Advances in Gene Therapy and CRISPR-Based Interventions for Beta-Thalassemia and Sickle Cell Disease: Evaluating the Clinical Efficacy and Safety of Emerging Therapeutic Approaches in Hemoglobinopathies

Abstract

Introduction: Hemoglobinopathies such as Beta-Thalassemia and Sickle Cell Disease (SCD) are inherited blood disorders caused by mutations in the hemoglobin gene, leading to anemia, pain crises, and organ damage. Traditional treatments like blood transfusions and bone marrow transplants have limitations, including iron overload, inadequate donor matching, and rejection risks. In recent years, gene therapy, particularly using CRISPR-based gene editing, has emerged as a promising curative solution. Objective: To evaluate the clinical outcomes of CRISPR-based gene therapies in patients with Beta-Thalassemia and Sickle Cell Disease, focusing on hemoglobin levels, transfusion dependency, and the occurrence of disease-related complications. Methodology: 85 patients with either Beta-Thalassemia or Sickle Cell Disease were treated with CRISPR-based gene editing or lentiviral gene transfer. Clinical outcomes were monitored over a 6-month period, including hemoglobin levels, transfusion requirements, and the incidence of pain crises (in SCD patients). Safety was assessed by tracking adverse events. Results: The therapy resulted in significant improvements in hemoglobin levels, reduced transfusion dependence, and decreased pain crises in SCD patients. The safety profile was favorable, with minor adverse effects reported. Conclusion: CRISPR-based gene therapies offer a promising and safe approach for the treatment of Beta-Thalassemia and Sickle Cell Disease, providing a potential long-term solution to hemoglobinopathies.