Gene therapy for sickle cell disease: Practice recommendations from the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy - PubMed
7 hours ago
- #clinical recommendations
- #gene therapy
- #sickle cell disease
- Gene therapy is now a transformative treatment for sickle cell disease, with recent regulatory approvals.
- The American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy provide consensus recommendations.
- Key areas covered include patient selection, stem cell mobilization, manufacturing, conditioning, and long-term follow-up.
- Recommendations aim to standardize clinical practice, support decision-making, and ensure safe, equitable delivery of gene therapy.