Mutations in Medicine: Advances in CRISPR and Sickle Cell Disease
Harty, Alana
Harty, Alana
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Abstract
Sickle Cell Disease is an inherited blood disorder caused by a mutation in the ?-globin gene that produces hemoglobin. This leads to the production of abnormal sickle and rigid red blood cells which impairs oxygen delivery causing chronic pain, anemia, and organ damage. Current treatments, (i.e. including blood transfusions and bone marrow transplantation) are limited by availability, risks, and accessibility. CRISPR-Cas9 gene editing offers promise as a potential therapeutic strategy. CRISPR can be used to correct the mutated gene or reactivate fetal hemoglobin (HbF) production, which reduces sickling of red blood cells. Early clinical trials have demonstrated significant improvements in patient outcomes such as reducing pain episodes and decreased dependence on transfusions. Despite its potential, there is concern with long-term safety, off-target effects, and equitable access to treatment. Nevertheless, CRISPR-based therapies represent a transformative approach that may lead to a functional cure for Sickle Cell Disease.
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Date
1/1/2026
Student Status
Senior (Graduating in 2026)
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Poster
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Program/Major
Nutrition and Food Science
College/School
College of Agriculture and Life Sciences
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Clinical Science