Emerging Genetic Therapy for Sickle Cell Disease

The new frontier of genome engineering with CRISPR-Cas9

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Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

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Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

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Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia

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Sickle Cell Anemia, a Molecular Disease

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An efficient targeted nuclease strategy for high-resolution mapping of DNA binding sites

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Genomewide Association Studies and Assessment of the Risk of Disease

Manolio, T. A. (2010) · New England Journal of Medicine · doi:10.1056/nejmra0905980 · Cited by 1,512

Gene therapy comes of age

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Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

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Global Burden of Sickle Cell Anaemia in Children under Five, 2010–2050: Modelling Based on Demographics, Excess Mortality, and Interventions

Piel, F. B., Hay, S. I., Gupta, S., Weatherall, D. J., & Williams, T. N. (2013) · PLoS Medicine · doi:10.1371/journal.pmed.1001484 · Cited by 1,144

Chromothripsis as an on-target consequence of CRISPR–Cas9 genome editing

Leibowitz, M. L., Papathanasiou, S., Doerfler, P. A., Blaine, L. J., Sun, L., Yao, Y., Zhang, C.-Z., Weiss, M. J., & Pellman, D. (2021) · Nature Genetics · doi:10.1038/s41588-021-00838-7 · Cited by 583

Recent Advances in the Treatment of Sickle Cell Disease

Cisneros, G. S., & Thein, S. L. (2020) · Frontiers in Physiology · doi:10.3389/fphys.2020.00435 · Cited by 210

Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease

Lattanzi, A., Camarena, J., Lahiri, P., Segal, H., Srifa, W., Vakulskas, C. A., Frock, R. L., Kenrick, J., Lee, C. M., Talbott, N., et al. (2021) · Science Translational Medicine · doi:10.1126/scitranslmed.abf2444 · Cited by 171

Fetal hemoglobin in sickle cell anemia

Steinberg, M. H. (2020) · Blood · doi:10.1182/blood.2020007645 · Cited by 136

Hematopoietic stem cells through the ages: A lifetime of adaptation to organismal demands

Kasbekar, M., Mitchell, C. A., Proven, M. A., & Passegue, E. A. (2023) · Cell stem cell · doi:10.1016/j.stem.2023.09.013 · Cited by 130

Genetic engineering of hematopoiesis: current stage of clinical translation and future perspectives

Naldini, L. (2019) · EMBO Molecular Medicine · doi:10.15252/emmm.201809958 · Cited by 115

In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice

Li, C., Georgakopoulou, A., Mishra, A., Gil, S., Hawkins, R. D., Yannaki, E., & Lieber, A. (2021) · Blood Advances · doi:10.1182/bloodadvances.2020003702 · Cited by 96

Emerging disease-modifying therapies for sickle cell disease

Carden, M. A., & Little, J. A. (2019) · Haematologica · doi:10.3324/haematol.2018.207357 · Cited by 100

Sickle Cell Disease, a Review

Tebbi, C. K. (2022) · Hemato · doi:10.3390/hemato3020024 · Cited by 110

Artificial Intelligence, Healthcare, Clinical Genomics, and Pharmacogenomics Approaches in Precision Medicine

Abdelhalim, H., Berber, A., Lodi, M. K., Jain, R., Nair, A. S., Pappu, A., Patel, K., Venkat, V., Venkatesan, C., Wable, R., et al. (2022) · Frontiers in Genetics · doi:10.3389/fgene.2022.929736 · Cited by 99

ZNF410 represses fetal globin by singular control of CHD4

Vinjamur, D. S., Yao, Q., Cole, M. A., McGuckin, C., Ren, C., Zeng, J., Hossain, M. A., Luk, K., Wolfe, S. A., Pinello, L., et al. (2021) · Nature Genetics · doi:10.1038/s41588-021-00843-w · Cited by 83

Treating sickle cell anemia

Tisdale, J. F., Thein, S. L., & Eaton, W. A. (2020) · Science · doi:10.1126/science.aba3827 · Cited by 85

Fetal Hemoglobin in Sickle Hemoglobinopathies: High HbF Genotypes and Phenotypes

Steinberg, M. H. (2020) · Journal of Clinical Medicine · doi:10.3390/jcm9113782 · Cited by 78

