserted-by":"crossref","first-page":"2178","DOI":"10.1016\/j.bbmt.2017.08.038","article-title":"Relationship Between Mixed Donor\u2010Recipient Chimerism and Disease Recurrence After Hematopoietic Cell Transplantation for Sickle Cell Disease","volume":"23","author":"Abraham A.","year":"2017","journal-title":"Biology of Blood and Marrow Transplantation"},{"issue":"23","key":"e_1_2_10_47_1","doi-asserted-by":"crossref","first-page":"2514","DOI":"10.1182\/blood.2022017960","article-title":"Increased Incidence of Hematologic Malignancies in SCD After HCT in Adults With Graft Failure and Mixed Chimerism","volume":"140","author":"Lawal R. A.","year":"2022","journal-title":"Blood"},{"issue":"12","key":"e_1_2_10_48_1","doi-asserted-by":"crossref","first-page":"2227","DOI":"10.1200\/JCO.22.01203","article-title":"Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease","volume":"41","author":"Eapen M.","year":"2023","journal-title":"Journal of Clinical Oncology"},{"issue":"10","key":"e_1_2_10_49_1","doi-asserted-by":"crossref","first-page":"1562","DOI":"10.1200\/JCO.1988.6.10.1562","article-title":"Regimen\u2010Related Toxicity in Patients Undergoing Bone Marrow Transplantation","volume":"6","author":"Bearman S. I.","year":"1988","journal-title":"Journal of Clinical Oncology"},{"issue":"9","key":"e_1_2_10_50_1","doi-asserted-by":"crossref","first-page":"574","DOI":"10.1097\/01.mph.0000136453.93704.2e","article-title":"Validity of the Child Health Questionnaire for Use in Children With Sickle Cell Disease","volume":"26","author":"Panepinto J. A.","year":"2004","journal-title":"Journal of Pediatric Hematology\/Oncology"},{"issue":"7","key":"e_1_2_10_51_1","article-title":"Sickle Cell Transplantation Evaluation of Long\u2010Term and Late Effects Registry (STELLAR) to Compare Long\u2010Term Outcomes After Hematopoietic Cell Transplantation to Those in Siblings Without Sickle Cell Disease and in Nontransplanted Individuals With Sickle Cell Disease: Design and Feasibility Study","volume":"11","author":"Krishnamurti L.","year":"2022","journal-title":"Journal of Medical Internet Research Protocol"},{"issue":"4","key":"e_1_2_10_52_1","doi-asserted-by":"crossref","first-page":"725","DOI":"10.1002\/pbc.24036","article-title":"Health\u2010Related Quality of Life (HRQL) in Children With Sickle Cell Disease and Thalassemia Following Hematopoietic Stem Cell Transplant (HSCT)","volume":"59","author":"Kelly M. J.","year":"2012","journal-title":"Pediatric Blood & Cancer"},{"issue":"22","key":"e_1_2_10_53_1","doi-asserted-by":"crossref","first-page":"4285","DOI":"10.1182\/blood-2012-07-438408","article-title":"HLA\u2010Haploidentical Bone Marrow Transplantation With Posttransplant Cyclophosphamide Expands the Donor Pool for Patients With Sickle Cell Disease","volume":"120","author":"Bolanos\u2010Meade J.","year":"2012","journal-title":"Blood"},{"issue":"8","key":"e_1_2_10_54_1","doi-asserted-by":"crossref","first-page":"523","DOI":"10.1038\/bmt.2008.203","article-title":"Reduced Intensity HLA\u2010Haploidentical BMT With Post Transplantation Cyclophosphamide in Nonmalignant Hematologic Diseases","volume":"42","author":"Brodsky R. A.","year":"2008","journal-title":"Bone Marrow Transplantation"},{"issue":"12","key":"e_1_2_10_55_1","doi-asserted-by":"crossref","first-page":"780","DOI":"10.1056\/NEJM198409203111207","article-title":"Bone\u2010Marrow Transplantation in a Patient With Sickle\u2010Cell Anemia","volume":"311","author":"Johnson F. L.","year":"1984","journal-title":"New England Journal of Medicine"},{"issue":"8","key":"e_1_2_10_56_1","doi-asserted-by":"crossref","first-page":"519","DOI":"10.1016\/S1083-8791(03)00192-7","article-title":"Results of Minimally Toxic Nonmyeloablative Transplantation in Patients With Sickle Cell Anemia and Beta\u2010Thalassemia","volume":"9","author":"Iannone R.","year":"2003","journal-title":"Biology of Blood and Marrow Transplantation"},{"issue":"12","key":"e_1_2_10_57_1","doi-asserted-by":"crossref","first-page":"1093","DOI":"10.1002\/ajh.24183","article-title":"Successful Matched Sibling Donor Marrow Transplantation Following Reduced Intensity Conditioning in Children With Hemoglobinopathies","volume":"90","author":"King A. A.","year":"2015","journal-title":"American Journal of Hematology"},{"issue":"24","key":"e_1_2_10_58_1","doi-asserted-by":"crossref","first-page":"2309","DOI":"10.1056\/NEJMoa0904971","article-title":"Allogeneic Hematopoietic Stem\u2010Cell Transplantation for Sickle Cell Disease","volume":"361","author":"Hsieh M. M.","year":"2009","journal-title":"New England Journal of Medicine"},{"issue":"11","key":"e_1_2_10_59_1","doi-asserted-by":"crossref","first-page":"e585","DOI":"10.1016\/S2352-3026(19)30154-1","article-title":"Effect of Donor Type and Conditioning Regimen