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Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells.
Smith, Miles C; Belur, Lalitha R; Karlen, Andrea D; Erlanson, Olivia; Podetz-Pedersen, Kelly M; McKenzie, Jessica; Detellis, Jenn; Gagnidze, Khatuna; Parsons, Geoffrey; Robinson, Nicholas; Labarre, Shelby; Shah, Saumil; Furcich, Justin; Lund, Troy C; Tsai, Hsing-Chen; McIvor, R Scott; Bonner, Melissa.
Afiliación
  • Smith MC; Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, Minnesota, USA.
  • Belur LR; Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, Minnesota, USA.
  • Karlen AD; Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, Minnesota, USA.
  • Erlanson O; Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, Minnesota, USA.
  • Podetz-Pedersen KM; Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, Minnesota, USA.
  • McKenzie J; bluebird bio, Inc., Cambridge, Massachusetts, USA.
  • Detellis J; bluebird bio, Inc., Cambridge, Massachusetts, USA.
  • Gagnidze K; bluebird bio, Inc., Cambridge, Massachusetts, USA.
  • Parsons G; bluebird bio, Inc., Cambridge, Massachusetts, USA.
  • Robinson N; bluebird bio, Inc., Cambridge, Massachusetts, USA.
  • Labarre S; bluebird bio, Inc., Cambridge, Massachusetts, USA.
  • Shah S; bluebird bio, Inc., Cambridge, Massachusetts, USA.
  • Furcich J; Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
  • Lund TC; Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
  • Tsai HC; bluebird bio, Inc., Cambridge, Massachusetts, USA.
  • McIvor RS; Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, Minnesota, USA.
  • Bonner M; bluebird bio, Inc., Cambridge, Massachusetts, USA.
Hum Gene Ther ; 33(23-24): 1279-1292, 2022 12.
Article en En | MEDLINE | ID: mdl-36226412
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Mucopolisacaridosis II / Iduronato Sulfatasa Límite: Animals / Humans Idioma: En Año: 2022 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Mucopolisacaridosis II / Iduronato Sulfatasa Límite: Animals / Humans Idioma: En Año: 2022 Tipo del documento: Article