Microfluidic assessment of red blood cell mediated microvascular occlusion

Man, Y., Kucukal, E., An, R., Watson, Q. D., Bosch, J., Zimmerman, P. A., Little, J. A., & Gurkan, U. A. (2020) · Lab on a Chip · doi:10.1039/d0lc00112k · Cited by 73

Emerging role of ZBTB7A as an oncogenic driver and transcriptional repressor

Gupta, S., Singh, A. K., Prajapati, K. S., Kushwaha, P. P., Shuaib, M., & Kumar, S. (2020) · Cancer Letters · doi:10.1016/j.canlet.2020.04.015 · Cited by 61

Adenovirus vectors in hematopoietic stem cell genome editing

Li, C., & Lieber, A. (2019) · FEBS Letters · doi:10.1002/1873-3468.13668 · Cited by 53

Hemoglobin S polymerization and sickle cell disease: A retrospective on the occasion of the 70th anniversary of Pauling's Science paper

Eaton, W. A. (2019) · American Journal of Hematology · doi:10.1002/ajh.25687 · Cited by 57

Reactivation of a developmentally silenced embryonic globin gene

King, A. J., Songdej, D., Downes, D. J., Beagrie, R. A., Liu, S., Buckley, M., Peng, H., Suciu, M., Oudelaar, A. M., Hanssen, L. L. P., et al. (2021) · Nature Communications · doi:10.1038/s41467-021-24402-3 · Cited by 57

A specific G9a inhibitor unveils BGLT3 lncRNA as a universal mediator of chemically induced fetal globin gene expression

Takase, S., Hiroyama, T., Shirai, F., Maemoto, Y., Nakata, A., Arata, M., Matsuoka, S., Sonoda, T., Niwa, H., Sato, S., et al. (2023) · Nature Communications · doi:10.1038/s41467-022-35404-0 · Cited by 50

Structural insights into the recognition of γ-globin gene promoter by BCL11A

Yang, Y., Xu, Z. Z., He, C. J., Zhang, B., Shi, Y., & Li, F. (2019) · Cell Research · doi:10.1038/s41422-019-0221-0 · Cited by 40

Gene Therapy of the Hemoglobinopathies

Kunz, J. B., & Kulozik, A. E. (2020) · HemaSphere · doi:10.1097/hs9.0000000000000479 · Cited by 40

Structural basis for human ZBTB7A action at the fetal globin promoter

Yang, Y., Ren, R., Ly, L. C., Horton, J. R., Li, F., Quinlan, K. G. R., Crossley, M., Shi, Y., & Cheng, X. (2021) · Cell Reports · doi:10.1016/j.celrep.2021.109759 · Cited by 37

Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells

Zeng, J., Nguyen, M. A., Liu, P., da Silva, L. F., Levesque, S., Lin, L. Y., Justus, D. G., Petri, K., Clement, K., Porter, S. N., et al. (2024) · Cell stem cell · doi:10.1016/j.stem.2024.11.001 · Cited by 36

Affordable Pricing of CRISPR Treatments is a Pressing Ethical Imperative

Rueda, J., Beriaín, Í. D. M., & Montoliu, L. (2024) · The CRISPR Journal · doi:10.1089/crispr.2024.0042 · Cited by 32

Molecular Medicine: Found in Translation

Orkin, S. H. (2021) · Med · doi:10.1016/j.medj.2020.12.011 · Cited by 36

Genetic therapies for the first molecular disease

Doerfler, P. A., Sharma, A., Porter, J. S., Zheng, Y., Tisdale, J. F., & Weiss, M. J. (2021) · Journal of Clinical Investigation · doi:10.1172/jci146394 · Cited by 30

Genome editing strategies for fetal hemoglobin induction in beta-hemoglobinopathies

Demirci, S., Leonard, A. K., & Tisdale, J. F. (2020) · Human Molecular Genetics · doi:10.1093/hmg/ddaa088 · Cited by 32

Health Supervision for Children and Adolescents With Sickle Cell Disease: Clinical Report

Yates, A. M., Aygün, B., Nuss, R., Rogers, Z. R., Hematology/Oncology, S. O., Wetmore, C., Dickens, D., Majumdar, S., Stoffman, J. M., Warwick, A. B., et al. (2024) · PEDIATRICS · doi:10.1542/peds.2024-066842 · Cited by 30

Embryonic erythropoiesis and hemoglobin switching require transcriptional repressor ETO2 to modulate chromatin organization

