{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,16]],"date-time":"2026-04-16T01:39:33Z","timestamp":1776303573577,"version":"3.50.1"},"reference-count":208,"publisher":"MDPI AG","issue":"12","license":[{"start":{"date-parts":[[2021,6,10]],"date-time":"2021-06-10T00:00:00Z","timestamp":1623283200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004663","name":"Ministry of Science and Technology, Taiwan","doi-asserted-by":"publisher","award":["MOST105\u20132628-B-038-008-MY3"],"award-info":[{"award-number":["MOST105\u20132628-B-038-008-MY3"]}],"id":[{"id":"10.13039\/501100004663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004663","name":"Ministry of Science and Technology, Taiwan","doi-asserted-by":"publisher","award":["MOST106\u20133114-B-038-001"],"award-info":[{"award-number":["MOST106\u20133114-B-038-001"]}],"id":[{"id":"10.13039\/501100004663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004663","name":"Ministry of Science and Technology, Taiwan","doi-asserted-by":"publisher","award":["MOST107\u20132321-B-038-002"],"award-info":[{"award-number":["MOST107\u20132321-B-038-002"]}],"id":[{"id":"10.13039\/501100004663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004663","name":"Ministry of Science and Technology, Taiwan","doi-asserted-by":"publisher","award":["MOST107\u20132314-B-038-057"],"award-info":[{"award-number":["MOST107\u20132314-B-038-057"]}],"id":[{"id":"10.13039\/501100004663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004663","name":"Ministry of Science and Technology, Taiwan","doi-asserted-by":"publisher","award":["MOST107\u20132314-B-038-061"],"award-info":[{"award-number":["MOST107\u20132314-B-038-061"]}],"id":[{"id":"10.13039\/501100004663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004663","name":"Ministry of Science and Technology, Taiwan","doi-asserted-by":"publisher","award":["MOST108-2314-B-038-006"],"award-info":[{"award-number":["MOST108-2314-B-038-006"]}],"id":[{"id":"10.13039\/501100004663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004663","name":"Ministry of Science and Technology, Taiwan","doi-asserted-by":"publisher","award":["MOST108\u20132321-B-038-003"],"award-info":[{"award-number":["MOST108\u20132321-B-038-003"]}],"id":[{"id":"10.13039\/501100004663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004663","name":"Ministry of Science and Technology, Taiwan","doi-asserted-by":"publisher","award":["MOST 109-2314-B-038-135"],"award-info":[{"award-number":["MOST 109-2314-B-038-135"]}],"id":[{"id":"10.13039\/501100004663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004663","name":"Ministry of Science and Technology, Taiwan","doi-asserted-by":"publisher","award":["MOST 109-2321-B-038-003"],"award-info":[{"award-number":["MOST 109-2321-B-038-003"]}],"id":[{"id":"10.13039\/501100004663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004663","name":"Ministry of Science and Technology, Taiwan","doi-asserted-by":"publisher","award":["MOST 109-2320-B-002-068"],"award-info":[{"award-number":["MOST 109-2320-B-002-068"]}],"id":[{"id":"10.13039\/501100004663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004663","name":"Ministry of Science and Technology, Taiwan","doi-asserted-by":"publisher","award":["MOST 107-2321-B-038-002"],"award-info":[{"award-number":["MOST 107-2321-B-038-002"]}],"id":[{"id":"10.13039\/501100004663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004663","name":"Ministry of Science and Technology, Taiwan","doi-asserted-by":"publisher","award":["MOST 107-2314-B-038-061"],"award-info":[{"award-number":["MOST 107-2314-B-038-061"]}],"id":[{"id":"10.13039\/501100004663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004663","name":"Ministry of Science and Technology, Taiwan","doi-asserted-by":"publisher","award":["MOST108-2314-B-038-006"],"award-info":[{"award-number":["MOST108-2314-B-038-006"]}],"id":[{"id":"10.13039\/501100004663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004663","name":"Ministry of Science and Technology, Taiwan","doi-asserted-by":"publisher","award":["MOST108-2321-B-038-003"],"award-info":[{"award-number":["MOST108-2321-B-038-003"]}],"id":[{"id":"10.13039\/501100004663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004663","name":"Ministry of Science and Technology, Taiwan","doi-asserted-by":"publisher","award":["MOST109-2314-B-038-135"],"award-info":[{"award-number":["MOST109-2314-B-038-135"]}],"id":[{"id":"10.13039\/501100004663","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>Hereditary anemia has various manifestations, such as sickle cell disease (SCD), Fanconi anemia, glucose-6-phosphate dehydrogenase deficiency (G6PDD), and thalassemia. The available management strategies for these disorders are still unsatisfactory and do not eliminate the main causes. As genetic aberrations are the main causes of all forms of hereditary anemia, the optimal approach involves repairing the defective gene, possibly through the transplantation of normal hematopoietic stem cells (HSCs) from a normal matching donor or through gene therapy approaches (either in vivo or ex vivo) to correct the patient\u2019s HSCs. To clearly illustrate the importance of cell and gene therapy in hereditary anemia, this paper provides a review of the genetic aberration, epidemiology, clinical features, current management, and cell and gene therapy endeavors related to SCD, thalassemia, Fanconi anemia, and G6PDD. Moreover, we expound the future research direction of HSC derivation from induced pluripotent stem cells (iPSCs), strategies to edit HSCs, gene therapy risk mitigation, and their clinical perspectives. In conclusion, gene-corrected hematopoietic stem cell transplantation has promising outcomes for SCD, Fanconi anemia, and thalassemia, and it may overcome the limitation of the source of allogenic bone marrow transplantation.<\/jats:p>","DOI":"10.3390\/ijms22126275","type":"journal-article","created":{"date-parts":[[2021,6,10]],"date-time":"2021-06-10T21:34:38Z","timestamp":1623360878000},"page":"6275","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":33,"title":["Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing"],"prefix":"10.3390","volume":"22","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3292-3139","authenticated-orcid":false,"given":"Dito","family":"Anurogo","sequence":"first","affiliation":[{"name":"International PhD Program for Cell Therapy and Regeneration Medicine, College of Medicine, Taipei Medical University, Taipei 11031, Taiwan"},{"name":"Department of Biochemistry and Molecular Cell Biology, School of Medicine, College of Medicine, Taipei Medical University, Taipei 11031, Taiwan"},{"name":"Faculty of Medicine and Health Sciences, Universitas Muhammadiyah Makassar, Makassar 90221, Indonesia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2233-4629","authenticated-orcid":false,"given":"Nova","family":"Yuli Prasetyo Budi","sequence":"additional","affiliation":[{"name":"International PhD Program for Cell Therapy and Regeneration Medicine, College of Medicine, Taipei Medical University, Taipei 11031, Taiwan"},{"name":"Department of Biochemistry and Molecular Cell Biology, School of Medicine, College of Medicine, Taipei Medical University, Taipei 11031, Taiwan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mai-Huong","family":"Thi Ngo","sequence":"additional","affiliation":[{"name":"International PhD Program for Cell Therapy and Regeneration Medicine, College of Medicine, Taipei Medical University, Taipei 11031, Taiwan"},{"name":"Department of Biochemistry and Molecular Cell Biology, School of Medicine, College of Medicine, Taipei Medical University, Taipei 11031, Taiwan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6020-9998","authenticated-orcid":false,"given":"Yen-Hua","family":"Huang","sequence":"additional","affiliation":[{"name":"International PhD Program for Cell Therapy and Regeneration Medicine, College of Medicine, Taipei Medical University, Taipei 11031, Taiwan"},{"name":"Department of Biochemistry and Molecular Cell Biology, School of Medicine, College of Medicine, Taipei Medical University, Taipei 11031, Taiwan"},{"name":"Research Center of Cell Therapy and Regeneration Medicine, Taipei Medical University, Taipei 11031, Taiwan"},{"name":"Graduate Institute of Medical Sciences, College of Medicine, Taipei Medical University, Taipei 11031, Taiwan"},{"name":"Center for Reproductive Medicine, Taipei Medical