Intensity on Allogeneic Transplantation Outcomes in Patients With Sickle Cell Disease: A Retrospective Multicentre, Cohort Study","volume":"6","author":"Eapen M.","year":"2019","journal-title":"Lancet. Haematology"},{"key":"e_1_2_10_60_1","doi-asserted-by":"crossref","first-page":"1799","DOI":"10.1182\/blood-2021-144454","article-title":"Nonmyeloablative HLA\u2010Identical Sibling Donor Transplantation for Children and Young Adults With Sickle Cell Disease: Interim Results of the SUN Multicenter Phase II Trial","volume":"138","author":"Nickel R. S.","year":"2021","journal-title":"Blood"},{"issue":"1","key":"e_1_2_10_61_1","doi-asserted-by":"crossref","DOI":"10.1182\/blood-2023-189914","article-title":"Excellent Outcomes of HLA Matched Related Donor Transplant for Adults With Severe Sickle Cell Disease Using a Non\u2010Myeloablative Conditioning With Thiotepa and Post\u2010Transplant Cyclophosphamide: Multi\u2010Center International Experience","volume":"142","author":"Alasbali R.","year":"2023","journal-title":"Blood"},{"issue":"1","key":"e_1_2_10_62_1","doi-asserted-by":"crossref","first-page":"48","DOI":"10.1001\/jama.2014.7192","article-title":"Nonmyeloablative HLA\u2010Matched Sibling Allogeneic Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Phenotype","volume":"312","author":"Hsieh M. M.","year":"2014","journal-title":"Journal of the American Medical Association"},{"issue":"12","key":"e_1_2_10_63_1","doi-asserted-by":"crossref","first-page":"665","DOI":"10.1053\/bbmt.2001.v7.pm11787529","article-title":"Stable Mixed Hematopoietic Chimerism After Bone Marrow Transplantation for Sickle Cell Anemia","volume":"7","author":"Walters M. C.","year":"2001","journal-title":"Biology of Blood and Marrow Transplantation"},{"issue":"11","key":"e_1_2_10_64_1","doi-asserted-by":"crossref","DOI":"10.3390\/jcm11113160","article-title":"Clonal Hematopoiesis and the Risk of Hematologic Malignancies After Curative Therapies for Sickle Cell Disease","volume":"11","author":"Gondek L. P.","year":"2022","journal-title":"Journal of Clinical Medicine"},{"issue":"21","key":"e_1_2_10_65_1","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 S.","year":"2016","journal-title":"Blood"},{"issue":"16","key":"e_1_2_10_66_1","first-page":"3894","article-title":"Abatacept Is Effective as GVHD Prophylaxis in Unrelated Donor Stem Cell Transplantation for Children With Severe Sickle Cell Disease","volume":"4","author":"Ngwube A.","year":"2020","journal-title":"Blood Advances"},{"issue":"8","key":"e_1_2_10_67_1","doi-asserted-by":"crossref","first-page":"1265","DOI":"10.1016\/j.bbmt.2012.01.019","article-title":"Unrelated Donor Cord Blood Transplantation for Children With Severe Sickle Cell Disease: Results of One Cohort From the Phase II Study From the Blood and Marrow Transplant Clinical Trials Network (BMT CTN)","volume":"18","author":"Kamani N. R.","year":"2012","journal-title":"Biology of Blood and Marrow Transplantation"},{"issue":"9","key":"e_1_2_10_68_1","doi-asserted-by":"crossref","first-page":"1587","DOI":"10.1016\/j.bbmt.2017.05.027","article-title":"Unrelated Umbilical Cord Blood Transplantation for Sickle Cell Disease Following Reduced\u2010Intensity Conditioning: Results of a Phase I Trial","volume":"23","author":"Abraham A.","year":"2017","journal-title":"Biology of Blood and Marrow Transplantation"},{"key":"e_1_2_10_69_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2025017413"},{"key":"e_1_2_10_70_1","doi-asserted-by":"publisher","DOI":"10.1056\/EVIDoa2400192"},{"key":"e_1_2_10_71_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2023023301"},{"issue":"2","key":"e_1_2_10_72_1","doi-asserted-by":"crossref","first-page":"98","DOI":"10.1016\/j.hemonc.2019.12.006","article-title":"Alternative Donor: a\u00df\/CD19 T\u2010Cell\u2010Depleted Haploidentical Hematopoietic Stem Cell Transplantation for Sickle Cell Disease","volume":"13","author":"Foell J.","year":"2020","journal-title":"Hematology\/Oncology and Stem Cell Therapy"},{"key":"e_1_2_10_73_1","doi-asserted-by":"crossref","first-page":"339","DOI":"10.1056\/NEJMsa1311707","article-title":"HLA\u2010Match Likelihoods for Hematopoietic Stem\u2010Cell Grafts in the US Registry","volume":"371","author":"Garget L.","year":"2014","journal-title":"New England Journal of Medicine"},{"issue":"1","key":"e_1_2_10_74_1","doi-asserted-by":"crossref","first-page":"234","DOI":"10.1182\/hematology.2024000550","article-title":"Transfusions, Disease\u2010Modifying Treatments, and Curative Therapies for Sickle Cell Anemia in Africa: Where Are We Now?","volume":"2024","author":"Odame I.","year":"2024","journal-title":"Hematology. American Society of Hematology. Education Program"},{"issue":"2","key":"e_1_2_10_75_1","doi-asserted-by":"crossref","first-page":"e59","DOI":"10.1111\/bjh.17532","article-title":"Overcoming