Human artificial chromosome-based gene delivery vectors for biomedicine and biotechnology

Expert Opin Drug Deliv. 2014 Apr;11(4):517-35. doi: 10.1517/17425247.2014.882314. Epub 2014 Jan 30.


Introduction: Human artificial chromosomes (HACs) have several advantages over viruses as gene delivery vectors, including stable episomal maintenance in a single copy and the ability to carry large gene inserts.

Areas covered: In this review, we summarise recent work on gene transfer into mammalian cells using the HACs. HACs allow therapeutic transgenes to be expressed in target cells under conditions that recapitulate the physiological regulation of endogenous loci.

Expert opinion: Based on the published data, the HAC vectors have a great potential for gene therapy, regenerative medicine, screening of anticancer drugs and biotechnology.

Publication types

  • Research Support, N.I.H., Intramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Biomedical Technology
  • Chromosomes, Artificial*
  • Gene Transfer Techniques*
  • Genetic Engineering / methods
  • Genetic Therapy / methods*
  • Genetic Vectors*
  • Humans
  • Stem Cells / physiology*