University Hospital, Taipei 11031, Taiwan"},{"name":"Comprehensive Cancer Center, Taipei Medical University, Taipei 11031, Taiwan"},{"name":"Research Center of Cancer Translational Medicine, Taipei Medical University, Taipei 11031, Taiwan"},{"name":"PhD Program for Translational Medicine, College of Medical Science and Technology, Taipei Medical University, Taipei 11031, Taiwan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6551-5238","authenticated-orcid":false,"given":"Jeanne Adiwinata","family":"Pawitan","sequence":"additional","affiliation":[{"name":"Department of Histology, Faculty of Medicine, Universitas Indonesia, Jakarta 10430, Indonesia"},{"name":"Stem Cell Medical Technology Integrated Service Unit, Cipto Mangunkusumo Central Hospital, Faculty of Medicine, Universitas Indonesia, Jakarta 10430, Indonesia"},{"name":"Stem Cell and Tissue Engineering Research Center, Indonesia Medical Education and Research Institute (IMERI), Faculty of Medicine, Universitas Indonesia, Jakarta 10430, Indonesia"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2021,6,10]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","unstructured":"Adam, M.A., Adam, N.K., and Mohamed, B.A. (2019). Prevalence of sickle cell disease and sickle cell trait among children admitted to Al Fashir Teaching Hospital North Darfur State, Sudan. BMC Res. Notes, 12.","DOI":"10.1186\/s13104-019-4682-5"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"339","DOI":"10.1016\/j.stemcr.2017.12.006","article-title":"Cell-cycle-specific function of p53 in Fanconi anemia hematopoietic stem and progenitor cell proliferation","volume":"10","author":"Li","year":"2018","journal-title":"Stem Cell Rep."},{"key":"ref_3","doi-asserted-by":"crossref","unstructured":"Garc\u00eda-de-Teresa, B., Rodr\u00edguez, A., and Frias, S. (2020). Chromosome instability in Fanconi anemia: From breaks to phenotypic consequences. Genes, 11.","DOI":"10.20944\/preprints202011.0563.v2"},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"693","DOI":"10.1002\/em.22358","article-title":"Fanconi anemia and the underlying causes of genomic instability","volume":"61","author":"Rageul","year":"2020","journal-title":"Environ. Mol. Mutagen."},{"key":"ref_5","doi-asserted-by":"crossref","unstructured":"Gregg, X.T., and Prchal, J.T. (2018). Chapter 44\u2014Red Blood Cell Enzymopathies. Hematology, Elsevier. [7th ed.].","DOI":"10.1016\/B978-0-323-35762-3.00044-5"},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"84","DOI":"10.1016\/j.freeradbiomed.2020.10.002","article-title":"The potential link between inherited G6PD deficiency, oxidative stress, and vitamin D deficiency and the racial inequities in mortality associated with COVID-19","volume":"161","author":"Jain","year":"2020","journal-title":"Free Radic. Biol. Med."},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"L508","DOI":"10.1152\/ajplung.00165.2020","article-title":"Glucose-6-phosphate dehydrogenase deficiency contributes to metabolic abnormality and pulmonary hypertension","volume":"320","author":"Varghese","year":"2021","journal-title":"Am. J. Physiol. Lung Cell Mol. Physiol."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"42","DOI":"10.26838\/MEDRECH.2021.8.1.471","article-title":"Review article on thalassemia","volume":"8","author":"Iqbal","year":"2021","journal-title":"Med. Res. Chronicles"},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"1549","DOI":"10.1002\/sctm.20-0247","article-title":"Hematopoietic stem cells from pluripotent stem cells: Clinical potential, challenges, and future perspectives","volume":"9","author":"Demirci","year":"2020","journal-title":"Stem Cells Transl. Med."},{"key":"ref_10","doi-asserted-by":"crossref","unstructured":"Meier, E.R., Abraham, A., and Fasano, R.M. (2018). Clinical manifestations of sickle cell disease across the lifespan. Sickle Cell Disease and Hematopoietic Stem Cell Transplantation, Springer.","DOI":"10.1007\/978-3-319-62328-3"},{"key":"ref_11","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"},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1038\/nrdp.2018.10","article-title":"Sickle cell disease","volume":"4","author":"Kato","year":"2018","journal-title":"Nat. Rev. Dis. Primers"},{"key":"ref_13","doi-asserted-by":"crossref","unstructured":"Oron, A.P., Chao, D.L., Ezeanolue, E.E., Ezenwa, L.N., Piel, F.B., Ojogun, O.T., Uyoga, S., Williams, T.N., and Nnodu, O.E. (2020). Caring for Africa\u2019s sickle cell children: Will we rise to the challenge?. BMC Med., 18.","DOI":"10.1186\/s12916-020-01557-2"},{"key":"ref_14","first-page":"417","article-title":"Review of medication therapy for the prevention of sickle cell crisis","volume":"43","author":"Riley","year":"2018","journal-title":"Phys. Ther."},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.blre.2019.05.004","article-title":"Sickle cell disease: Clinical presentation and management of a global health challenge","volume":"37","author":"Houwing","year":"2019","journal-title":"Blood Rev."},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1111\/bjh.14594","article-title":"Sickle cell disease in Africa: An overview of the integrated approach to health, research, education and advocacy in Tanzania, 2004\u20132016","volume":"177","author":"Tluway","year":"2017","journal-title":"Br. J. Haematol."},{"key":"ref_17","first-page":"262","article-title":"The prevalence of leukocyte abnormalities among Sudanese patients with sickle cell disease","volume":"9","author":"Ahmed","year":"2021","journal-title":"WJARR"},{"key":"ref_18","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."},{"key":"ref_19","doi-asserted-by":"crossref","first-page":"e1458","DOI":"10.1016\/S2214-109X(19)30328-6","article-title":"The epidemiology of sickle cell disease in children recruited in infancy in Kilifi, Kenya: A prospective cohort study","volume":"7","author":"Uyoga","year":"2019","journal-title":"Lancet Glob. Health"},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"925","DOI":"10.1007\/s00277-020-03977-9","article-title":"Pathophysiology and recent therapeutic insights of sickle cell disease","volume":"99","author":"Shah","year":"2020","journal-title":"Ann. Hematol."},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"126","DOI":"10.31254\/phyto.2021.10209","article-title":"Sickle hemoglobin polymerization inhibition and antisickling medicinal plants","volume":"10","author":"Ohiagu","year":"2021","journal-title":"J. Phytopharmacol."},{"key":"ref_22","doi-asserted-by":"crossref","first-page":"30492","DOI":"10.1074\/jbc.M110.206516","article-title":"K-Cl cotransporter gene expression during human and murine erythroid differentiation","volume":"286","author":"Pan","year":"2011","journal-title":"J. Biol. Chem."},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"1651","DOI":"10.1007\/s00424-013-1308-y","article-title":"Identification of the Ca2+ entry pathway involved in deoxygenation-induced phosphatidylserine exposure in red blood cells from patients with sickle cell disease","volume":"465","author":"Cytlak","year":"2013","journal-title":"Pflugers Arch. Eur. J. Physiol."},{"key":"ref_24","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","year":"2019","journal-title":"Annu. Rev. Pathol."},{"key":"ref_25","doi-asserted-by":"crossref","first-page":"34","DOI":"10.1016\/j.jbiomech.2016.11.022","article-title":"Biomechanics and biorheology of red blood cells in sickle cell anemia","volume":"50","author":"Li","year":"2017","journal-title":"J. Biomech."},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"345","DOI":"10.1146\/annurev-bioeng-070909-105339","article-title":"Sickle cell biomechanics","volume":"12","author":"Barabino","year":"2010","journal-title":"Annu. Rev. Biomed. Eng."},{"key":"ref_27","doi-asserted-by":"crossref","first-page":"3892","DOI":"10.1182\/blood-2013-05-498311","article-title":"Vaso-occlusion in sickle cell disease: Pathophysiology and novel targeted therapies","volume":"122","author":"Manwani","year":"2013","journal-title":"Blood"},{"key":"ref_28","doi-asserted-by":"crossref","first-page":"645","DOI":"10.1080\/17474086.2020.1758555","article-title":"Sickle cell disease as a vascular disorder","volume":"13","year":"2020","journal-title":"Expert Rev. Hematol."},{"key":"ref_29","doi-asserted-by":"crossref","first-page":"1137","DOI":"10.1016\/j.bpj.2012.01.050","article-title":"Epinephrine modulates BCAM\/Lu and ICAM-4 expression on the sickle cell trait red blood cell membrane","volume":"102","author":"Maciaszek","year":"2012","journal-title":"Biophys. J."