T cell correction pipeline for Inborn Errors of Immunity
Katariina Mamia, Sólrún Kolbeinsdóttir, Kornel Labun, Zhuokun Li, Anna Zofia Komisarczuk, Salla Keskitalo, Ganna Reint, Frida Loe Haugen, Britt Olaug Lindestad, Siv Skundberg Jensen, Thea Johanne Gjerdingen, Antti Tuhkala, Carolina Wieczorek Ervik, Pavel Kopcil, Nail Fatkhutdinov, , Emma Maria Haapaniemi
This paper presents a T cell gene correction pipeline using CRISPR/Cas9 and HDR to correct monogenic mutations in IEI models, achieving up to 80% bi-allelic editing with no detectable off-target effects.
Molecular Therapy · Aug 12, 2025Peer-reviewedBioengineering