Guo, X., Plank-Bazinet, J. L., Krivega, I. V., Dale, R., & Dean, A. (2020) · Nucleic Acids Research · doi:10.1093/nar/gkaa736 · Cited by 30

Evolution of nanobodies specific for BCL11A

Yin, M., Izadi, M., Tenglin, K., Viennet, T., Zhai, L., Zheng, G., Arthanari, H., Dassama, L. M. K., & Orkin, S. H. (2023) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2218959120 · Cited by 28

Microfluidics in Sickle Cell Disease Research: State of the Art and a Perspective Beyond the Flow Problem

Aich, A., Lamarre, Y., Sacomani, D. P., Kashima, S. H., Covas, D. T., & de la Torre, L. G. (2021) · Frontiers in Molecular Biosciences · doi:10.3389/fmolb.2020.558982 · Cited by 27

Welcoming the Era of Gene Editing in Medicine

Daley, G. Q. (2024) · New England Journal of Medicine · doi:10.1056/nejmp2314279 · Cited by 25

Research in Sickle Cell Disease: From Bedside to Bench to Bedside

Cisneros, G. S., & Thein, S. L. (2021) · HemaSphere · doi:10.1097/hs9.0000000000000584 · Cited by 25

Gene therapy strategies for rare monogenic disorders with nuclear or mitochondrial gene mutations

Wang, Y., Hu, L., Zhou, T., Qi, L., Xing, L., Lee, J., Wang, F., Oh, Y., & Jiang, H. (2021) · Biomaterials · doi:10.1016/j.biomaterials.2021.121108 · Cited by 25

Manifestations of HbSE sickle cell disease: a systematic review

Khamees, I., Ata, F., Choudry, H. A., Soliman, A. T., Sanctis, V. D., & Yassin, M. A. (2021) · Journal of Translational Medicine · doi:10.1186/s12967-021-02931-1 · Cited by 24

Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations

Woodard, K. J., Doerfler, P. A., Mayberry, K. D., Sharma, A., Levine, R. M., Yen, J. S., Valentine, V. A., Palmer, L. E., Valentine, M. B., & Weiss, M. J. (2022) · Disease Models & Mechanisms · doi:10.1242/dmm.049463 · Cited by 25

Abdominal Manifestations of Sickle Cell Disease

Kinger, N. P., Moreno, C. C., Miller, F. H., & Mittal, P. (2020) · Current Problems in Diagnostic Radiology · doi:10.1067/j.cpradiol.2020.05.012 · Cited by 21

The Meaning of Informed Consent: Genome Editing Clinical Trials for Sickle Cell Disease

Desine, S., Hollister, B. M., Abdallah, K. E., Persaud, A., Hull, S. C., & Bonham, V. L. (2020) · AJOB Empirical Bioethics · doi:10.1080/23294515.2020.1818876 · Cited by 23

Impact of hemoglobin biophysical studies on molecular pathogenesis and drug therapy for sickle cell disease

Eaton, W. A. (2021) · Molecular Aspects of Medicine · doi:10.1016/j.mam.2021.100971 · Cited by 22

Society for Maternal-Fetal Medicine Special Statement: Beyond the scalpel: in utero fetal gene therapy and curative medicine

Shanahan, M. A., Aagaard, K. M., McCullough, L. B., Chervenak, F. A., & Shamshirsaz, A. A. (2021) · American Journal of Obstetrics and Gynecology · doi:10.1016/j.ajog.2021.09.001 · Cited by 22

CRISPR/Cas9-based multiplex genome editing of BCL11A and HBG efficiently induces fetal hemoglobin expression

Han, Y., Tan, X., Jin, T., Zhao, S., Li, H., Zhang, W., Kurita, R., Nakamura, Y., Liu, J., Li, D., et al. (2022) · European Journal of Pharmacology · doi:10.1016/j.ejphar.2022.174788 · Cited by 21

Hematopoietic stem cell collection for sickle cell disease gene therapy

Leonard, A. K., & Weiss, M. J. (2024) · Current Opinion in Hematology · doi:10.1097/moh.0000000000000807 · Cited by 18

Non-invasive urinary biomarkers of renal function in sickle cell disease: an overview

Laurentino, M. R., Filho, S. L. A. P., Parente, L. L. C., da Silva, G. B., Daher, E. D. F., & Lemes, R. P. G. (2019) · Annals of Hematology · doi:10.1007/s00277-019-03813-9 · Cited by 18