},{"key":"ref_30","doi-asserted-by":"crossref","first-page":"1391","DOI":"10.1038\/nm.2507","article-title":"Ischemia and reperfusion from mechanism to translation","volume":"17","author":"Eltzschig","year":"2011","journal-title":"Nat. Med."},{"key":"ref_31","doi-asserted-by":"crossref","first-page":"1600","DOI":"10.1182\/blood-2017-12-819870","article-title":"HO-1hi patrolling monocytes protect against vaso-occlusion in sickle cell disease","volume":"131","author":"Liu","year":"2018","journal-title":"Blood"},{"key":"ref_32","doi-asserted-by":"crossref","first-page":"43","DOI":"10.1016\/j.cyto.2014.11.030","article-title":"Serum IL-6, IL-10, and TNF\u03b1 levels in pediatric sickle cell disease patients during vasoocclusive crisis and steady state condition","volume":"72","author":"Sarray","year":"2015","journal-title":"Cytokine"},{"key":"ref_33","doi-asserted-by":"crossref","unstructured":"Vona, R., Sposi, N.M., Mattia, L., Gambardella, L., Straface, E., and Pietraforte, D. (2021). Sickle cell disease: Role of oxidative stress and antioxidant therapy. Antioxidants, 10.","DOI":"10.3390\/antiox10020296"},{"key":"ref_34","doi-asserted-by":"crossref","first-page":"167","DOI":"10.1016\/j.neulet.2019.02.008","article-title":"Immune cell neural interactions and their contributions to sickle cell disease","volume":"699","author":"Sagi","year":"2019","journal-title":"Neurosci. Lett."},{"key":"ref_35","doi-asserted-by":"crossref","first-page":"263","DOI":"10.3233\/CH-189012","article-title":"Inflammation in sickle cell disease","volume":"68","author":"Conran","year":"2018","journal-title":"Clin. Hemorheol. Microcirc."},{"key":"ref_36","doi-asserted-by":"crossref","first-page":"237","DOI":"10.1111\/ejh.13430","article-title":"The vaso-occlusive pain crisis in sickle cell disease: Definition, pathophysiology, and management","volume":"105","author":"Darbari","year":"2020","journal-title":"Eur. J. Haematol."},{"key":"ref_37","doi-asserted-by":"crossref","first-page":"1397","DOI":"10.1172\/JCI45336","article-title":"CXCL1 and its receptor, CXCR2, mediate murine sickle cell vaso-occlusion during hemolytic transfusion reactions","volume":"121","author":"Jang","year":"2011","journal-title":"J. Clin. Investig."},{"key":"ref_38","doi-asserted-by":"crossref","first-page":"138","DOI":"10.1097\/MOH.0000000000000639","article-title":"Biophysical and rheological biomarkers of red blood cell physiology and pathophysiology","volume":"28","author":"Gurkan","year":"2021","journal-title":"Curr. Opin. Hematol."},{"key":"ref_39","doi-asserted-by":"crossref","first-page":"454","DOI":"10.3389\/fimmu.2020.00454","article-title":"The red blood cell-inflammation vicious circle in sickle cell disease","volume":"11","author":"Nader","year":"2020","journal-title":"Front. Immunol."},{"key":"ref_40","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.imlet.2020.04.012","article-title":"Immune mechanisms involved in sickle cell disease pathogenesis: Current knowledge and perspectives","volume":"224","author":"Malmegrim","year":"2020","journal-title":"Immunol. Lett."},{"key":"ref_41","doi-asserted-by":"crossref","first-page":"196","DOI":"10.5581\/1516-8484.20120049","article-title":"Sickle cell disease: Acute clinical manifestations in early childhood and molecular characteristics in a group of children in Rio de Janeiro","volume":"34","author":"Ribeiro","year":"2012","journal-title":"Rev. Bras. Hematol. Hemoter."},{"key":"ref_42","doi-asserted-by":"crossref","first-page":"408","DOI":"10.1111\/bjh.16437","article-title":"Curative options for sickle cell disease: Haploidentical stem cell transplantation or gene therapy?","volume":"189","author":"Leonard","year":"2020","journal-title":"Br. J. Haematol."},{"key":"ref_43","doi-asserted-by":"crossref","first-page":"1197","DOI":"10.1182\/blood-2011-01-332510","article-title":"Allogeneic hematopoietic stem cell transplantation for sickle cell disease: The time is now","volume":"118","author":"Hsieh","year":"2011","journal-title":"Blood"},{"key":"ref_44","doi-asserted-by":"crossref","first-page":"492","DOI":"10.1038\/s41409-020-01054-3","article-title":"Pre-transplant myeloid and immune suppression, upfront plerixafor mobilization and post-transplant cyclophosphamide: Novel strategy for haploidentical transplant in sickle cell disease","volume":"56","author":"Kharya","year":"2021","journal-title":"Bone Marrow Transplant."},{"key":"ref_45","unstructured":"Malik, P., Rees, D., Pirenne, F., Taher, A., Chakravorty, S., Tshilolo, L., Osunkwo, I., James, J., and Colombatti, R. (2020, January 11\u201321). The impact of sickle cell disease: Updates in therapeutics, treatment, management, and future directions. Proceedings of the The Virtual 25th European Hematology Association (EHA25), Frankfurt, Germany."},{"key":"ref_46","doi-asserted-by":"crossref","first-page":"303","DOI":"10.1586\/ehm.12.20","article-title":"Novel approaches to the treatment of sickle cell disease: The potential of histone deacetylase inhibitors","volume":"5","author":"Okam","year":"2012","journal-title":"Expert. Rev. Hematol."},{"key":"ref_47","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1177\/2040620720955000","article-title":"Current and novel therapies for the prevention of vaso-occlusive crisis in sickle cell disease","volume":"11","author":"Osunkwo","year":"2020","journal-title":"Ther. Adv. Hematol."},{"key":"ref_48","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1016\/j.yapd.2020.03.001","article-title":"Advances in sickle cell disease management","volume":"67","author":"Engel","year":"2020","journal-title":"Adv. Pediatr."},{"key":"ref_49","doi-asserted-by":"crossref","first-page":"273","DOI":"10.3324\/haematol.2019.229989","article-title":"Innate immune cells, major protagonists of sickle cell disease pathophysiology","volume":"105","author":"Allali","year":"2020","journal-title":"Haematologica"},{"key":"ref_50","doi-asserted-by":"crossref","first-page":"493","DOI":"10.1182\/asheducation-2018.1.493","article-title":"New insights into the pathophysiology and development of novel therapies for sickle cell disease","volume":"2018","author":"Moerdler","year":"2018","journal-title":"Hematol. Am. Soc. Hematol. Educ. Program."},{"key":"ref_51","doi-asserted-by":"crossref","first-page":"509","DOI":"10.1056\/NEJMoa1903212","article-title":"A phase 3 randomized trial of voxelotor in sickle cell disease","volume":"381","author":"Vichinsky","year":"2019","journal-title":"N. Engl. J. Med."},{"key":"ref_52","doi-asserted-by":"crossref","first-page":"e497","DOI":"10.3324\/haematol.2019.236182","article-title":"Safe and efficient peripheral blood stem cell collection in patients with sickle cell disease using plerixafor","volume":"105","author":"Uchida","year":"2020","journal-title":"Haematologica"},{"key":"ref_53","doi-asserted-by":"crossref","first-page":"1749","DOI":"10.1517\/14740338.2015.1088827","article-title":"Hydroxyurea therapy for sickle cell anemia","volume":"14","author":"McGann","year":"2015","journal-title":"Expert. Opin. Drug Saf."},{"key":"ref_54","doi-asserted-by":"crossref","first-page":"767","DOI":"10.1586\/17474086.2014.959922","article-title":"Hydroxyurea therapy contributes to infertility in adult men with sickle cell disease: A review","volume":"7","author":"DeBaun","year":"2014","journal-title":"Expert. Rev. Hematol."},{"key":"ref_55","doi-asserted-by":"crossref","first-page":"205","DOI":"10.1056\/NEJMoa2029392","article-title":"Post-transcriptional genetic silencing of BCL11A to treat sickle cell disease","volume":"384","author":"Esrick","year":"2020","journal-title":"N. Engl. J. Med."},{"key":"ref_56","doi-asserted-by":"crossref","first-page":"93","DOI":"10.1016\/j.blre.2016.10.002","article-title":"Recent discoveries in the molecular pathogenesis of the inherited bone marrow failure syndrome Fanconi anemia","volume":"31","author":"Mamrak","year":"2017","journal-title":"Blood Rev."},{"key":"ref_57","doi-asserted-by":"crossref","first-page":"893","DOI":"10.1038\/nrc2009","article-title":"Guido Fanconi (1892\u20131979): A jack of all trades","volume":"6","author":"Lobitz","year":"2006","journal-title":"Nat. Rev. Cancer"},{"key":"ref_58","unstructured":"Bhandari, J., Thada, P.K., and Puckett, Y. (2020). Fanconi anemia. StatPearls, Treasure Island."