Single-Cell RNA sequencing reveals prophylactic Prednisone blunts cellular immunity in hemophilia B gene therapy. Single-Cell RNA sequencing reveals prophylactic Prednisone blunts cellular immunity in hemophilia B gene therapy

Seven hemophilia B gene therapy participants were taken blood for single cell sequencing at four timepoints, and then compared the immune status of T and antigen presentation cells of theses patients according to therapy efficacy and different time points. The results showed that cellular immunity was elevated in patients with FIX:C less then 40IU/dl at week 26, glucocorticoids (GCs) effectively suppressed T cell immunity and GCs inhibited T cell immunity indirectly by effecting on APCs. Overall design: Examination of single cell mRNA profile in peripheral blood mononuclear cell from seven hemophilia B gene therapy participants at following time points：Day-7(before glucocorticoids treatment), Day 0（before AAV infusion）, Day 7(after AAV infusion) and 2 weeks after glucocorticoids withdrawal(stop GCs) .