Nature Communications (Oct 2016)

Semi-automated closed system manufacturing of lentivirus gene-modified haematopoietic stem cells for gene therapy

  • Jennifer E. Adair,
  • Timothy Waters,
  • Kevin G. Haworth,
  • Sara P. Kubek,
  • Grant D. Trobridge,
  • Jonah D. Hocum,
  • Shelly Heimfeld,
  • Hans-Peter Kiem

DOI
https://doi.org/10.1038/ncomms13173
Journal volume & issue
Vol. 7, no. 1
pp. 1 – 10

Abstract

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Current methods for haematopoietic stem cell gene therapy are laborious and require special licensed facilities. Here the authors develop a semi-automated protocol using a commercially available device to allow for benchtop generation of gene-modified blood cell products for transplantation, that meet current standards.