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REVIEW ARTICLE

Sickle cell disease: A 75-year journey

Samira Bolo1 ,  Tulika Mishra2 ,  Uzoamaka Eziri1 ,  Tiara Calvo Leon3 ,  Deepti Mankar2 ,  Frank Navarrete4 ,  Abrar Khan2 ,  Malpe Surekha Bhat5*
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1 American University School of Medicine Aruba, Oranjestad, Aruba
2 Department of Pathological Processes and Therapeutics, American University School of Medicine Aruba, Oranjestad, Aruba
3 Department of Functional and Diagnostic Sciences, American University School of Medicine Aruba, Oranjestad, Aruba
4 Department of Mind, Behavior and Global Health, American University School of Medicine Aruba, Oranjestad, Aruba
5 Department of Nutritional, Biochemical and Molecular Sciences, American University School of Medicine Aruba, Oranjestad, Aruba
GPD, 4361 https://doi.org/10.36922/gpd.4361
Submitted: 29 July 2024 | Revised: 22 January 2025 | Accepted: 24 January 2025 | Published: 27 February 2025
© 2025 by the Author(s). This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution 4.0 International License ( https://creativecommons.org/licenses/by/4.0/ )
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Abstract

Sickle cell disease (SCD) is a disorder characterized by the polymerization of hemoglobin chains in the deoxy-form, sickling of red blood cells, and hence vaso-occlusive crisis, multiple organ damage, and increased mortality due to an inherited defect in hemoglobin structure. SCD can also lead to a host of complications, which include acute chest syndrome, avascular necrosis, stroke, pulmonary hypertension, splenic sequestration, gallstones, deep vein thrombosis, pregnancy complications, and end-organ damage. Complications are of varying complexities and can be as grave as life-threatening. According to a report in 2005, the median life expectancy for male and female patients with SCD in the United States (US) was around 42 and 38 years, respectively. However, the survival rate of SCD patients in high-income countries has steadily improved. Treatment options that were mainly for symptomatic relief and led to better well-being of the patient, containment of complication recurrence, and decrease in mortality rates have evolved into curative treatment options such as stem cell transplantations and gene therapy. The present paper is a review of the disease, its complications and implications on the community, and a historical tracking of the evolution of treatment options up to modern-day gene therapy.

Graphical abstract
Keywords
Sickle cell disease
Treatments
Myeloablative transplantation
Gene therapy
Casgevy
Lentiviral
Funding
None.
Conflict of interest
The authors declare they have no competing interests.
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Gene & Protein in Disease, Electronic ISSN: 2811-003X Published by AccScience Publishing
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