the Hurdles and Establishing a Haematopoietic Stem Cell Transplant Centre in a Resource Poor Setting: An Example From Dhaka, Bangladesh","volume":"194","author":"Mahfuz H.","year":"2021","journal-title":"British Journal of Haematology"},{"issue":"1","key":"e_1_2_10_76_1","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1111\/bjh.17106","article-title":"Haematopoietic Stem Cell Transplantation in Tanzania","volume":"192","author":"Mtenga J.","year":"2021","journal-title":"British Journal of Haematology"},{"issue":"8","key":"e_1_2_10_77_1","first-page":"S47","article-title":"Haematopoietic Stem Cell Transplantation in South Africa: Current Limitations and Future Perspectives","volume":"109","author":"Grobbelaar S.","year":"2019","journal-title":"South African Medical Journal"},{"key":"e_1_2_10_78_1","doi-asserted-by":"crossref","first-page":"474","DOI":"10.3109\/10428194.2015.1055485","article-title":"Antifungal Prophylaxis With Amphotericin B Deoxycholate Emulsified in Lipids for Acute Myeloid Leukemia Patients Treated in Low Economy Countries","volume":"57","author":"Poudyal B. S.","year":"2016","journal-title":"Leukemia & Lymphoma"},{"key":"e_1_2_10_79_1","doi-asserted-by":"crossref","first-page":"267.e1","DOI":"10.1016\/j.jtct.2020.12.015","article-title":"Worldwide Network for Blood and Marrow Transplantation (WBMT) Recommendations Regarding Essential Medications Required to Establish an Early Stage Hematopoietic Cell Transplantation Program","volume":"27","author":"El Fakih R.","year":"2021","journal-title":"Transplantation and Cellular Therapy"},{"key":"e_1_2_10_80_1","doi-asserted-by":"crossref","DOI":"10.1186\/s12916-020-01892-4","article-title":"Undernutrition: A Major but Potentially Preventable Cause of Poor Outcomes in Children Living With Sickle Cell Disease in Africa","volume":"19","author":"Williams T. N.","year":"2021","journal-title":"BMC Medicine"},{"key":"e_1_2_10_81_1","unstructured":"Statistics South Africa \u201cMid\u2010Year Population Estimates 2018 \u201daccessed February 7 2021 https:\/\/www.statssa.gov.za\/publications\/P0302\/P03022018.pdf."},{"issue":"13","key":"e_1_2_10_82_1","doi-asserted-by":"crossref","first-page":"1597","DOI":"10.1182\/blood-2017-05-783233","article-title":"Increased Risk of Leukemia Among Sickle Cell Disease Patients in California","volume":"130","author":"Brunson A.","year":"2017","journal-title":"Blood"},{"issue":"8","key":"e_1_2_10_83_1","doi-asserted-by":"crossref","first-page":"303","DOI":"10.1177\/0141076816651037","article-title":"Risk of Individual Malignant Neoplasms in Patients With Sickle Cell Disease: English National Record Linkage Study","volume":"109","author":"Seminog O. O.","year":"2016","journal-title":"Journal of the Royal Society of Medicine"},{"issue":"12","key":"e_1_2_10_84_1","doi-asserted-by":"crossref","first-page":"2649","DOI":"10.1111\/j.1537-2995.2010.02746.x","article-title":"Delayed Recovery After Autologous Peripheral Hematopoietic Cell Transplantation: Potential Effect of a High Number of Total Nucleated Cells in the Graft","volume":"50","author":"Trebeden\u2010Negre H.","year":"2010","journal-title":"Transfusion"},{"issue":"12","key":"e_1_2_10_85_1","doi-asserted-by":"crossref","first-page":"1390","DOI":"10.1038\/s41409-023-02092-3","article-title":"No Impact of CD34+ Cell Dose on Outcome Among Children Undergoing Autologous Hematopoietic Stem Cell Transplant for High\u2010Risk Neuroblastoma","volume":"58","author":"Knight T. E.","year":"2023","journal-title":"Bone Marrow Transplantation"},{"issue":"3","key":"e_1_2_10_86_1","doi-asserted-by":"crossref","first-page":"61","DOI":"10.1089\/scd.2024.0178","article-title":"PEGylated Granulocyte Colony\u2010Stimulating Factor and Plerixafor Enhance Autologous Stem and Progenitor Cell Mobilization and Transplantation in Pediatric Patients","volume":"34","author":"Hochheuser C.","year":"2025","journal-title":"Stem Cells and Development"},{"issue":"1","key":"e_1_2_10_87_1","doi-asserted-by":"crossref","first-page":"31","DOI":"10.1016\/j.blre.2014.01.001","article-title":"Advances in Stem Cell Mobilization","volume":"28","author":"Hopman R. K.","year":"2014","journal-title":"Blood Reviews"},{"issue":"4","key":"e_1_2_10_88_1","doi-asserted-by":"crossref","first-page":"1131","DOI":"10.1182\/blood.V92.4.1131","article-title":"Ex Vivo Expansion of Genetically Marked Rhesus Peripheral Blood Progenitor Cells Results in Diminished Long\u2010Term Repopulating Ability","volume":"92","author":"Tisdale J. F.","year":"1998","journal-title":"Blood"},{"issue":"7","key":"e_1_2_10_89_1","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 J.","year":"2022","journal-title":"New England Journal of