Exogenous gene expression and growth regulation of hematopoietic cells via a novel human artificial chromosome

J Hum Genet. 2006;51(2):147-150. doi: 10.1007/s10038-005-0334-9. Epub 2005 Dec 7.

Abstract

A number of gene delivery systems are currently being developed for potential use in gene therapy. Here, we demonstrate the feasibility of 21deltaqHAC, a newly developed human artificial chromosome (HAC), as a gene delivery system. We first introduced a 21deltaqHAC carrying an EGFP reporter gene and a geneticin-resistant gene (EGFP-21deltaqHAC) into hematopoietic cells by microcell-mediated chromosome transfer. These HAC-containing hematopoietic cells showed resistance to geneticin, expressed EGFP and retained the ability to differentiate into various lineages, and the EGFP-21deltaqHAC was successfully transduced into primary hematopoietic cells. Hematopoietic cells harboring the EGFP-21deltaqHAC could still be detected at two weeks post-transplantation in immunodeficient mice. We also showed effective expansion of hematopoietic cells by introducing the 21deltaqHAC containing ScFvg, a gp130-based chimeric receptor that transmits growth signals in response to specific-antigen of this receptor. All of these results demonstrate the usefulness of HAC in gene therapy.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Chromosomes, Artificial, Human / genetics*
  • Chromosomes, Human, Pair 21 / genetics*
  • Cord Blood Stem Cell Transplantation / methods*
  • DNA Primers
  • Flow Cytometry
  • Gene Expression*
  • Genetic Therapy / methods*
  • Genetic Vectors / genetics
  • Gentamicins
  • Green Fluorescent Proteins / metabolism
  • Hematopoietic Stem Cells / metabolism*
  • Humans
  • Mice

Substances

  • DNA Primers
  • Gentamicins
  • enhanced green fluorescent protein
  • Green Fluorescent Proteins
  • antibiotic G 418