Рет қаралды 449
Join the report on results from the phase 1/2 clinical trial (NCT03897361) evaluating safety and efficacy of CTNS-RD-04 in adult patients with cystinosis. CTNS-RD-04 consists of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) transduced with a lentiviral vector (LV) carrying the CTNS cDNA encoding for cystinosin. The trial is fully enrolled, and six participants (ages 20 to 46 years) have been treated with CTNS-RD-04 with follow-up ranging from 1 to 36 months. Jordan, patient 1 of the clinical trial, will share his experience.