End Organ Affection in Sickle Cell Disease
Name
cells-13-00934.pdf
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928.67 KB
Format
Adobe PDF
Checksum (MD5)
cf07c6916505523e5d7269c8887e53d1
Author(s)
Bathla, Tanvi
Lotfollahzadeh, Saran
Quisel, Matthew
Mehta, Mansi
Malikova, Marina
Chitalia, Vipul C.
Date Issued
May 29, 2024
Journal
Cells
Publisher
MDPI AG
Citation
Bathla, T.; Lotfollahzadeh, S.; Quisel, M.; Mehta, M.; Malikova, M.; Chitalia, V.C. End Organ Affection in Sickle Cell Disease. Cells 2024, 13, 934.
Version
Final published version
Abstract
Sickle cell disease is an orphan disease affecting ethnic minorities and characterized by profound systemic manifestations. Although around 100,000 individuals with SCD are living in the US, the exact number of individuals is unknown, and it is considered an orphan disease. This single-gene disorder leads to red blood cell sickling and the deoxygenation of hemoglobin, resulting in hemolysis. SCD is associated with acute complications such as vaso-occlusive crisis, infections, and chronic target organ complications such as pulmonary disease and renal failure. While genetic therapy holds promise to alter the fundamental disease process, the major challenge in the field remains the target end organ damage and ways to mitigate or reverse it. Here, we provide an overview of the clinical manifestations and pathogenesis with a focus on end-organ damage and current therapeutic options, including recent FDA-approved stem cell and gene editing therapies.
MIT Department
Massachusetts Institute of Technology. Institute for Medical Engineering & Science
Terms of Use
Creative Commons Attribution
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DOI of Published Version
https://doi.org/10.3390/cells13110934
