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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, 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.
Affiliation
  • Goodwin M; Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
  • Lee E; Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
  • Lakshmanan U; Institute for Stem Cell Biology and Regenerative Medicine (ISCBRM), Stanford University School of Medicine, Stanford, CA, USA.
  • Shipp S; Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
  • Froessl L; Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
  • Barzaghi F; Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
  • Passerini L; Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy.
  • Narula M; Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy.
  • Sheikali A; Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
  • Lee CM; Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
  • Bao G; APC Microbiome Ireland, University College Cork, Cork, Ireland.
  • Bauer CS; Department of Bioengineering, George R. Brown School of Engineering, Rice University, Houston, TX, USA.
  • Miller HK; Phoenix Children's Hospital, Phoenix, AZ, USA.
  • Garcia-Lloret M; Phoenix Children's Hospital, Phoenix, AZ, USA.
  • Butte MJ; Division of Immunology, Allergy, and Rheumatology, Department of Pediatrics, University of California, Los Angeles, Los Angeles, CA, USA.
  • Bertaina A; Division of Immunology, Allergy, and Rheumatology, Department of Pediatrics, University of California, Los Angeles, Los Angeles, CA, USA.
  • Shah A; Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
  • Pavel-Dinu M; Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
  • Hendel A; Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
  • Porteus M; Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
  • Roncarolo MG; The Institute for Advanced Materials and Nanotechnology, The Mina and Everard Goodman Faculty of Life Sciences, Ramat-Gan 52900, Israel.
  • Bacchetta R; Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
Sci Adv ; 6(19): eaaz0571, 2020 05.
Article in En | MEDLINE | ID: mdl-32494707
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Diseases, X-Linked / Gene Editing Limits: Child / Humans Language: En Journal: Sci Adv Year: 2020 Document type: Article
Fulltext
Print
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PubMed Links
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Diseases, X-Linked / Gene Editing Limits: Child / Humans Language: En Journal: Sci Adv Year: 2020 Document type: Article