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.
Comprehensive clinical guidelines now help specialized centers safely implement gene therapies that offer sickle cell patients a potential path toward disease remission.
This joint practice guideline from ASTCT and ISCT> establishes the first comprehensive clinical recommendations for implementing FDA-approved gene therapies for sickle cell disease, covering patient selection, pre-treatment evaluation, and long-term monitoring. The guidelines address a critical implementation gap as gene therapies become available in specialized centers.
What the study was
- Study design
- Expert consensus / practice guideline
- Population
- Patients with sickle cell disease eligible for gene therapy
- Category
- Treatment Innovation
- Maturity
- Potentially Practice-Changing
- Journal
- Transplantation and Cellular Therapy
Why it surfaced
First joint societal practice guidelines for FDA-approved SCD gene therapies; directly actionable for centers implementing these therapies; rare disease with very high unmet need and life-altering treatment impact.
A plain-language summary of published research — not medical advice. Talk to a clinician about your own care.