Medicine"},{"issue":"3","key":"e_1_2_10_90_1","doi-asserted-by":"crossref","first-page":"252","DOI":"10.1056\/NEJMoa2031054","article-title":"CRISPR\u2010Cas9 Gene Editing for Sickle Cell Disease and Beta\u2010Thalassemia","volume":"384","author":"Frangoul H.","year":"2021","journal-title":"New England Journal of Medicine"},{"key":"e_1_2_10_91_1","volume-title":"CASGEVY (STN125785). January 16, 2024, Summary Basis for Regulatory Action","author":"US FDA","year":"2024"},{"key":"e_1_2_10_92_1","volume-title":"LYFGENIA (STN: 125788). Summary Basis for Regulatory Action","author":"US FDA","year":"2024"},{"issue":"4","key":"e_1_2_10_93_1","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1080\/14653240902849788","article-title":"Granulocyte Colony\u2010Stimulating Factor (G\u2010CSF) Administration in Individuals With Sickle Cell Disease: Time for a Moratorium?","volume":"11","author":"Fitzhugh C. D.","year":"2009","journal-title":"Cytotherapy"},{"issue":"23","key":"e_1_2_10_94_1","doi-asserted-by":"crossref","first-page":"2071","DOI":"10.1182\/blood.2019002227","article-title":"Pathologic Angiogenesis in the Bone Marrow of Humanized Sickle Cell Mice Is Reversed by Blood Transfusion","volume":"135","author":"Park S. Y.","year":"2020","journal-title":"Blood"},{"issue":"2","key":"e_1_2_10_95_1","doi-asserted-by":"crossref","first-page":"286","DOI":"10.1111\/bjh.15902","article-title":"Bone Marrow Characterization in Sickle Cell Disease: Inflammation and Stress Erythropoiesis Lead to Suboptimal CD34 Recovery","volume":"186","author":"Leonard A.","year":"2019","journal-title":"British Journal of Haematology"},{"issue":"1","key":"e_1_2_10_96_1","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1002\/ajh.26741","article-title":"Lovo\u2010Cel Gene Therapy for Sickle Cell Disease: Treatment Process Evolution and Outcomes in the Initial Groups of the HGB\u2010206 Study","volume":"98","author":"Kanter J.","year":"2023","journal-title":"American Journal of Hematology"},{"key":"e_1_2_10_97_1","doi-asserted-by":"crossref","DOI":"10.1016\/j.bcmd.2021.102588","article-title":"Evidence for Continued Dose Escalation of Plerixafor for Hematopoietic Progenitor Cell Collections in Sickle Cell Disease","volume":"90","author":"Boulad F.","year":"2021","journal-title":"Blood Cells, Molecules & Diseases"},{"issue":"9","key":"e_1_2_10_98_1","doi-asserted-by":"crossref","first-page":"E239","DOI":"10.1002\/ajh.25867","article-title":"Safety and Feasibility of Hematopoietic Progenitor Stem Cell Collection by Mobilization With Plerixafor Followed by Apheresis vs. Bone Marrow Harvest in Patients With Sickle Cell Disease in the Multi\u2010Center HGB\u2010206 Trial","volume":"95","author":"Tisdale J. F.","year":"2020","journal-title":"American Journal of Hematology"},{"issue":"9","key":"e_1_2_10_99_1","doi-asserted-by":"crossref","first-page":"2403","DOI":"10.1182\/bloodadvances.2021004232","article-title":"Disease Severity Impacts Plerixafor\u2010Mobilized Stem Cell Collection in Patients With Sickle Cell Disease","volume":"5","author":"Leonard A.","year":"2021","journal-title":"Blood Advances"},{"issue":"9","key":"e_1_2_10_100_1","doi-asserted-by":"crossref","first-page":"2775","DOI":"10.1111\/trf.16551","article-title":"Process and Procedural Adjustments to Improve CD34+ Collection Efficiency of Hematopoietic Progenitor Cell Collections in Sickle Cell Disease","volume":"61","author":"Avecilla S. T.","year":"2021","journal-title":"Transfusion"},{"issue":"4","key":"e_1_2_10_101_1","doi-asserted-by":"crossref","first-page":"740","DOI":"10.1111\/bjh.18311","article-title":"Optimizing Haematopoietic Stem and Progenitor Cell Apheresis Collection From Plerixafor\u2010Mobilized Patients With Sickle Cell Disease","volume":"198","author":"Sharma A.","year":"2022","journal-title":"British Journal of Haematology"},{"issue":"6039","key":"e_1_2_10_102_1","doi-asserted-by":"crossref","first-page":"218","DOI":"10.1126\/science.1201219","article-title":"Isolation of Single Human Hematopoietic Stem Cells Capable of Long\u2010Term Multilineage Engraftment","volume":"333","author":"Notta F.","year":"2011","journal-title":"Science"},{"issue":"4","key":"e_1_2_10_103_1","doi-asserted-by":"crossref","first-page":"977","DOI":"10.1016\/j.stemcr.2017.02.010","article-title":"Efficient Ex\u00a0Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy","volume":"8","author":"Zonari E.","year":"2017","journal-title":"Stem Cell Reports"},{"issue":"9","key":"e_1_2_10_104_1","doi-asserted-by":"crossref","first-page":"2163","DOI":"10.1016\/j.ymthe.2017.05.023","article-title":"Improving Gene Therapy Efficiency Through the Enrichment of Human Hematopoietic Stem Cells","volume":"25","author":"Masiuk K. E.","year":"2017","journal-title":"Molecular