Abstract
<jats:p>This chapter provides a comprehensive overview of stem cell transplantation (SCT) and gene therapy as medical advances in the treatment of sickle cell disease (SCD). The discussion introduces hematopoietic stem cell transplant (HSCT) as the most effective and recommended treatment for SCD, offering high cure rates and survival rates because it can fully replace the patient’s hematopoietic system. While gene therapy has the potential to transform SCD management, additional long-term studies are necessary to confirm its safety and efficacy. The chapter describes, in general terms, the transplantation process and its clinical indications. Ethical and socioeconomic considerations, as well as implementation challenges and barriers to HSCT, are also addressed, with particular attention to donor availability, treatment costs, and accessibility in low-resource settings. The community needs to address barriers to accessing this treatment. In addition, approaches to SCT are explored, including myeloablative preparative and reduced-intensity conditioning regimens, haploidentical transplantation, and gene-based therapies such as gene addition and CRISPR/Cas9 gene editing. These innovative strategies are promising because they lower the risk of GVHD and do not require a donor. In conclusion, the chapter highlights posttransplant outcomes and integrates established evidence-based practices in nursing care for patients posttransplantation, including nursing interventions undertaken after a transplant operation. The chapter emphasizes how these interventions can help reduce the burden on caregivers and patients. Other factors discussed include improvements in organ function and health-related quality of life posttransplantation.</jats:p>