Nonintegrating Gene Therapy Vectors

Hematol Oncol Clin North Am. 2017 Oct;31(5):753-770. doi: 10.1016/j.hoc.2017.06.007.


Gene delivery vectors that do not rely on host cell genome integration offer several advantages for gene transfer, chiefly the avoidance of insertional mutagenesis and position effect variegation. However, unless engineered for replication and segregation, nonintegrating vectors will dilute progressively in proliferating cells, and are not exempt of epigenetic effects. This article provides an overview of the main nonintegrating viral (adenoviral, adeno-associated viral, integration-deficient retro-lentiviral, poxviral), and nonviral (plasmid vectors, artificial chromosomes) vectors used for preclinical and clinical cell and gene therapy applications. Particular emphasis is placed on their use in hematologic disease.

Keywords: Adeno-associated virus vectors; Adenovirus vectors; Gene therapy; Genome editing; Integration-deficient lentiviral vectors (IDLVs); Nonintegrating; Plasmid; Poxvirus vector.

Publication types

  • Historical Article
  • Review
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenoviridae / genetics
  • Animals
  • Clinical Trials as Topic / history
  • Dependovirus / genetics
  • Gene Editing
  • Gene Expression
  • Gene Transfer Techniques
  • Genetic Therapy* / adverse effects
  • Genetic Therapy* / history
  • Genetic Therapy* / methods
  • Genetic Vectors / classification
  • Genetic Vectors / genetics*
  • History, 20th Century
  • History, 21st Century
  • Humans
  • Plasmids / genetics
  • Poxviridae / genetics
  • Transduction, Genetic