Therapy"},{"issue":"1","key":"e_1_2_10_105_1","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1038\/sj.bmt.1703792","article-title":"Large\u2010Scale Isolation of CD133+ Progenitor Cells From G\u2010CSF Mobilized Peripheral Blood Stem Cells","volume":"31","author":"Gordon P. R.","year":"2003","journal-title":"Bone Marrow Transplantation"},{"issue":"414","key":"e_1_2_10_106_1","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aan1145","article-title":"A Distinct Hematopoietic Stem Cell Population for Rapid Multilineage Engraftment in Nonhuman Primates","volume":"9","author":"Radtke S.","year":"2017","journal-title":"Science Translational Medicine"},{"key":"e_1_2_10_107_1","doi-asserted-by":"crossref","first-page":"679","DOI":"10.1016\/j.omtm.2020.07.010","article-title":"Purification of Human CD34+CD90+ HSCs Reduces Target Cell Population and Improves Lentiviral Transduction for Gene Therapy","volume":"18","author":"Radtke S.","year":"2020","journal-title":"Molecular Therapy \u2010 Methods & Clinical Development"},{"issue":"1","key":"e_1_2_10_108_1","doi-asserted-by":"crossref","first-page":"198","DOI":"10.1016\/j.stemcr.2020.10.007","article-title":"Beta\u2010Globin Lentiviral Vectors Have Reduced Titers due to Incomplete Vector RNA Genomes and Lowered Virion Production","volume":"16","author":"Han J.","year":"2021","journal-title":"Stem Cell Reports"},{"issue":"4","key":"e_1_2_10_109_1","doi-asserted-by":"crossref","first-page":"1407","DOI":"10.1016\/j.ymthe.2025.02.036","article-title":"A Differentiated Beta\u2010Globin Gene Replacement Strategy Uses Heterologous Introns to Restore Physiological Expression","volume":"33","author":"Wallace K. A.","year":"2025","journal-title":"Molecular Therapy"},{"key":"e_1_2_10_110_1","doi-asserted-by":"crossref","first-page":"999","DOI":"10.1016\/j.omtm.2020.04.006","article-title":"Creating New Beta\u2010Globin\u2010Expressing Lentiviral Vectors by High\u2010Resolution Mapping of Locus Control Region Enhancer Sequences","volume":"17","author":"Morgan R. A.","year":"2020","journal-title":"Molecular Therapy \u2010 Methods & Clinical Development"},{"issue":"2","key":"e_1_2_10_111_1","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2024.101254","article-title":"A Novel High\u2010Titer, Bifunctional Lentiviral Vector for Autologous Hematopoietic Stem Cell Gene Therapy of Sickle Cell Disease","volume":"32","author":"Hart K. L.","year":"2024","journal-title":"Molecular Therapy \u2010 Methods & Clinical Development"},{"issue":"12","key":"e_1_2_10_112_1","doi-asserted-by":"crossref","first-page":"3728","DOI":"10.1002\/bit.28834","article-title":"Auto\u2010Transduction in Lentiviral Vector Bioprocessing: A Quantitative Assessment and a Novel Inhibition Strategy","volume":"121","author":"Williams\u2010Fegredo T.","year":"2024","journal-title":"Biotechnology and Bioengineering"},{"key":"e_1_2_10_113_1","doi-asserted-by":"crossref","DOI":"10.3389\/fbioe.2024.1409203","article-title":"Degradation of Specific Glycosaminoglycans Improves Transfection Efficiency and Vector Production in Transient Lentiviral Vector Manufacturing Processes","volume":"12","author":"Williams\u2010Fegredo T.","year":"2024","journal-title":"Frontiers in Bioengineering and Biotechnology"},{"issue":"5","key":"e_1_2_10_114_1","doi-asserted-by":"crossref","DOI":"10.1002\/biot.202400090","article-title":"Generation of Stable Suspension Producer Cell Lines for Serum\u2010Free Lentivirus Production","volume":"19","author":"Klimpel M.","year":"2024","journal-title":"Biotechnology Journal"},{"issue":"2","key":"e_1_2_10_115_1","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2024.101270","article-title":"Genetic Alteration of SJ293TS Cells and Modification of Serum\u2010Free Media Enhances Lentiviral Vector Production","volume":"32","author":"Bauler M.","year":"2024","journal-title":"Molecular Therapy \u2010 Methods & Clinical Development"},{"key":"e_1_2_10_116_1","first-page":"134","article-title":"Development of a Novel Anti\u2010CD19 Chimeric Antigen Receptor: A Paradigm for an Affordable CAR T Cell Production at Academic Institutions","volume":"12","author":"Castella M.","year":"2019","journal-title":"Molecular Therapy"},{"key":"e_1_2_10_117_1","doi-asserted-by":"crossref","DOI":"10.1038\/ncomms13173","article-title":"Semi\u2010Automated Closed System Manufacturing of Lentivirus Gene\u2010Modified Haematopoietic Stem Cells for Gene Therapy","volume":"7","author":"Adair J. E.","year":"2016","journal-title":"Nature Communications"},{"issue":"6203","key":"e_1_2_10_118_1","doi-asserted-by":"crossref","first-page":"1509","DOI":"10.1126\/science.1256337","article-title":"Cord Blood Expansion. Pyrimidoindole Derivatives Are Agonists of Human Hematopoietic Stem Cell Self\u2010Renewal","volume":"345","author":"Fares