},{"key":"ref_59","doi-asserted-by":"crossref","first-page":"3013","DOI":"10.1172\/JCI92069","article-title":"Biallelic mutations in the ubiquitin ligase RFWD3 cause Fanconi anemia","volume":"127","author":"Knies","year":"2017","journal-title":"J. Clin. Investig."},{"key":"ref_60","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1016\/j.pbiomolbio.2020.08.005","article-title":"Fanconi anemia-independent DNA inter-strand crosslink repair in eukaryotes","volume":"158","author":"Rogers","year":"2020","journal-title":"Prog. Biophys. Mol. Biol."},{"key":"ref_61","doi-asserted-by":"crossref","first-page":"160","DOI":"10.3389\/fcell.2020.00160","article-title":"Fanconi anemia pathway: Mechanisms of breast cancer predisposition development and potential therapeutic targets","volume":"8","author":"Fang","year":"2020","journal-title":"Front. Cell Dev. Biol."},{"key":"ref_62","doi-asserted-by":"crossref","first-page":"337","DOI":"10.1038\/nrm.2016.48","article-title":"The Fanconi anaemia pathway: New players and new functions","volume":"17","author":"Ceccaldi","year":"2016","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"ref_63","doi-asserted-by":"crossref","first-page":"R986","DOI":"10.1016\/j.cub.2017.07.043","article-title":"Fanconi anemia pathway","volume":"27","year":"2017","journal-title":"Curr. Biol."},{"key":"ref_64","doi-asserted-by":"crossref","unstructured":"Milletti, G., Strocchio, L., Pagliara, D., Girardi, K., Carta, R., Mastronuzzi, A., Locatelli, F., and Nazio, F. (2020). Canonical and noncanonical roles of fanconi anemia proteins: Implications in cancer predisposition. Cancers, 12.","DOI":"10.3390\/cancers12092684"},{"key":"ref_65","doi-asserted-by":"crossref","first-page":"335","DOI":"10.1007\/s12185-017-2283-4","article-title":"Recent insights into the molecular basis of Fanconi anemia: Genes, modifiers, and drivers","volume":"106","author":"Cheung","year":"2017","journal-title":"Int. J. Hematol."},{"key":"ref_66","first-page":"00026","article-title":"The role of gene therapy in Fanconi anemia: A systematic and literature review with future directions","volume":"S1658-3876","author":"Shafqat","year":"2021","journal-title":"Hematol. Oncol. Stem Cell Ther."},{"key":"ref_67","first-page":"971","article-title":"Comparison of the clinical phenotype and haematological course of siblings with Fanconi anaemia","volume":"10","author":"Jung","year":"2020","journal-title":"Br. J. Haematol."},{"key":"ref_68","doi-asserted-by":"crossref","first-page":"492","DOI":"10.1182\/asheducation-2011.1.492","article-title":"Fanconi anemia","volume":"2011","author":"Soulier","year":"2011","journal-title":"Hematol. Am. Soc. Hematol. Educ. Program."},{"key":"ref_69","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1016\/j.mrfmmm.2009.01.013","article-title":"Fanconi anemia and its diagnosis","volume":"668","author":"Auerbach","year":"2009","journal-title":"Mutat. Res."},{"key":"ref_70","doi-asserted-by":"crossref","first-page":"147","DOI":"10.1053\/j.seminhematol.2006.04.005","article-title":"Fanconi anemia","volume":"43","author":"Bagby","year":"2006","journal-title":"Semin. Hematol."},{"key":"ref_71","doi-asserted-by":"crossref","first-page":"350","DOI":"10.1182\/blood-2004-01-0083","article-title":"Individualized risks of first adverse events in patients with Fanconi anemia","volume":"104","author":"Rosenberg","year":"2004","journal-title":"Blood"},{"key":"ref_72","doi-asserted-by":"crossref","first-page":"32","DOI":"10.1016\/j.gde.2015.07.002","article-title":"Fanconi anemia: A model disease for studies on human genetics and advanced therapeutics","volume":"33","author":"Bogliolo","year":"2015","journal-title":"Curr. Opin. Genet. Dev."},{"key":"ref_73","doi-asserted-by":"crossref","first-page":"134","DOI":"10.1080\/16078454.2021.1876393","article-title":"Hematopoietic stem cell transplantation for inherited bone marrow failure syndromes: Alternative donor and disease-specific conditioning regimen with unmanipulated grafts","volume":"26","author":"Lu","year":"2021","journal-title":"Hematology"},{"key":"ref_74","doi-asserted-by":"crossref","first-page":"348","DOI":"10.1159\/000502422","article-title":"HLA matching in pediatric stem cell transplantation","volume":"46","author":"Eyrich","year":"2019","journal-title":"Transfus. Med. Hemother."},{"key":"ref_75","doi-asserted-by":"crossref","first-page":"338","DOI":"10.2174\/1566523217666170119113029","article-title":"Lessons learned from two decades of clinical trial experience in gene therapy for Fanconi anemia","volume":"16","author":"Adair","year":"2016","journal-title":"Curr. Gene Ther."},{"key":"ref_76","doi-asserted-by":"crossref","first-page":"297","DOI":"10.2174\/1566523217666170109114309","article-title":"Gene therapy in Fanconi anemia: A matter of time, safety and gene transfer tool efficiency","volume":"16","author":"Verhoeyen","year":"2017","journal-title":"Curr. Gene Ther."},{"key":"ref_77","first-page":"1","article-title":"Effective CRISPR\/Cas9-mediated correction of a Fanconi anemia defect by error-prone end joining or templated repair","volume":"9","author":"Harmsen","year":"2019","journal-title":"Sci. Rep."},{"key":"ref_78","doi-asserted-by":"crossref","first-page":"2270","DOI":"10.1172\/JCI133530","article-title":"Donor glucose-6-phosphate dehydrogenase deficiency decreases blood quality for transfusion","volume":"130","author":"Francis","year":"2020","journal-title":"J. Clin. Investig."},{"key":"ref_79","doi-asserted-by":"crossref","unstructured":"Ryan, K., and Tekwani, B.L. (2020). Current investigations on clinical pharmacology and therapeutics of glucose-6-phosphate dehydrogenase deficiency. Pharmacol. Ther., 107788.","DOI":"10.1016\/j.pharmthera.2020.107788"},{"key":"ref_80","doi-asserted-by":"crossref","first-page":"47","DOI":"10.3329\/fmcj.v12i1.33491","article-title":"Glucose-6-phosphate dehydrogenase deficiency: A case report","volume":"12","author":"Hassan","year":"2017","journal-title":"Faridpur Med. Coll J."},{"key":"ref_81","doi-asserted-by":"crossref","first-page":"1","DOI":"10.3389\/fphar.2021.638885","article-title":"G6PD variants and haemolytic sensitivity to primaquine and other drugs","volume":"12","author":"Bancone","year":"2021","journal-title":"Front. Pharmacol."},{"key":"ref_82","doi-asserted-by":"crossref","first-page":"484","DOI":"10.1126\/science.124.3220.484.b","article-title":"Enzymatic deficiency in primaquine-sensitive erythrocytes","volume":"124","author":"Carson","year":"1956","journal-title":"Science"},{"key":"ref_83","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1002\/humu.10036","article-title":"G6PDdb, an integrated database of glucose-6-phosphate dehydrogenase (G6PD) mutations","volume":"19","author":"Kwok","year":"2002","journal-title":"Hum. Mutat."},{"key":"ref_84","doi-asserted-by":"crossref","first-page":"1849","DOI":"10.1002\/j.1460-2075.1986.tb04436.x","article-title":"Structural analysis of the X-linked gene encoding human glucose 6-phosphate dehydrogenase","volume":"5","author":"Martini","year":"1986","journal-title":"EMBO J."},{"key":"ref_85","doi-asserted-by":"crossref","first-page":"1028","DOI":"10.3109\/10715762.2014.913788","article-title":"Glucose-6-phosphate dehydrogenase\u2013beyond the realm of red cell biology","volume":"48","author":"Ho","year":"2014","journal-title":"Free Radic. Res."},{"key":"ref_86","first-page":"137","article-title":"Glucose-6-phosphate dehydrogenase deficiency in the newborn: Its prevalence and relation to neonatal jaundice","volume":"26","author":"Tanphaichitr","year":"1995","journal-title":"Southeast Asian J. Trop Med. Public Health"},{"key":"ref_87","doi-asserted-by":"crossref","first-page":"42","DOI":"10.4314\/eamj.v79i1.8924","article-title":"Molecular epidemiology and activity of erythrocyte G6PD variants in a homogenous Nigerian population","volume":"79","author":"Ademowo","year":"2002","journal-title":"East Afr. Med. J."},{"key":"ref_88","first-page":"1277","article-title":"Diagnosis and management of G6PD deficiency","volume":"72","author":"Frank","year":"2005","journal-title":"Am. Fam. Physician"},{"key":"ref_89","first-page":"97","article-title":"Review and drug therapy implications of glucose-6-phosphate dehydrogenase deficiency","volume":"75","author":"Belfield","year":"2018","journal-title":"Am. J. Hosp. Pharm."},{"key":"ref_90","doi-asserted-by":"crossref","first-page":"373","DOI":"10.1016\/j.hoc.2015.11.006","article-title":"Glucose-6-phosphate dehydrogenase deficiency","volume":"30","author":"Luzzatto","year":"2016","journal-title":"Hematol. Oncol. Clin. N. Am."