Potential causal role of l-glutamine in sickle cell disease painful crises: A Mendelian randomization analysis

Ilboudo, Y., Garrett, M. E., Bartolucci, P., Brugnara, C., Clish, C. B., Hirschhorn, J. N., Galactéros, F., Ashley‐Koch, A. E., Telen, M. J., & Lettre, G. (2020) · Blood Cells Molecules and Diseases · doi:10.1016/j.bcmd.2020.102504 · Cited by 18

Delta‐globin gene expression improves sickle cell disease in a humanised mouse model

Porcu, S., Simbula, M., Marongiu, M. F., Perra, A., Poddie, D., Perseu, L., Kowalik, M. A., Littera, R., Barella, S., Caria, C. A., et al. (2021) · British Journal of Haematology · doi:10.1111/bjh.17561 · Cited by 19

Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease

Xu, L., Lahiri, P., Skowronski, J., Bhatia, N., Lattanzi, A., & Porteus, M. H. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.07.009 · Cited by 16

Combined genetic disruption of K-Cl cotransporters and Gardos channel KCNN4 rescues erythrocyte dehydration in the SAD mouse model of sickle cell disease

Shmukler, B. E., Rivera, A., Bhargava, P., Nishimura, K. K., Hsu, A., Kim, E. H., Trudel, M., Rust, M. B., Hübner, C. A., Brugnara, C., et al. (2019) · Blood Cells Molecules and Diseases · doi:10.1016/j.bcmd.2019.102346 · Cited by 16

Challenges in the Management of Sickle Cell Disease During SARS-CoV-2 Pandemic

Alsayegh, F., & Mousa, S. A. (2020) · Clinical and Applied Thrombosis/Hemostasis · doi:10.1177/1076029620955240 · Cited by 18

Hidden behind thromboinflammation: revealing the roles of von Willebrand factor in sickle cell disease pathophysiology

Vital, E. F., & Lam, W. A. (2023) · Current Opinion in Hematology · doi:10.1097/moh.0000000000000755 · Cited by 15

Discovery of potent DNMT1 inhibitors against sickle cell disease using structural-based virtual screening, MM-GBSA and molecular dynamics simulation-based approaches

Chandu, A., Joshi, R. P., Gupta, P., Ramalingam, S., & Murugesan, S. (2023) · Journal of Biomolecular Structure and Dynamics · doi:10.1080/07391102.2023.2199081 · Cited by 13

Modeling islet enhancers using deep learning identifies candidate causal variants at loci associated with T2D and glycemic traits

Hudaiberdiev, S., Taylor, D. L., Song, W., Narisu, N., Bhuiyan, R. M., Taylor, H. J., Tang, X., Yan, T., Swift, A. J., Bonnycastle, L. L., et al. (2023) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2206612120 · Cited by 13

Epidemiological, clinical, and severity characterization of sickle cell disease in a population from the Brazilian Amazon

Cesar, P., Dhyani, A., Schwade, L. A., Acordi, P., de Albuquerque, C. X., Martins, R. N., Paula, E. V. D., & Fraiji, N. A. (2019) · Hematology/Oncology and Stem Cell Therapy · doi:10.1016/j.hemonc.2019.04.002 · Cited by 14

Optimization of Protein Thermostability and Exploitation of Recognition Behavior to Engineer Altered Protein-DNA Recognition

Lambert, A. R., Hallinan, J. P., Werther, R. A., Głów, D., & Stoddard, B. L. (2020) · Structure · doi:10.1016/j.str.2020.04.009 · Cited by 12

Characterization and statistical modeling of glycosylation changes in sickle cell disease

Ashwood, H. E., Ashwood, C., Schmidt, A. P., Gundry, R. L., Hoffmeister, K. M., & Anani, W. Q. (2021) · Blood Advances · doi:10.1182/bloodadvances.2020003376 · Cited by 11

Gene Therapy as the New Frontier for Sickle Cell Disease

Garg, H., Tatiossian, K. J., Peppel, K., Kato, G. J., & Herzog, E. (2021) · Current Medicinal Chemistry · doi:10.2174/0929867328666210527092456 · Cited by 12

The Cas9 Hammer—and Sickle: A Challenge for Genome Editors

Urnov, F. D. (2021) · The CRISPR Journal · doi:10.1089/crispr.2021.29120.fur · Cited by 11

