Dr. Hendel Ayal – CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells

Dr. Ayal Hendel

Abstract The prototypical genetic autoimmune disease is immune dysregulation polyendocrinopathy enteropathy X-linked (IPEX) syndrome, a severe pediatric disease with limited treatment options. IPEX syndrome is caused by mutations in the forkhead box protein 3 (FOXP3) gene, which plays a critical role in immune regulation. As a monogenic disease, IPEX is an ideal candidate for a…

Read More