},{"key":"ref_91","doi-asserted-by":"crossref","first-page":"64","DOI":"10.1016\/S0140-6736(08)60073-2","article-title":"Glucose-6-phosphate dehydrogenase deficiency","volume":"371","author":"Cappellini","year":"2008","journal-title":"Lancet"},{"key":"ref_92","doi-asserted-by":"crossref","unstructured":"G\u00f3mez-Manzo, S., Marcial-Quino, J., Vanoye-Carlo, A., Serrano-Posada, H., Ortega-Cuellar, D., Gonz\u00e1lez-Valdez, A., Castillo-Rodr\u00edguez, R.A., Hern\u00e1ndez-Ochoa, B., Sierra-Palacios, E., and Rodr\u00edguez-Bustamante, E. (2016). Glucose-6-phosphate dehydrogenase: Update and analysis of new mutations around the world. Int. J. Mol. Sci., 17.","DOI":"10.3390\/ijms17122069"},{"key":"ref_93","doi-asserted-by":"crossref","first-page":"e11553","DOI":"10.1097\/MD.0000000000011553","article-title":"Mutation of glucose-6-phosphate dehydrogenase deficiency in Chinese Han children in eastern Fujian","volume":"97","author":"Chen","year":"2018","journal-title":"Medicine"},{"key":"ref_94","doi-asserted-by":"crossref","first-page":"6680925","DOI":"10.1155\/2021\/6680925","article-title":"Genotype-phenotype correlation of G6PD mutations among central Thai children with G6PD deficiency","volume":"2021","author":"Boonyawat","year":"2021","journal-title":"Anemia"},{"key":"ref_95","doi-asserted-by":"crossref","unstructured":"Pfeffer, D.A., Ley, B., Howes, R.E., Adu, P., Alam, M.S., Bansil, P., Boum, Y., Brito, M., Charoenkwan, P., and Clements, A. (2020). Quantification of glucose-6-phosphate dehydrogenase activity by spectrophotometry: A systematic review and meta-analysis. PLoS Med., 17.","DOI":"10.1371\/journal.pmed.1003311"},{"key":"ref_96","doi-asserted-by":"crossref","unstructured":"Islam, M.T., Sarker, S.K., Talukder, S., Bhuyan, G.S., Rahat, A., Islam, N.N., Mahmud, H., Hossain, M.A., Muraduzzaman, A., and Rahman, J. (2018). High resolution melting curve analysis enables rapid and reliable detection of G6PD variants in heterozygous females. BMC Genet., 19.","DOI":"10.1186\/s12863-018-0664-1"},{"key":"ref_97","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1038\/s41467-018-06447-z","article-title":"Correcting glucose-6-phosphate dehydrogenase deficiency with a small-molecule activator","volume":"9","author":"Hwang","year":"2018","journal-title":"Nat. Commun."},{"key":"ref_98","unstructured":"Dean, L., and Kane, M. (2012). Pegloticase therapy and G6PD genotype, Medical Genetics Summaries."},{"key":"ref_99","unstructured":"Dean, L., and Kane, M. (2012). Rasburicase therapy and G6PD and CYB5R genotype, Medical Genetics Summaries."},{"key":"ref_100","unstructured":"Dean, L., and Kane, M. (2012). Tafenoquine therapy and G6PD genotype, Medical Genetics Summaries."},{"key":"ref_101","doi-asserted-by":"crossref","first-page":"1053","DOI":"10.1007\/s40265-020-01341-9","article-title":"Beta thalassemia: New therapeutic options beyond transfusion and iron chelation","volume":"80","author":"Motta","year":"2020","journal-title":"Drugs"},{"key":"ref_102","doi-asserted-by":"crossref","first-page":"165","DOI":"10.1016\/j.hoc.2017.11.008","article-title":"The evolving spectrum of the epidemiology of thalassemia","volume":"32","author":"Weatherall","year":"2018","journal-title":"Hematol. Oncol. Clin. N. Am."},{"key":"ref_103","doi-asserted-by":"crossref","first-page":"322","DOI":"10.1016\/j.clinthera.2019.12.003","article-title":"Systematic literature review of the burden of disease and treatment for transfusion-dependent \u03b2-thalassemia","volume":"42","author":"Betts","year":"2020","journal-title":"Clin. Ther."},{"key":"ref_104","doi-asserted-by":"crossref","first-page":"837","DOI":"10.1016\/S2222-1808(16)61142-8","article-title":"Thalassemia: Impact of consanguineous marriages on most prevalent monogenic disorders of humans","volume":"6","author":"Saeed","year":"2016","journal-title":"Asian Pac. J. Trop Dis."},{"key":"ref_105","doi-asserted-by":"crossref","unstructured":"Apidechkul, T., Yeemard, F., Chomchoei, C., Upala, P., and Tamornpark, R. (2021). Epidemiology of thalassemia among the hill tribe population in Thailand. PLoS ONE, 16.","DOI":"10.1371\/journal.pone.0246736"},{"key":"ref_106","doi-asserted-by":"crossref","unstructured":"Hassan, T., and Zakaria, M. (2020). Updates in thalassemia. Beta Thalassemia, IntechOpen.","DOI":"10.5772\/intechopen.92414"},{"key":"ref_107","doi-asserted-by":"crossref","first-page":"160","DOI":"10.1182\/hematology.2020000102","article-title":"Challenges in chronic transfusion for patients with thalassemia","volume":"2020","author":"Lal","year":"2020","journal-title":"Hematol. Am. Soc. Hematol. Educ. Program."},{"key":"ref_108","doi-asserted-by":"crossref","first-page":"100588","DOI":"10.1016\/j.blre.2019.100588","article-title":"Challenges of blood transfusions in \u03b2-thalassemia","volume":"37","author":"Shah","year":"2019","journal-title":"Blood Rev."},{"key":"ref_109","doi-asserted-by":"crossref","first-page":"319","DOI":"10.1007\/s12098-021-03698-5","article-title":"Dual oral iron chelation in thalassemia: Need for obust evidence","volume":"88","author":"Peyam","year":"2021","journal-title":"Indian J. Pediatr."},{"key":"ref_110","doi-asserted-by":"crossref","first-page":"114","DOI":"10.1186\/s13023-021-01757-w","article-title":"A comprehensive review of hydroxyurea for \u03b2-haemoglobinopathies: The role revisited during COVID-19 pandemic","volume":"16","author":"Yasara","year":"2021","journal-title":"Orphanet. J. Rare Dis."},{"key":"ref_111","doi-asserted-by":"crossref","first-page":"406","DOI":"10.1080\/03630269.2020.1845722","article-title":"Association of exon 14 of the SOX6 gene sequence variations with response to hydroxyurea therapy in patients carrying non transfusion-dependent thalassemia","volume":"44","author":"Mohammadi","year":"2020","journal-title":"Hemoglobin"},{"key":"ref_112","doi-asserted-by":"crossref","first-page":"288","DOI":"10.1186\/s13287-020-01765-w","article-title":"The safety and effectiveness of genetically corrected iPSCs derived from \u03b2-thalassaemia patients in nonmyeloablative \u03b2-thalassaemic mice","volume":"11","author":"Xian","year":"2020","journal-title":"Stem Cell Res. Ther."},{"key":"ref_113","doi-asserted-by":"crossref","first-page":"1106","DOI":"10.1016\/j.bbmt.2020.01.002","article-title":"Hematopoietic stem cell transplantation for severe thalassemia patients from haploidentical donors using a novel conditioning regimen","volume":"26","author":"Anurathapan","year":"2020","journal-title":"Biol. Blood Marrow. Transplant."},{"key":"ref_114","doi-asserted-by":"crossref","first-page":"589","DOI":"10.1016\/j.omtm.2020.03.015","article-title":"Preclinical evaluation of a novel lentiviral vector driving lineage-specific BCL11A knockdown for sickle cell gene therapy","volume":"17","author":"Brendel","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"ref_115","doi-asserted-by":"crossref","first-page":"570","DOI":"10.1182\/bloodadvances.2020002948","article-title":"A systematic review of quality of life in sickle cell disease and thalassemia after stem cell transplant or gene therapy","volume":"5","author":"Badawy","year":"2021","journal-title":"Blood Adv."},{"key":"ref_116","doi-asserted-by":"crossref","first-page":"252","DOI":"10.1056\/NEJMoa2031054","article-title":"CRISPR-Cas9 gene editing for sickle cell disease and \u03b2-thalassemia","volume":"384","author":"Frangoul","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"ref_117","doi-asserted-by":"crossref","first-page":"48","DOI":"10.3892\/br.2020.1355","article-title":"Novel genetic therapeutic approaches for modulating the severity of \u03b2-thalassemia (Review)","volume":"13","author":"Amjad","year":"2020","journal-title":"Biomed. Rep."},{"key":"ref_118","doi-asserted-by":"crossref","first-page":"570","DOI":"10.1016\/j.ajhg.2020.02.016","article-title":"De novo EIF2AK1 and EIF2AK2 variants are associated with developmental delay, leukoencephalopathy, and neurologic decompensation","volume":"106","author":"Mao","year":"2020","journal-title":"Am. J. Hum. Genet."},{"key":"ref_119","doi-asserted-by":"crossref","first-page":"111973","DOI":"10.1016\/j.ejmech.2019.111973","article-title":"New activators of eIF2\u03b1 kinase heme-regulated inhibitor (HRI) with improved biophysical properties","volume":"187","author":"Zhang","year":"2020","journal-title":"Eur. J. Med. Chem."