HemoglobinOct 15, 2026

Personalized Treatments for Sickle Cell Disease: From Biological Processes to Potential Cures

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗OA ↗

Updated Oct 16, 2026

Abstract

Sickle cell disease (SCD) exhibits substantial variability in severity and treatment response, influencing long-term outcomes.

SCD is linked to hemoglobin S polymerization, hemolysis, and vaso-occlusion.
Key pathways involved in SCD include hemoglobin polymerization, vascular adhesion, and inflammation.
Targeted agents like hydroxyurea, voxelotor, crizanlizumab, and L-glutamine have been developed to address specific pathogenic mechanisms.
Pharmacogenomic factors, particularly involving fetal hemoglobin regulatory genes, contribute to variability in disease expression and treatment response.
Emerging therapies, including allogeneic stem cell transplantation and gene editing techniques, offer potential curative options.
The integration of genomic profiling and biomarker stratification is necessary for advancing individualized care in SCD.

Simplified

Sickle cell disease (SCD) is a monogenic β-globin disorder characterized by hemoglobin S polymerization, hemolysis, and vaso-occlusion, yet it exhibits remarkable clinical heterogeneity. Despite advances in supportive care and disease-modifying therapies, substantial variability in severity, treatment response, and long-term outcomes persists. Therefore, this review aims to examine the biological and clinical foundations of precision medicine in SCD, emphasizing genomic modifiers, molecular pathways, and phenotype-driven therapeutic strategies. Key pathogenic pathways including hemoglobin polymerization, vascular adhesion, and inflammatory and oxidative amplification have enabled the development of targeted agents such as hydroxyurea, voxelotor, crizanlizumab, and L-glutamine, each demonstrating domain-specific targets. Pharmacogenomic insights, particularly involving fetal hemoglobin regulatory loci (e.g. BCL11A and HBS1L-MYB), further elucidate variability in disease expression and therapeutic response. Emerging curative approaches, including allogeneic hematopoietic stem cell transplantation, lentiviral gene addition, and CRISPR-Cas9-mediated genome editing, represent transformative applications of precision hematology. Collectively, these advances support a paradigm shift from generalized symptom management toward biologically informed, individualized care. Continued integration of genomic profiling, biomarker stratification, and equitable implementation frameworks will be essential to translate precision strategies into sustainable improvements in global SCD outcomes.

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Personalized Treatments for Sickle Cell Disease: From Biological Processes to Potential Cures

Abstract

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