Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis

Blood. 2003 Feb 15;101(4):1284-9. doi: 10.1182/blood-2002-07-2238. Epub 2002 Oct 17.

Abstract

Efficient vector transduction of hematopoietic stem cells is a requirement for successful gene therapy of hematologic disorders. We asked whether human umbilical cord blood CD34(+)CD38(lo) nonobese diabetic/severe combined immunodeficiency (NOD/SCID) repopulating cells (SRCs) could be efficiently transduced using lentiviral vectors, with a particular focus on the average number of vector copies integrating into these primitive progenitor cells. Mouse bone marrow was analyzed by fluorescence-activated cell-sorter scanner and by semiquantitative polymerase chain reaction (PCR) to determine the transduction efficiency into SRCs. Lentiviral vector transduction resulted in an average of 22% (range, 3%-90%) of the human cells expressing green fluorescent protein (GFP), however, multiple vector copies were present in human hematopoietic cells, with an average of 5.6 +/- 3.3 (n = 12) copies per transduced cell. To confirm the ability of lentiviral vectors to integrate multiple vector copies into SRCs, linear amplification mediated (LAM)-PCR was used to analyze the integration site profile of a selected mouse showing low-level engraftment and virtually all human cells expressing GFP. Individually picked granulocyte macrophage colony-forming unit colonies derived from the bone marrow of this mouse were analyzed and shown to have the same 5 vector integrants within each colony. Interestingly, one integration site of the 5 that were sequenced in this mouse was located in a known tumor-suppressor gene, BRCA1. Therefore, these findings demonstrate the ability of lentiviral vectors to transduce multiple copies into a subset of NOD/SCID repopulating cells. While this is efficient in terms of transduction and transgene expression, it may increase the risk of insertional mutagenesis.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • ADP-ribosyl Cyclase / analysis
  • ADP-ribosyl Cyclase 1
  • Animals
  • Antigens, CD / analysis
  • Antigens, CD34 / analysis
  • Bone Marrow Cells
  • Colony-Forming Units Assay
  • Fetal Blood / cytology
  • Flow Cytometry
  • Genes, Tumor Suppressor
  • Genetic Vectors*
  • Granulocytes
  • Green Fluorescent Proteins
  • Humans
  • Lentivirus / genetics*
  • Luminescent Proteins / genetics
  • Macrophages
  • Membrane Glycoproteins
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Mutagenesis, Insertional*
  • Polymerase Chain Reaction
  • Stem Cell Transplantation
  • Transfection*
  • Virus Integration*

Substances

  • Antigens, CD
  • Antigens, CD34
  • Luminescent Proteins
  • Membrane Glycoproteins
  • Green Fluorescent Proteins
  • ADP-ribosyl Cyclase
  • CD38 protein, human
  • Cd38 protein, mouse
  • ADP-ribosyl Cyclase 1