},{"key":"ref_120","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1038\/s41467-018-04894-2","article-title":"In utero nanoparticle delivery for site-specific genome editing","volume":"9","author":"Ricciardi","year":"2018","journal-title":"Nat. Commun."},{"key":"ref_121","doi-asserted-by":"crossref","unstructured":"Ricciardi, A.S., Quijano, E., Putman, R., Saltzman, W.M., and Glazer, P.M. (2018). Peptide nucleic acids as a tool for site-specific gene editing. Molecules, 23.","DOI":"10.3390\/molecules23030632"},{"key":"ref_122","doi-asserted-by":"crossref","unstructured":"Economos, N.G., Oyaghire, S., Quijano, E., Ricciardi, A.S., Saltzman, W.M., and Glazer, P.M. (2020). Peptide nucleic acids and gene editing: Perspectives on structure and repair. Molecules, 25.","DOI":"10.3390\/molecules25030735"},{"key":"ref_123","doi-asserted-by":"crossref","first-page":"295","DOI":"10.1089\/hum.2016.037","article-title":"Cell and gene therapy for the beta-thalassemias: Advances and prospects","volume":"27","author":"Riviere","year":"2016","journal-title":"Hum. Gene Ther."},{"key":"ref_124","doi-asserted-by":"crossref","first-page":"167","DOI":"10.2147\/TACG.S178546","article-title":"Gene therapy for beta-thalassemia: Updated perspectives","volume":"12","author":"Karponi","year":"2019","journal-title":"Appl. Clin. Genet."},{"key":"ref_125","doi-asserted-by":"crossref","first-page":"1551","DOI":"10.1056\/NEJMe1802169","article-title":"Gene therapy as a curative option for \u03b2-thalassemia","volume":"378","author":"Biffi","year":"2018","journal-title":"N. Engl. J. Med."},{"key":"ref_126","doi-asserted-by":"crossref","first-page":"1479","DOI":"10.1056\/NEJMoa1705342","article-title":"Gene therapy in patients with transfusion-dependent \u03b2-thalassemia","volume":"378","author":"Thompson","year":"2018","journal-title":"N. Engl. J. Med."},{"key":"ref_127","doi-asserted-by":"crossref","first-page":"1114","DOI":"10.1089\/hum.2018.124","article-title":"Advances in gene therapy for Fanconi anemia","volume":"29","author":"Navarro","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"ref_128","doi-asserted-by":"crossref","first-page":"2021","DOI":"10.1182\/blood.2020009111","article-title":"Analysis of disease model iPSCs derived from patients with a novel Fanconi anemia-like IBMFS ADH5\/ALDH2 deficiency","volume":"137","author":"Mu","year":"2021","journal-title":"Blood"},{"key":"ref_129","first-page":"1","article-title":"iPSC modeling of severe aplastic anemia reveals impaired differentiation and telomere shortening in blood progenitors","volume":"9","author":"Xu","year":"2018","journal-title":"Cell Death Dis."},{"key":"ref_130","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1242\/jcs.203018","article-title":"Hematopoietic stem cells from induced pluripotent stem cells\u2013considering the role of microRNA as a cell differentiation regulator","volume":"131","author":"Ferreira","year":"2018","journal-title":"J. Cell Sci."},{"key":"ref_131","doi-asserted-by":"crossref","first-page":"1920","DOI":"10.1126\/science.1152092","article-title":"Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin","volume":"318","author":"Hanna","year":"2007","journal-title":"Science"},{"key":"ref_132","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1016\/S0092-8674(02)00681-5","article-title":"Correction of a genetic defect by nuclear transplantation and combined cell and gene therapy","volume":"109","author":"Hochedlinger","year":"2002","journal-title":"Cell"},{"key":"ref_133","doi-asserted-by":"crossref","first-page":"631","DOI":"10.1016\/j.cell.2008.01.025","article-title":"Hematopoiesis: An evolving paradigm for stem cell biology","volume":"132","author":"Orkin","year":"2008","journal-title":"Cell"},{"key":"ref_134","doi-asserted-by":"crossref","first-page":"85","DOI":"10.1016\/j.stem.2008.06.001","article-title":"Efficient hematopoietic differentiation of human embryonic stem cells on stromal cells derived from hematopoietic niches","volume":"3","author":"Ledran","year":"2008","journal-title":"Cell Stem Cell"},{"key":"ref_135","doi-asserted-by":"crossref","first-page":"1502","DOI":"10.1172\/JCI34371","article-title":"High incidence of leukemia in large animals after stem cell gene therapy with a HOXB4-expressing retroviral vector","volume":"118","author":"Zhang","year":"2008","journal-title":"J. Clin. Investig."},{"key":"ref_136","doi-asserted-by":"crossref","first-page":"3748","DOI":"10.1182\/blood-2010-07-298596","article-title":"In vitro generation of HSC-like cells from murine ESCs\/iPSCs by enforced expression of LIM-homeobox transcription factor Lhx2","volume":"117","author":"Kitajima","year":"2011","journal-title":"Blood"},{"key":"ref_137","doi-asserted-by":"crossref","first-page":"2180","DOI":"10.1073\/pnas.1718446115","article-title":"Respecifying human iPSC-derived blood cells into highly engraftable hematopoietic stem and progenitor cells with a single factor","volume":"115","author":"Tan","year":"2018","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_138","doi-asserted-by":"crossref","first-page":"1024","DOI":"10.1016\/j.stemcr.2017.08.010","article-title":"In vivo generation of engraftable murine hematopoietic stem cells by Gfi1b, c-Fos, and Gata2 overexpression within teratoma","volume":"9","author":"Tsukada","year":"2017","journal-title":"Stem Cell Rep."},{"key":"ref_139","doi-asserted-by":"crossref","first-page":"194","DOI":"10.5045\/br.2015.50.4.194","article-title":"Hematopoietic stem cell expansion and generation: The ways to make a breakthrough","volume":"50","author":"Park","year":"2015","journal-title":"Blood Res."},{"key":"ref_140","doi-asserted-by":"crossref","first-page":"1243","DOI":"10.1172\/JCI79328","article-title":"Vascular niche promotes hematopoietic multipotent progenitor formation from pluripotent stem cells","volume":"125","author":"Gori","year":"2015","journal-title":"J. Clin. Investig."},{"key":"ref_141","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1038\/s41422-019-0251-7","article-title":"Guiding T lymphopoiesis from pluripotent stem cells by defined transcription factors","volume":"30","author":"Guo","year":"2020","journal-title":"Cell Res."},{"key":"ref_142","doi-asserted-by":"crossref","first-page":"459","DOI":"10.1016\/j.stem.2013.09.002","article-title":"Induction of multipotential hematopoietic progenitors from human pluripotent stem cells via respecification of lineage-restricted precursors","volume":"13","author":"Doulatov","year":"2013","journal-title":"Cell Stem Cell"},{"key":"ref_143","doi-asserted-by":"crossref","first-page":"1255","DOI":"10.1182\/blood-2012-06-434407","article-title":"In vivo generation of transplantable human hematopoietic cells from induced pluripotent stem cells","volume":"121","author":"Amabile","year":"2013","journal-title":"Blood"},{"key":"ref_144","doi-asserted-by":"crossref","first-page":"1424","DOI":"10.1038\/mt.2013.71","article-title":"Generation of engraftable hematopoietic stem cells from induced pluripotent stem cells by way of teratoma formation","volume":"21","author":"Suzuki","year":"2013","journal-title":"Mol. Ther."},{"key":"ref_145","doi-asserted-by":"crossref","unstructured":"Lu, S.-J., Feng, Q., and Lanza, R. (2015). Generation of hemangioblasts from human pluripotent stem cells. Hematopoietic Differentiation of Human Pluripotent Stem Cells, Springer.","DOI":"10.1007\/978-94-017-7312-6_1"},{"key":"ref_146","doi-asserted-by":"crossref","first-page":"413","DOI":"10.2217\/rme.13.36","article-title":"3D microcarrier system for efficient differentiation of human induced pluripotent stem cells into hematopoietic cells without feeders and serum","volume":"8","author":"Lu","year":"2013","journal-title":"Regen. Med."},{"key":"ref_147","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1007\/s12015-019-09935-x","article-title":"Advances in pluripotent stem cells: History, mechanisms, technologies, and applications","volume":"16","author":"Liu","year":"2020","journal-title":"Stem Cell Rev. Rep."},{"key":"ref_148","doi-asserted-by":"crossref","first-page":"e36","DOI":"10.1002\/cpet.36","article-title":"Genome editing with CRISPR-Cas: An overview","volume":"19","author":"Robb","year":"2019","journal-title":"Curr. Protoc. Essent. Lab. Tech."},{"key":"ref_149","doi-asserted-by":"crossref","first-page":"535","DOI":"10.1038\/s41591-020-0790-y","article-title":"Therapeutic base editing of human hematopoietic stem cells","volume":"26","author":"Zeng","year":"2020","journal-title":"Nat. Med."