Preclinical efficacy and safety assessment of engineered regulatory T cells for treatment of IPEX and other autoimmune disorders - PubMed
5 hours ago
- #regulatory T cells
- #gene therapy
- #autoimmune disorders
- Engineered regulatory T cells (EngTreg) were developed using HDR-based editing of the FOXP3 gene in CD4+ T cells.
- EngTreg therapy shows potential for treating IPEX syndrome, a severe autoimmune disorder caused by FOXP3 mutations.
- Pre-clinical studies demonstrated EngTreg's efficacy in suppressing effector T cells in vitro and reversing GvHD in vivo.
- Safety assessments in mouse models confirmed EngTreg's safety in humoral responses and viral infection control.
- EngTreg presents a promising cellular therapeutic approach for IPEX and possibly other autoimmune disorders.