Beyond DNA-targeting in Cancer Chemotherapy. Emerging Frontiers - A Review

Mbugua, S. N., Njenga, L. W., Odhiambo, R. A., Wandiga, S. O., & Onani, M. O. (2020) · Current Topics in Medicinal Chemistry · doi:10.2174/1568026620666200819160213 · Cited by 11

CRISPR–Cas9 can cause chromothripsis

Urnov, F. D. (2021) · Nature Genetics · doi:10.1038/s41588-021-00881-4 · Cited by 9

Considerations for Selecting Cognitive Endpoints and Psychological Patient-Reported Outcomes for Clinical Trials in Pediatric Patients With Sickle Cell Disease

Hood, A., Crosby, L. E., Stotesbury, H., Kölbel, M., & Kirkham, F. J. (2022) · Frontiers in Neurology · doi:10.3389/fneur.2022.835823 · Cited by 11

Multimeric transcription factor BCL11A utilizes two zinc-finger tandem arrays to bind clustered short sequence motifs

Horton, J. R., Yu, M., Zhou, J., Tran, M. L., Anakal, R. R., Lu, Y., Blumenthal, R., Zhang, X., Huang, Y., Zhang, X., et al. (2025) · Nature Communications · doi:10.1038/s41467-025-58998-7 · Cited by 11

Sickle cell disease in the era of precision medicine: looking to the future

Steinberg, M. H., Kumar, S. S., Murphy, G. J., & Vanuytsel, K. (2019) · Expert Review of Precision Medicine and Drug Development · doi:10.1080/23808993.2019.1688658 · Cited by 9

From Basic Biology to Patient Mutational Spectra of GATA2 Haploinsufficiencies: What Are the Mechanisms, Hurdles, and Prospects of Genome Editing for Treatment

Koyunlar, C., & de Pater, E. (2020) · Frontiers in Genome Editing · doi:10.3389/fgeed.2020.602182 · Cited by 10

Accelerating the Science of SCD Therapies—Is a Cure Possible?

Benz, E. J. J., Mondoro, T. H., & Gibbons, G. H. (2019) · JAMA · doi:10.1001/jama.2019.11419 · Cited by 9

Challenges and limitations of mobilization and stem cell collection for gene therapy of sickle cell disease

Tanhehco, Y. C., Thibodeaux, S. R., Shi, P. A., Chou, S. T., Kelly, S., Monis, G. F., Newton, C., West, K. A., Zheng, Y., Fernandez, C. M., et al. (2025) · Blood Advances · doi:10.1182/bloodadvances.2025016054 · Cited by 8

New and emerging treatments for vaso-occlusive pain in sickle cell disease

Dampier, C. (2019) · Expert Review of Hematology · doi:10.1080/17474086.2019.1649131 · Cited by 7

Assessment of Hemoglobin Variants in Patients Receiving Health Care at the Ho Teaching Hospital: A Three-Year Retrospective Study

Awaitey, D. K., Akorsu, E. E., Allotey, E. A., Kwasie, D. A., Kwadzokpui, P. K., Tawiah, P. A., Amankwah, S. A., & Abaka‐Yawson, A. (2020) · Advances in Hematology · doi:10.1155/2020/7369731 · Cited by 7

CRISPR-Cas9 Genomic Editing as an Innovation in the Management of Sickle Cell Disease: A Systematic Review

Ikwuka, A. O., Musa, S., Udeh, F. C., Musa, A. A., & Chukwuezie, U. C. (2023) · American Journal of Medical Science and Innovation · doi:10.54536/ajmsi.v2i2.1760 · Cited by 7

A critical review of therapeutic interventions in sickle cell disease: Progress and challenges

Chandu, A., Joshi, R. P., Gupta, P., Goswami, S. G., Ramalingam, S., Gowri, C. S. K. V., & Murugesan, S. (2024) · Archiv der Pharmazie · doi:10.1002/ardp.202400381 · Cited by 6

Allogeneic Transplant and Gene Therapy

Lawal, R. A., Walters, M. C., & Fitzhugh, C. D. (2022) · Hematology/Oncology Clinics of North America · doi:10.1016/j.hoc.2022.06.007 · Cited by 5

Tackling sickle cell crisis in Nigeria: the need for newer therapeutic solutions in sickle cell crisis management – short communication

