Advancements and Challenges in Gene Therapy Approaches for Sickle Cell Disease: A Comprehensive Review

Abstract

Sickle cell disease (SCD) is an autosomal recessive genetic blood disorder that occurs when both alleles of the HBB gene have mutations, leading to the production of abnormal haemoglobin (HbS). The presence of HbS causes red blood cells (RBCs) to take on the distinctive sickle-shaped form associated with the disease. This, in turn, leads to blockages in blood vessels, decreased blood circulation, and organs’ damage. Traditional treatments such as blood transfusions and hydroxyurea offer relief but come with their own limitations and associated risks. Gene therapy has emerged as a promising paradigm shift in the quest to cure SCD, offering personalised solutions by targeting the genetic root of the disease. This review article explores the principles and recent advancements in gene therapy for SCD. However, before gene therapy can become the main curative strategy for this disease, several challenges need to be overcome including the need for long-term safety and efficacy evaluations. Ongoing research and innovation hold the promise of enhanced treatments and the potential for a widely available gene therapy, ultimately improving the quality of life for individuals living with SCD.