The Journey of Gene Therapy in Sickle Cell Disease: How Molecular Advances Meet Clinical Care

Highlights

What are the main findings?

• Gene therapy for sickle cell disease has progressed from lentiviral gene addition strategies to precise genome editing approaches targeting fetal hemoglobin regulation or the β-globin mutation.
• Clinical translation has demonstrated the feasibility of durable disease modification, but implementation remains constrained by conditioning toxicity, delivery challenges, and complex treatment pathways.

What are the implications of the main findings?

• Optimizing gene editing technologies and delivery platforms will be essential to improve safety, durability, and physiological control of therapeutic hemoglobin expression.
• Addressing patient selection, long-term outcomes, and global accessibility is critical for translating gene therapy advances into equitable clinical care.