Article
CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells.
Science advances - 1 May 2020
Goodwin M, Lee E, Lakshmanan U, Shipp S, Froessl L, Barzaghi F, Passerini L, Narula M, Sheikali A, Lee C M, Bao G, Bauer C S, Miller H K, Garcia-Lloret M, Butte M J, Bertaina A, Shah A, Pavel-Dinu M, Hendel A, Porteus M, Roncarolo M G, Bacchetta R
Abstract excerpt
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 therapeutic approach in which autologous...
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