Nnagha, E. M., MATTHEW, K. A., Izevbizua, E. A., Uwishema, O., Nazir, A., & Wellington, J. (2023) · Annals of Medicine and Surgery · doi:10.1097/ms9.0000000000000696 · Cited by 6

Engineering Cell-Specific Protein Delivery Vehicles for Erythroid Lineage Cells

Setegne, M. T., Cabral, A. T., Tiwari, A., Shen, F., Thiam, H. R., & Dassama, L. M. K. (2025) · ACS Bio & Med Chem Au · doi:10.1021/acsbiomedchemau.4c00098 · Cited by 5

Executive Function and Processing Speed in Children Living with Sickle Cell Anemia

Kelleher, S. C., Kirkham, F. J., & Hood, A. (2023) · Children · doi:10.3390/children10101585 · Cited by 5

CD14+ monocytes repress gamma globin expression at early stages of erythropoiesis

Heshusius, S., Heideveld, E., Lindern, M. V., & van den Akker, E. (2021) · Scientific Reports · doi:10.1038/s41598-021-81060-7 · Cited by 5

Protocol for “Genetic composition of sickle cell disease in the Arab population: A systematic review”

Ata, F., Yousaf, Z., Sardar, S., Javed, S., Iqbal, P., Khamees, I., Malkawi, L., & Yassin, M. A. (2022) · Health Science Reports · doi:10.1002/hsr2.450 · Cited by 4

Editorial: New Methods for Red Blood Cell Research and Diagnosis

Bianchi, P. I., & van Wijk, R. (2021) · Frontiers in Physiology · doi:10.3389/fphys.2021.755664 · Cited by 4

Extending Gene Medicines to All in Need

McCune, J. M., & Kiem, H. (2024) · New England Journal of Medicine · doi:10.1056/nejme2403104 · Cited by 4

Targeting ZNF410 as a potential β-hemoglobinopathy therapy

Tumburu, L., & Thein, S. L. (2021) · Nature Genetics · doi:10.1038/s41588-021-00817-y · Cited by 3

BET inhibitors enhance embryonic and fetal globin expression in erythroleukemia cell lines

Cao, J. Z., Bigelow, K. M., Wickrema, A., & Godley, L. A. (2021) · Haematologica · doi:10.3324/haematol.2021.278791 · Cited by 3

Cyclin A1 (CCNA1) inhibits osteoporosis by suppressing transforming growth factor-beta (TGF-beta) pathway in osteoblasts

Du, X., Zang, C., & Wang, Q. (2024) · BMC Musculoskeletal Disorders · doi:10.1186/s12891-024-07303-6 · Cited by 3

Comparison of transcriptome profiles of nucleated red blood cells in cord blood between preterm and full-term neonates

Han, Y., Huang, L., Zhou, M., Tan, X., Gong, S., Zhang, Z., Jin, T., Fang, X., Jia, Y., & Huang, S. (2022) · Hematology · doi:10.1080/16078454.2022.2029255 · Cited by 3

Fetal hemoglobin and F-cell variance in mobilized CD34+ cell-transplanted rhesus monkeys

Demirci, S., Haro‐Mora, J. J., Yapundich, M., Drysdale, C. M., Gamer, J., Nassehi, T., Bonifacino, A. C., Krouse, A. E., Linde, N. S., Donahue, R. E., et al. (2019) · Experimental Hematology · doi:10.1016/j.exphem.2019.05.005 · Cited by 3

The BCL11A transcription factor stimulates the enzymatic activities of the OGG1 DNA glycosylase

Petrachkova, T., Солдаткина, О. И., Leduy, L., & Nepveu, A. (2024) · Biological Chemistry · doi:10.1515/hsz-2024-0088 · Cited by 2

Efficacy and Safety of Allogeneic Hematopoietic Stem Cell Transplantation in Curing Sickle Cell Disease: A Systematic Review and Meta-Analysis of Single-Arm Studies

Folarin, M., Al-Zubaidi, H., Moore, E., Eze, U. A., & Palanisamy, N. (2025) · Transplantation and Cellular Therapy · doi:10.1016/j.jtct.2025.09.023 · Cited by 2

Emerging Therapies for Sickle Cell Disease: From Symptom Management to Curative Gene Therapy

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Hematopoietic stem cell transplantation for patients with sickle cell disease in the Eastern Mediterranean

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