Population
Patients with sickle cell disease (SCD).
Intervention
Systematic review to determine whether gene therapy can improve survival and prevent symptoms and complications associated with SCD, and to examine the risks of gene therapy against the potential longāterm gain.
Comparison
Outcome
Twenty-six studies were identified but none were eligible for inclusion. Searches were conducted in the trial registries: World Health Organization International Clinical Trials Registry Platform, ClinicalTrials.gov, and the Cochrane Cystic Fibrosis and Genetic Disorders Group Haemoglobinopathies Trials Register (compiled from electronic searches of CENTRAL and MEDLINE). No trials of gene therapy for SCD were found after searching relevant published and unpublished trials without restrictions on language, year or publication status.