CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells.
Goodwin M, Lee E, Lakshmanan U, Shipp S, Froessl L, Barzaghi F, Passerini L, Narula M, Sheikali A, Lee CM, Bao G, Bauer CS, Miller HK, Garcia-Lloret M, Butte MJ, Bertaina A, Shah A, Pavel-Dinu M, Hendel A, Porteus M, Roncarolo MG, Bacchetta R.
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Sci Adv. 2020 May 6;6(19):eaaz0571. doi: 10.1126/sciadv.aaz0571. eCollection 2020 May.
Sci Adv. 2020.
PMID: 32494707
Free PMC article.