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First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B.
Harmatz, Paul; Prada, Carlos E; Burton, Barbara K; Lau, Heather; Kessler, Craig M; Cao, Liching; Falaleeva, Marina; Villegas, Andres G; Zeitler, Jennifer; Meyer, Kathleen; Miller, Weston; Wong Po Foo, Cheryl; Vaidya, Sagar; Swenson, Wendy; Shiue, Lisa H; Rouy, Didier; Muenzer, Joseph.
Affiliation
  • Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, CA 94609, USA. Electronic address: paul.harmatz@ucsf.edu.
  • Prada CE; Division of Genetics, Birth Defects & Metabolism, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA; Department of Pediatrics, Feinberg School of Medicine of Northwestern University, Chicago, IL, USA; Division of Human Genetics, Cincinnati Children's Hospital Medical Cen
  • Burton BK; Division of Genetics, Birth Defects & Metabolism, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA; Department of Pediatrics, Feinberg School of Medicine of Northwestern University, Chicago, IL, USA.
  • Lau H; Department of Neurology, NYU School of Medicine, New York, NY, USA.
  • Kessler CM; Georgetown University, Washington, DC, USA.
  • Cao L; Sangamo Therapeutics, Inc., Brisbane, CA, USA.
  • Falaleeva M; Sangamo Therapeutics, Inc., Brisbane, CA, USA.
  • Villegas AG; Sangamo Therapeutics, Inc., Brisbane, CA, USA.
  • Zeitler J; Sangamo Therapeutics, Inc., Brisbane, CA, USA.
  • Meyer K; Sangamo Therapeutics, Inc., Brisbane, CA, USA.
  • Miller W; Sangamo Therapeutics, Inc., Brisbane, CA, USA.
  • Wong Po Foo C; Sangamo Therapeutics, Inc., Brisbane, CA, USA.
  • Vaidya S; Sangamo Therapeutics, Inc., Brisbane, CA, USA.
  • Swenson W; Sangamo Therapeutics, Inc., Brisbane, CA, USA.
  • Shiue LH; Sangamo Therapeutics, Inc., Brisbane, CA, USA.
  • Rouy D; Sangamo Therapeutics, Inc., Brisbane, CA, USA.
  • Muenzer J; University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
Mol Ther ; 30(12): 3587-3600, 2022 12 07.
Article de En | MEDLINE | ID: mdl-36299240
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Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Sujet principal: Nucléases à doigts de zinc Limites: Humans Langue: En Journal: Mol Ther Sujet du journal: BIOLOGIA MOLECULAR / TERAPEUTICA Année: 2022 Type de document: Article Pays de publication: États-Unis d'Amérique
Texte intégral
Ajouter à My VHL
Imprimer
XML
PubMed Links
Recherche sur Google

Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Sujet principal: Nucléases à doigts de zinc Limites: Humans Langue: En Journal: Mol Ther Sujet du journal: BIOLOGIA MOLECULAR / TERAPEUTICA Année: 2022 Type de document: Article Pays de publication: États-Unis d'Amérique