},{"key":"ref_150","doi-asserted-by":"crossref","first-page":"1059","DOI":"10.1007\/s00439-016-1678-2","article-title":"TALEN gene editing takes aim on HIV","volume":"135","author":"Benjamin","year":"2016","journal-title":"Hum. Genet."},{"key":"ref_151","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1038\/s41392-019-0089-y","article-title":"Applications of genome editing technology in the targeted therapy of human diseases: Mechanisms, advances and prospects","volume":"5","author":"Li","year":"2020","journal-title":"Signal. Transduct. Target. Ther."},{"key":"ref_152","doi-asserted-by":"crossref","first-page":"6762","DOI":"10.1093\/nar\/gku305","article-title":"TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity","volume":"42","author":"Mussolino","year":"2014","journal-title":"Nucleic Acids Res."},{"key":"ref_153","doi-asserted-by":"crossref","first-page":"1234","DOI":"10.1080\/10717544.2018.1474964","article-title":"Delivering CRISPR: A review of the challenges and approaches","volume":"25","author":"Lino","year":"2018","journal-title":"Drug Deliv."},{"key":"ref_154","doi-asserted-by":"crossref","first-page":"490","DOI":"10.1038\/s41580-019-0131-5","article-title":"The next generation of CRISPR\u2013Cas technologies and applications","volume":"20","author":"Gersbach","year":"2019","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"ref_155","doi-asserted-by":"crossref","first-page":"35","DOI":"10.1016\/j.sbi.2021.02.005","article-title":"CRISPR\/Cas9-based directed evolution in mammalian cells","volume":"69","author":"Griesbeck","year":"2021","journal-title":"Curr. Opin. Struct. Biol."},{"key":"ref_156","doi-asserted-by":"crossref","first-page":"12267","DOI":"10.1002\/jcp.27972","article-title":"CRISPR\/Cas9 technology as a potent molecular tool for gene therapy","volume":"234","author":"Karimian","year":"2019","journal-title":"J. Cell Physiol."},{"key":"ref_157","doi-asserted-by":"crossref","first-page":"e1609","DOI":"10.1002\/wnan.1609","article-title":"Strategies for nonviral nanoparticle-based delivery of CRISPR\/Cas9 therapeutics","volume":"12","author":"Chen","year":"2020","journal-title":"Wiley Interdiscip. Rev. Nanomed. Nanobiotechnol."},{"key":"ref_158","doi-asserted-by":"crossref","first-page":"207","DOI":"10.1016\/j.biomaterials.2018.04.031","article-title":"Non-viral delivery systems for CRISPR\/Cas9-based genome editing: Challenges and opportunities","volume":"171","author":"Li","year":"2018","journal-title":"Biomaterials"},{"key":"ref_159","doi-asserted-by":"crossref","first-page":"894","DOI":"10.1093\/cvr\/cvz250","article-title":"CRISPR\/Cas9 gene-editing strategies in cardiovascular cells","volume":"116","author":"Vermersch","year":"2020","journal-title":"Cardiovasc. Res."},{"key":"ref_160","first-page":"931","article-title":"CRISPR-Cas9, a promising therapeutic tool for cancer therapy: A review","volume":"27","author":"Akram","year":"2020","journal-title":"Protein Pept. Lett."},{"key":"ref_161","doi-asserted-by":"crossref","unstructured":"Vuelta, E., Garc\u00eda-Tu\u00f1\u00f3n, I., Hern\u00e1ndez-Carabias, P., M\u00e9ndez, L., and S\u00e1nchez-Mart\u00edn, M. (2021). Future approaches for treating chronic myeloid leukemia: CRISPR therapy. Biology, 10.","DOI":"10.20944\/preprints202101.0212.v1"},{"key":"ref_162","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1007\/s13205-021-02680-4","article-title":"Genome-editing approaches and applications: A brief review on CRISPR technology and its role in cancer","volume":"11","author":"Siva","year":"2021","journal-title":"3 Biotech"},{"key":"ref_163","doi-asserted-by":"crossref","first-page":"773","DOI":"10.1534\/genetics.111.131433","article-title":"Genome engineering with zinc-finger nucleases","volume":"188","author":"Carroll","year":"2011","journal-title":"Genetics"},{"key":"ref_164","doi-asserted-by":"crossref","first-page":"636","DOI":"10.1038\/nrg2842","article-title":"Genome editing with engineered zinc finger nucleases","volume":"11","author":"Urnov","year":"2010","journal-title":"Nat. Rev. Genet."},{"key":"ref_165","doi-asserted-by":"crossref","first-page":"967","DOI":"10.1038\/nbt1125","article-title":"Gene targeting using zinc finger nucleases","volume":"23","author":"Porteus","year":"2005","journal-title":"Nat. Biotechnol."},{"key":"ref_166","doi-asserted-by":"crossref","first-page":"135","DOI":"10.1146\/annurev-genom-091212-153435","article-title":"Genome engineering at the dawn of the golden age","volume":"14","author":"Segal","year":"2013","journal-title":"Annu. Rev. Genom. Hum. Genet."},{"key":"ref_167","doi-asserted-by":"crossref","first-page":"1200","DOI":"10.1038\/mt.2008.114","article-title":"Zinc-finger nucleases: The next generation emerges","volume":"16","author":"Cathomen","year":"2008","journal-title":"Mol. Ther."},{"key":"ref_168","doi-asserted-by":"crossref","first-page":"397","DOI":"10.1016\/j.tibtech.2013.04.004","article-title":"ZFN, TALEN, and CRISPR\/Cas-based methods for genome engineering","volume":"31","author":"Gaj","year":"2013","journal-title":"Trends Biotechnol."},{"key":"ref_169","doi-asserted-by":"crossref","first-page":"11","DOI":"10.2174\/156652311794520111","article-title":"Meganucleases and other tools for targeted genome engineering: Perspectives and challenges for gene therapy","volume":"11","author":"Silva","year":"2011","journal-title":"Curr. Gene Ther."},{"key":"ref_170","doi-asserted-by":"crossref","first-page":"49","DOI":"10.2174\/156652307779940216","article-title":"Meganucleases and DNA double-strand break-induced recombination: Perspectives for gene therapy","volume":"7","author":"Duchateau","year":"2007","journal-title":"Curr. Gene Ther."},{"key":"ref_171","doi-asserted-by":"crossref","unstructured":"Tr\u00f6der, S.E., and Zevnik, B. (2021). History of genome editing: From meganucleases to CRISPR. Lab. Anim., 0023677221994613.","DOI":"10.1177\/0023677221994613"},{"key":"ref_172","doi-asserted-by":"crossref","first-page":"729","DOI":"10.1093\/nar\/gkq801","article-title":"Molecular basis of engineered meganuclease targeting of the endogenous human RAG1 locus","volume":"39","author":"Munoz","year":"2011","journal-title":"Nucleic Acids Res."},{"key":"ref_173","doi-asserted-by":"crossref","first-page":"315","DOI":"10.1093\/toxsci\/kfw220","article-title":"Viral vectors: The road to reducing genotoxicity","volume":"155","author":"David","year":"2017","journal-title":"Toxicol. Sci."},{"key":"ref_174","doi-asserted-by":"crossref","first-page":"634","DOI":"10.1016\/j.stem.2018.10.003","article-title":"The harmful consequences of increased fitness in hematopoietic stem cells","volume":"23","author":"McNerney","year":"2018","journal-title":"Cell Stem Cell"},{"key":"ref_175","doi-asserted-by":"crossref","first-page":"e131698","DOI":"10.1172\/JCI131698","article-title":"Concerted roles of PTEN and ATM in controlling hematopoietic stem cell fitness and dormancy","volume":"131","author":"Fortin","year":"2021","journal-title":"J. Clin. Investig."},{"key":"ref_176","doi-asserted-by":"crossref","unstructured":"Martin, F., S\u00e1nchez-Hern\u00e1ndez, S., Guti\u00e9rrez-Guerrero, A., Pinedo-Gomez, J., and Benabdellah, K. (2016). Biased and unbiased methods for the detection of off-target cleavage by CRISPR\/Cas9: An overview. Int. J. Mol. Sci., 17.","DOI":"10.3390\/ijms17091507"},{"key":"ref_177","doi-asserted-by":"crossref","first-page":"24751","DOI":"10.3390\/ijms161024751","article-title":"Minimizing off-target mutagenesis risks caused by programmable nucleases","volume":"16","author":"Ishida","year":"2015","journal-title":"Int. J. Mol. Sci."},{"key":"ref_178","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1016\/j.ymeth.2018.11.016","article-title":"Current strategies for site-directed RNA editing using ADARs","volume":"156","author":"Quiroz","year":"2019","journal-title":"Methods"},{"key":"ref_179","doi-asserted-by":"crossref","unstructured":"Baba\u010di\u0107, H., Mehta, A., Merkel, O., and Schoser, B. (2019). CRISPR-cas gene-editing as plausible treatment of neuromuscular and nucleotide-repeat-expansion diseases: A systematic review. PLoS ONE, 14.","DOI":"10.1371\/journal.pone.0212198"},{"key":"ref_180","doi-asserted-by":"crossref","first-page":"504","DOI":"10.1080\/15476286.2018.1504546","article-title":"PAM identification by CRISPR-Cas effector complexes: Diversified mechanisms and structures","volume":"16","author":"Gleditzsch","year":"2019","journal-title":"RNA Biol."