Article
Genome editing strategies for targeted correction of β-globin mutation in sickle cell disease: From bench to bedside.
Molecular therapy : the journal of the American Society of Gene Therapy - 7 May 2025
Butt Henna, Sathish Shruti, London Evan, Lee Johnson Taylor, Essawi Khaled, Leonard Alexis, Tisdale John F, Demirci Selami
Abstract excerpt
Sickle cell disease (SCD) includes a range of genotypes that result in a clinical syndrome, where abnormal red blood cell (RBC) physiology leads to widespread complications affecting nearly every organ system. Treatment strategies for SCD can be broadly categorized into disease-modifying therapies and those aimed toward a cure. Although several disease-modifying drugs have been approved, they do not fully address...
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