I.","year":"2014","journal-title":"Science"},{"issue":"5997","key":"e_1_2_10_119_1","doi-asserted-by":"crossref","first-page":"1345","DOI":"10.1126\/science.1191536","article-title":"Aryl Hydrocarbon Receptor Antagonists Promote the Expansion of Human Hematopoietic Stem Cells","volume":"329","author":"Boitano A. E.","year":"2010","journal-title":"Science"},{"issue":"2","key":"e_1_2_10_120_1","doi-asserted-by":"crossref","first-page":"104","DOI":"10.1089\/hgtb.2017.085","article-title":"Improving Lentiviral Transduction of CD34+ Hematopoietic Stem and Progenitor Cells","volume":"29","author":"Hauber I.","year":"2018","journal-title":"Human Gene Therapy Methods"},{"issue":"12","key":"e_1_2_10_121_1","doi-asserted-by":"crossref","first-page":"545","DOI":"10.1038\/s41434-020-0150-z","article-title":"Optimizing Lentiviral Vector Transduction of Hematopoietic Stem Cells for Gene Therapy","volume":"27","author":"Jang Y.","year":"2020","journal-title":"Gene Therapy"},{"key":"e_1_2_10_122_1","doi-asserted-by":"crossref","first-page":"147","DOI":"10.1007\/7651_2024_582","article-title":"Robust Expansion of Hematopoietic Stem Cells Ex\u00a0Vivo Using Small Molecule Cocktails","volume":"2960","author":"Kaewsakulthong W.","year":"2025","journal-title":"Methods in Molecular Biology"},{"issue":"12","key":"e_1_2_10_123_1","doi-asserted-by":"crossref","first-page":"5035","DOI":"10.1007\/s00277-024-05773-1","article-title":"Advances in Hematopoietic Stem Cells Ex\u00a0Vivo Expansion Associated With Bone Marrow Niche","volume":"103","author":"Deng J.","year":"2024","journal-title":"Annals of Hematology"},{"issue":"3","key":"e_1_2_10_124_1","doi-asserted-by":"crossref","first-page":"188","DOI":"10.1089\/hum.2021.089","article-title":"Preferential Expansion of Human CD34+CD133+CD90+ Hematopoietic Stem Cells Enhances Gene\u2010Modified Cell Frequency for Gene Therapy","volume":"33","author":"Christopher A. C.","year":"2022","journal-title":"Human Gene Therapy"},{"issue":"4","key":"e_1_2_10_125_1","first-page":"151","article-title":"Recent Advances in Ex\u00a0Vivo Expansion of Human Hematopoietic Stem Cells","volume":"6","author":"Sakurai M.","year":"2023","journal-title":"Blood Cell Therapy"},{"issue":"1","key":"e_1_2_10_126_1","doi-asserted-by":"crossref","first-page":"124","DOI":"10.1016\/j.ymthe.2023.11.020","article-title":"Removal of Innate Immune Barriers Allows Efficient Transduction of Quiescent Human Hematopoietic Stem Cells","volume":"32","author":"Valeri E.","year":"2024","journal-title":"Molecular Therapy"},{"issue":"5","key":"e_1_2_10_127_1","doi-asserted-by":"crossref","first-page":"575","DOI":"10.1007\/s12185-024-03853-6","article-title":"Expansion Effect of Romiplostim on Hematopoietic Stem and Progenitor Cells Versus Thrombopoietin and Eltrombopag","volume":"120","author":"Tezuka Y.","year":"2024","journal-title":"International Journal of Hematology"},{"issue":"9","key":"e_1_2_10_128_1","doi-asserted-by":"crossref","first-page":"820","DOI":"10.1056\/NEJMoa2215643","article-title":"CRISPR\u2010Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease","volume":"389","author":"Sharma A.","year":"2023","journal-title":"New England Journal of Medicine"},{"issue":"2","key":"e_1_2_10_129_1","doi-asserted-by":"crossref","first-page":"S2","DOI":"10.1016\/j.jtct.2025.01.008","article-title":"Gene Therapy With Reduced\u2010Intensity Conditioning for Sickle Cell Disease","volume":"31","author":"Stella M.","year":"2025","journal-title":"Transplantation and Cellular Therapy"},{"issue":"6","key":"e_1_2_10_130_1","doi-asserted-by":"crossref","first-page":"1672","DOI":"10.1016\/j.ymthe.2024.03.032","article-title":"Anti\u2010CD45 PBD\u2010Based Antibody\u2010Drug Conjugates Are Effective Targeted Conditioning Agents for Gene Therapy and Stem Cell Transplant","volume":"32","author":"Yeung J.","year":"2024","journal-title":"Molecular Therapy"},{"issue":"25","key":"e_1_2_10_131_1","doi-asserted-by":"crossref","first-page":"2978","DOI":"10.1182\/blood.2024024636","article-title":"cMPL\u2010Based Purification and Depletion of Human Hematopoietic Stem Cells: Implications for Pretransplant Conditioning","volume":"145","author":"Araki D.","year":"2025","journal-title":"Blood"},{"issue":"22","key":"e_1_2_10_132_1","doi-asserted-by":"crossref","first-page":"2235","DOI":"10.1056\/NEJMoa2504747","article-title":"Patient\u2010Specific In\u00a0Vivo Gene Editing to Treat a Rare Genetic Disease","volume":"392","author":"Musunuru K.","year":"2025","journal-title":"New England Journal of Medicine"},{"issue":"17","key":"e_1_2_10_133_1","first-page":"2085","article-title":"In Vivo HSC Prime Editing Rescues Sickle Cell Disease in a Mouse Model","volume":"141","author":"Li C.","year":"2023","journal-title":"Blood"},{"issue":"12","key":"e_1_2_10_134_1","doi-asserted-by":"crossref","first-page":"4353","DOI":"10.1016\/j.ymthe.2024.10.018","article-title":"Introducing