CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells

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 immu...

詳細記述

保存先:
書誌詳細
出版年:Sci Adv
主要な著者: 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.
フォーマット: Artigo
言語:Inglês
出版事項: American Association for the Advancement of Science 2020
主題:
Research Articles
オンライン･アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC7202871/
https://ncbi.nlm.nih.gov/pubmed/32494707
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.aaz0571
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