Therefore, a Phase 1/2 study is currently being conducted at the University of California San Diego (UC San Diego) to evaluate the safety and efficacy of a single transplantation of autologous CD34+ enriched cell fractions transduced with a lentiviral vector containing the complementary deoxyribonucleic acid sequence that encodes for human cystinosin, the lysosomal cystine transporter protein (product name CTNS-RD-04), in patients with cystinosis. The gene discussed is CTNS; the disease is cystinosis.