Optimal conditions for lentiviral transduction of engrafting human CD34+ cells

Gene Ther. 2011 Nov;18(11):1078-86. doi: 10.1038/gt.2011.63. Epub 2011 May 5.

Abstract

Cytokines are required for γ-retroviral transduction of human CD34+ cells. However, cytokines may reduce engraftment of CD34+ cells and may not be necessary for their lentiviral transduction. We sought to optimize transduction and engraftment of human CD34+ cells using lentiviral vectors. Single 24 h transduction of human CD34+ cells with human immunodeficiency virus type 1 (HIV1)-based lentiviral vectors in media containing stem cell factor (SCF), FMS-like tyrosine kinase 3 (FLT3) ligand, thrombopoietin (each 100 ng ml⁻¹) and 10% fetal bovine serum was compared with various cytokine conditions during ex vivo culture and assayed using humanized xenograft mice for 6 months after transplantation. Serum-free media improved transduction efficiency of human CD34+ cells. Interleukin-3 (20 ng ml⁻¹) had little effect on transduction efficiency or engraftment. Threefold higher cytokine mixture (each 300 ng ml⁻¹) reduced engraftment of CD34+ cells. SCF alone (100 ng ml⁻¹) proved insufficient for maintaining engraftment ability and reduced transduction efficiency. Short-term prestimulation had little effect on transduction efficiency or engraftment, yet 24 h prestimulation showed higher transduction efficiency, higher gene expression levels and lower engraftment. In summary, 24 h prestimulation followed by single 24-h lentiviral transduction in serum-free media with SCF, FLT3 ligand and thrombopoietin yields high transduction efficiency to engrafting human CD34+ cells, and is applicable in human clinical gene therapy trials.

Publication types

  • Research Support, N.I.H., Intramural

MeSH terms

  • Animals
  • Antigens, CD34 / metabolism*
  • Culture Media, Serum-Free
  • Genetic Vectors
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Interleukin-3 / pharmacology*
  • Lentivirus / genetics*
  • Male
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Models, Animal
  • Stem Cell Factor / immunology
  • Thrombopoietin / immunology
  • Transduction, Genetic*
  • Transplantation, Heterologous
  • fms-Like Tyrosine Kinase 3 / immunology

Substances

  • Antigens, CD34
  • Culture Media, Serum-Free
  • Interleukin-3
  • Stem Cell Factor
  • Thrombopoietin
  • fms-Like Tyrosine Kinase 3