a Hemoglobin G\u2010Makassar Variant in HSCs by In\u00a0Vivo Base Editing Treats Sickle Cell Disease in Mice","volume":"32","author":"Li C.","year":"2024","journal-title":"Molecular Therapy"},{"issue":"2","key":"e_1_2_10_135_1","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2025.101448","article-title":"In Vivo HSC Transduction in Humanized Mice Mediated by Novel Capsid\u2010Modified HDAd Vectors","volume":"33","author":"Georgakopoulou A.","year":"2025","journal-title":"Molecular Therapy \u2010 Methods & Clinical Development"},{"issue":"6656","key":"e_1_2_10_136_1","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 L.","year":"2023","journal-title":"Science"},{"issue":"1","key":"e_1_2_10_137_1","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-025-56578-3","article-title":"Base Editing HbS to HbG\u2010Makassar Improves Hemoglobin Function Supporting Its Use in Sickle Cell Disease","volume":"16","author":"Kostamo Z.","year":"2025","journal-title":"Nature Communications"},{"issue":"5","key":"e_1_2_10_138_1","doi-asserted-by":"crossref","first-page":"202","DOI":"10.1038\/s41434-023-00429-7","article-title":"Looking Ahead: Ethical and Social Challenges of Somatic Gene Therapy for Sickle Cell Disease in Africa","volume":"31","author":"Munung N. S.","year":"2024","journal-title":"Gene Therapy"},{"issue":"746","key":"e_1_2_10_139_1","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.adn1902","article-title":"The Translational Gap for Gene Therapies in Low\u2010 and Middle\u2010Income Countries","volume":"16","author":"Doxzen K. W.","year":"2024","journal-title":"Science Translational Medicine"},{"issue":"8","key":"e_1_2_10_140_1","doi-asserted-by":"crossref","first-page":"e633","DOI":"10.1016\/S2352-3026(23)00096-0","article-title":"Defining Global Strategies to Improve Outcomes in Sickle Cell Disease: A Lancet Haematology Commission","volume":"10","author":"Piel F. B.","year":"2023","journal-title":"Lancet. Haematology"},{"issue":"8","key":"e_1_2_10_141_1","doi-asserted-by":"crossref","first-page":"493","DOI":"10.1080\/17474086.2024.2372320","article-title":"Sickle Cell Disease and Infertility Risks: Implications for Counseling and Care of Affected Girls and Women","volume":"17","author":"Pecker L. H.","year":"2024","journal-title":"Expert Review of Hematology"},{"issue":"7","key":"e_1_2_10_142_1","doi-asserted-by":"crossref","first-page":"1111","DOI":"10.1182\/blood.2021012756","article-title":"Diminished Ovarian Reserve in Young Women With Sickle Cell Anemia","volume":"139","author":"Pecker L. H.","year":"2022","journal-title":"Blood"},{"issue":"9","key":"e_1_2_10_143_1","doi-asserted-by":"crossref","DOI":"10.3390\/jcm11092318","article-title":"Fertility After Curative Therapy for Sickle Cell Disease: A Comprehensive Review to Guide Care","volume":"11","author":"Nickel R. S.","year":"2022","journal-title":"Journal of Clinical Medicine"},{"issue":"7","key":"e_1_2_10_144_1","doi-asserted-by":"crossref","first-page":"445","DOI":"10.1097\/MPH.0b013e31806451ac","article-title":"Children With Sickle Cell Disease: Growth and Gonadal Function After Hematopoietic Stem Cell Transplantation","volume":"29","author":"Brachet C.","year":"2007","journal-title":"Journal of Pediatric Hematology\/Oncology"},{"issue":"2","key":"e_1_2_10_145_1","doi-asserted-by":"crossref","first-page":"130.e1","DOI":"10.1016\/j.jtct.2022.10.018","article-title":"Reduced\u2010Intensity Conditioning Mitigates Risk for Primary Ovarian Insufficiency but Does Not Decrease Risk for Infertility in Pediatric and Young Adult Survivors of Hematopoietic Stem Cell Transplantation","volume":"29","author":"Bender J. D.","year":"2023","journal-title":"Transplant and Cellular Therapy"},{"issue":"8","key":"e_1_2_10_146_1","doi-asserted-by":"crossref","first-page":"531.e1","DOI":"10.1016\/j.jtct.2023.05.004","article-title":"Female Reproductive Health Outcomes after Hematopoietic Cell Transplantation for Sickle Cell Disease: Is Reduced Intensity Better Than Myeloablative Conditioning?","volume":"29","author":"Meacham L. R.","year":"2023","journal-title":"Transplant and Cellular Therapy"},{"issue":"4","key":"e_1_2_10_147_1","doi-asserted-by":"crossref","first-page":"202","DOI":"10.1016\/j.hemonc.2019.05.003","article-title":"Special Report: Summary of the First Meeting of African Blood and Marrow Transplantation (AfBMT) Group, Casablanca, Morocco, April 19\u201321, 2018 Held Under the Auspices of the Worldwide Network for Blood and Marrow Transplantation (WBMT)","volume":"13","author":"Harif M.","year":"2020","journal-title":"Hematology\/Oncology and Stem Cell Therapy"},{"issue":"12","key":"e_1_2_10_148_1","doi-asserted-by":"crossref","first-page":"1002.e1","DOI":"10.1016\/j.jtct.2021.08.018","article-title":"Is