Gene correction of HAX1 reversed Kostmann disease phenotype in patient-specific induced pluripotent stem cells.
Pittermann E, Lachmann N, MacLean G, Emmrich S, Ackermann M, Gohring G, Schlegelberger B, Welte K, Schambach A, Heckl D, Orkin SH, Cantz T, Klusmann JH
Blood Adv. 2017 Jun 2;1(14):903-914. doi: 10.1182/bloodadvances.2016003798. eCollection 2017 Jun 13.
(Link opens in a new window)
PubMed
(Link opens in a new window)
Article
Plasmids from Article
No plasmids are currently publicly available from Addgene for this article.