CRISPR-based gene editing enables <i>FOXP3</i> gene repair in IPEX patient cells

Marianne Goodwin(Stanford University), Rosa Bacchetta(The San Raffaele Telethon Institute for Gene Therapy), Mara Pavel-Dinu(Stanford University), Maria Garcia-Lloret(University of California, Los Angeles), Uma Lakshmanan(Stanford University), Suzette Shipp(Stanford University), Mansi Narula(Stanford University), Ciaran M. Lee(University College Cork), Manish J. Butte(University of California, Los Angeles), Gang Bao(Rice University), Ami J. Shah(University of California, Los Angeles), Alice Bertaina(Stanford University), Federica Barzaghi(The San Raffaele Telethon Institute for Gene Therapy), Ayal Hendel(Bar-Ilan University), Adam Sheikali(Stanford University), Luise Froessl(Stanford University), Cindy S. Bauer(Phoenix Children's Hospital), Laura Passerini(The San Raffaele Telethon Institute for Gene Therapy), Matthew H. Porteus(Stanford University), Maria‐Grazia Roncarolo(Regenerative Medicine Institute), Holly Miller(Phoenix Children's Hospital)
Science Advances
May 6, 2020
Cited by 113


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