Haploidentical Hematopoietic Cell Transplantation Using Post\u2010Transplantation Cyclophosphamide Feasible in Sub\u2010Saharan Africa?","volume":"27","author":"Rudolph Toit J.","year":"2021","journal-title":"Transplant and Cellular Therapy"},{"issue":"3","key":"e_1_2_10_149_1","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1016\/j.jcyt.2021.09.003","article-title":"Curative Therapy for Hemoglobinopathies: An International Society for Cell & Gene Therapy Stem Cell Engineering Committee Review Comparing Outcomes, Accessibility and Cost of Ex\u00a0Vivo Stem Cell Gene Therapy Versus Allogeneic Hematopoietic Stem Cell Transplantation","volume":"24","author":"Leonard A.","year":"2022","journal-title":"Cytotherapy"},{"issue":"4","key":"e_1_2_10_150_1","doi-asserted-by":"crossref","first-page":"26","DOI":"10.1080\/15265161.2018.1431327","article-title":"The Role of the Health Care Provider in Building Trust Between Patients and Precision Medicine Research Programs","volume":"18","author":"Persaud A.","year":"2018","journal-title":"American Journal of Bioethics"},{"issue":"8","key":"e_1_2_10_151_1","doi-asserted-by":"crossref","first-page":"2007","DOI":"10.1007\/s12325-017-0587-7","article-title":"Patient Perspectives on Gene Transfer Therapy for Sickle Cell Disease","volume":"34","author":"Strong H.","year":"2017","journal-title":"Advances in Therapy"},{"issue":"5","key":"e_1_2_10_152_1","doi-asserted-by":"crossref","first-page":"1041","DOI":"10.1016\/j.bbmt.2017.11.018","article-title":"Experiences and Decision Making in Hematopoietic Stem Cell Transplant in Sickle Cell Disease: Patients and Caregivers Perspectives","volume":"24","author":"Khemani K.","year":"2018","journal-title":"Biology of Blood and Marrow Transplantation"},{"issue":"3","key":"e_1_2_10_153_1","doi-asserted-by":"crossref","first-page":"365","DOI":"10.1182\/bloodadvances.2021006281","article-title":"Lifetime Medical Costs Attributable to Sickle Cell Disease Among Nonelderly Individuals With Commercial Insurance","volume":"7","author":"Johnson K. M.","year":"2023","journal-title":"Blood Advances"},{"issue":"7","key":"e_1_2_10_154_1","first-page":"366","article-title":"The Cost of Hematopoietic Stem\u2010Cell Transplantation in the United States","volume":"10","author":"Broder M. S.","year":"2017","journal-title":"American Health & Drug Benefits"},{"issue":"8","key":"e_1_2_10_155_1","doi-asserted-by":"crossref","first-page":"1545","DOI":"10.1182\/blood-2012-05-426783","article-title":"Economics of Hematopoietic Cell Transplantation","volume":"120","author":"Khera N.","year":"2012","journal-title":"Blood"},{"key":"e_1_2_10_156_1","doi-asserted-by":"crossref","first-page":"167","DOI":"10.1016\/j.hemonc.2017.05.017","article-title":"Cost and Quality Issues in Establishing Hematopoietic Cell Transplant Program in Developing Countries","volume":"10","author":"Hashmi S. K.","year":"2017","journal-title":"Hematology\/Oncology and Stem Cell Therapy"},{"issue":"6","key":"e_1_2_10_157_1","doi-asserted-by":"crossref","first-page":"617","DOI":"10.1001\/jamapediatrics.2020.7140","article-title":"A Budget Impact Analysis of Gene Therapy for Sickle Cell Disease: The Medicaid Perspective","volume":"175","author":"DeMartino P.","year":"2021","journal-title":"JAMA Pediatrics"},{"issue":"8","key":"e_1_2_10_158_1","doi-asserted-by":"crossref","first-page":"611","DOI":"10.1007\/s40261-024-01384-w","article-title":"Budget Impact of Disease\u2010Modifying Treatments and a CRISPR Gene\u2010Edited Therapy for Sickle Cell Disease","volume":"44","author":"Abdallah K.","year":"2024","journal-title":"Clinical Drug Investigation"}],"container-title":["American Journal of Hematology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ajh.70221","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/ajh.70221","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ajh.70221","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,3,14]],"date-time":"2026-03-14T10:46:39Z","timestamp":1773485199000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/ajh.70221"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,3,14]]},"references-count":157,"journal-issue":{"issue":"S1","published-print":{"date-parts":[[2026,4]]}},"alternative-id":["10.1002\/ajh.70221"],"URL":"https:\/\/doi.org\/10.1002\/ajh.70221","archive":["Portico"],"relation":{},"ISSN":["0361-8609","1096-8652"],"issn-type":[{"value":"0361-8609","type":"print"},{"value":"1096-8652","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,3,14]]},"assertion":[{"value":"2025-12-12","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-01-21","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-03-14","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}