},{"key":"ref_181","doi-asserted-by":"crossref","unstructured":"Kantor, A., McClements, M.E., and MacLaren, R.E. (2020). CRISPR-Cas9 DNA base-editing and prime-editing. Int. J. Mol. Sci., 21.","DOI":"10.3390\/ijms21176240"},{"key":"ref_182","doi-asserted-by":"crossref","first-page":"1902312","DOI":"10.1002\/advs.201902312","article-title":"CRISPR\/Cas systems in genome editing: Methodologies and tools for sgRNA design, off-target evaluation, and strategies to mitigate off-target effects","volume":"7","author":"Manghwar","year":"2020","journal-title":"Adv. Sci."},{"key":"ref_183","doi-asserted-by":"crossref","first-page":"555","DOI":"10.1038\/s41467-020-20633-y","article-title":"CRISPR technologies and the search for the PAM-free nuclease","volume":"12","author":"Collias","year":"2021","journal-title":"Nat. Commun."},{"key":"ref_184","doi-asserted-by":"crossref","first-page":"1","DOI":"10.3390\/foods10020430","article-title":"Genome-edited plants: Opportunities and challenges for an anticipatory detection and identification framework","volume":"10","author":"Ribarits","year":"2021","journal-title":"Foods"},{"key":"ref_185","doi-asserted-by":"crossref","first-page":"468","DOI":"10.1016\/j.ymthe.2017.11.001","article-title":"Characterization of gene alterations following editing of the \u03b2-globin gene locus in hematopoietic stem\/progenitor cells","volume":"26","author":"Long","year":"2018","journal-title":"Mol. Ther."},{"key":"ref_186","doi-asserted-by":"crossref","first-page":"286","DOI":"10.1126\/science.aav9023","article-title":"Unbiased detection of CRISPR off-targets in vivo using DISCOVER-Seq","volume":"364","author":"Wienert","year":"2019","journal-title":"Science"},{"key":"ref_187","doi-asserted-by":"crossref","first-page":"416","DOI":"10.1038\/s41586-018-0500-9","article-title":"In vivo CRISPR editing with no detectable genome-wide off-target mutations","volume":"561","author":"Akcakaya","year":"2018","journal-title":"Nature"},{"key":"ref_188","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1038\/ncomms12463","article-title":"Non-homologous DNA increases gene disruption efficiency by altering DNA repair outcomes","volume":"7","author":"Richardson","year":"2016","journal-title":"Nat. Commun."},{"key":"ref_189","doi-asserted-by":"crossref","first-page":"18","DOI":"10.1038\/s41421-019-0088-8","article-title":"Optimizing genome editing strategy by primer-extension-mediated sequencing","volume":"5","author":"Yin","year":"2019","journal-title":"Cell Discov."},{"key":"ref_190","doi-asserted-by":"crossref","first-page":"i757","DOI":"10.1093\/bioinformatics\/bty558","article-title":"Recognition of CRISPR\/Cas9 off-target sites through ensemble learning of uneven mismatch distributions","volume":"34","author":"Peng","year":"2018","journal-title":"Bioinformatics"},{"key":"ref_191","doi-asserted-by":"crossref","first-page":"651","DOI":"10.1021\/acscentsci.9b00020","article-title":"Deciphering off-target effects in CRISPR-Cas9 through accelerated molecular dynamics","volume":"5","author":"Ricci","year":"2019","journal-title":"ACS Cent. Sci."},{"key":"ref_192","doi-asserted-by":"crossref","first-page":"614","DOI":"10.7150\/thno.47007","article-title":"Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR\/Cas9 genome editing","volume":"11","author":"Zhang","year":"2021","journal-title":"Theranostics"},{"key":"ref_193","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1111\/bjh.16504","article-title":"Emerging therapies in sickle cell disease","volume":"190","author":"Brousse","year":"2020","journal-title":"Br. J. Haematol."},{"key":"ref_194","doi-asserted-by":"crossref","first-page":"435","DOI":"10.3389\/fphys.2020.00435","article-title":"Recent advances in the treatment of sickle cell disease","volume":"11","author":"Thein","year":"2020","journal-title":"Front. Physiol."},{"key":"ref_195","doi-asserted-by":"crossref","first-page":"547","DOI":"10.1080\/17474086.2018.1486703","article-title":"Stem cell transplantation in sickle cell disease: Therapeutic potential and challenges faced","volume":"11","author":"Leonard","year":"2018","journal-title":"Expert. Rev. Hematol."},{"key":"ref_196","doi-asserted-by":"crossref","first-page":"131","DOI":"10.31486\/toj.18.0044","article-title":"Curative therapies for sickle cell disease","volume":"19","author":"Khemani","year":"2019","journal-title":"Ochsner. J."},{"key":"ref_197","doi-asserted-by":"crossref","first-page":"1809","DOI":"10.1002\/cncr.29271","article-title":"Unrelated cord blood compared with haploidentical grafts in patients with hematological malignancies","volume":"121","author":"Crocchiolo","year":"2015","journal-title":"Cancer"},{"key":"ref_198","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1007\/s11899-014-0245-y","article-title":"Alternative donor transplantation\u2014\u201cMixing and matching\u201d: The role of combined cord blood and haplo-identical donor transplantation (haplo-cord SCT) as a treatment strategy for patients lacking standard donors?","volume":"10","author":"Liu","year":"2015","journal-title":"Curr. Hematol. Malig. Rep."},{"key":"ref_199","doi-asserted-by":"crossref","unstructured":"Bertaina, A., and Andreani, M. (2018). Major histocompatibility complex and hematopoietic stem cell transplantation: Beyond the classical HLA polymorphism. Int. J. Mol. Sci., 19.","DOI":"10.3390\/ijms19020621"},{"key":"ref_200","doi-asserted-by":"crossref","first-page":"191","DOI":"10.3389\/fimmu.2020.00191","article-title":"Key aspects of the immunobiology of haploidentical hematopoietic cell transplantation","volume":"11","author":"Baumeister","year":"2020","journal-title":"Front. Immunol."},{"key":"ref_201","doi-asserted-by":"crossref","unstructured":"Frati, G., and Miccio, A. (2021). Genome editing for \u03b2-hemoglobinopathies: Advances and challenges. J. Clin. Med., 10.","DOI":"10.3390\/jcm10030482"},{"key":"ref_202","doi-asserted-by":"crossref","first-page":"892","DOI":"10.1038\/s41587-020-0491-6","article-title":"Directed evolution of adenine base editors with increased activity and therapeutic application","volume":"38","author":"Gaudelli","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"ref_203","doi-asserted-by":"crossref","first-page":"977","DOI":"10.1038\/nbt.4199","article-title":"An APOBEC3A-Cas9 base editor with minimized bystander and off-target activities","volume":"36","author":"Gehrke","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"ref_204","doi-asserted-by":"crossref","first-page":"27","DOI":"10.4103\/2277-9175.98152","article-title":"Viral and nonviral delivery systems for gene delivery","volume":"1","author":"Nayerossadat","year":"2012","journal-title":"Adv. Biomed. Res."},{"key":"ref_205","doi-asserted-by":"crossref","unstructured":"Chen, Y., Wen, R., Yang, Z., and Chen, Z. (2021). Genome editing using CRISPR\/Cas9 to treat hereditary hematological disorders. Gene Ther.","DOI":"10.1038\/s41434-021-00247-9"},{"key":"ref_206","doi-asserted-by":"crossref","first-page":"1396","DOI":"10.1038\/s41591-019-0550-z","article-title":"Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia","volume":"25","author":"Navarro","year":"2019","journal-title":"Nat. Med."},{"key":"ref_207","first-page":"1225","article-title":"Glucose-6-phosphate dehydrogenase deficiency","volume":"136","author":"Luzzatto","year":"2020","journal-title":"Blood J. Am. Soc. Hematol."},{"key":"ref_208","doi-asserted-by":"crossref","first-page":"609","DOI":"10.1038\/gim.2016.173","article-title":"\u03b2-Thalassemia","volume":"19","author":"Origa","year":"2017","journal-title":"Genet. Med."}],"container-title":["International Journal of Molecular Sciences"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/1422-0067\/22\/12\/6275\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,11]],"date-time":"2025-10-11T06:13:09Z","timestamp":1760163189000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/1422-0067\/22\/12\/6275"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,6,10]]},"references-count":208,"journal-issue":{"issue":"12","published-online":{"date-parts":[[2021,6]]}},"alternative-id":["ijms22126275"],"URL":"https:\/\/doi.org\/10.3390\/ijms22126275","relation":{},"ISSN":["1422-0067"],"issn-type":[{"value":"1422-0067","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,6,10]]}}}