New Methods for Disease Modeling Using Lentiviral Vectors

Trends Mol Med. 2018 Oct;24(10):825-837. doi: 10.1016/j.molmed.2018.08.001. Epub 2018 Sep 10.

Abstract

Lentiviral vectors (LVs) transduce quiescent cells and provide stable integration to maintain transgene expression. Several approaches have been adopted to optimize LV safety profiles. Similarly, LV targeting has been tailored through strategies including the modification of envelope components, the use of specific regulatory elements, and the selection of appropriate administration routes. Models of aortic disease based on a single injection of pleiotropic LVs have been developed that efficiently transduce the three aorta layers in wild type mice. This approach allows the dissection of pathways involved in aortic aneurysm formation and the identification of targets for gene therapy in aortic diseases. LVs provide a fast, efficient, and affordable alternative to genetically modified mice to study disease mechanisms and develop therapeutic tools.

Keywords: aortic aneurysm; disease models; gene therapy; lentiviral vectors; viral targeting.

Publication types

  • Review

MeSH terms

  • ADAMTS1 Protein / antagonists & inhibitors
  • ADAMTS1 Protein / genetics*
  • ADAMTS1 Protein / immunology
  • Animals
  • Aortic Aneurysm / genetics
  • Aortic Aneurysm / immunology
  • Aortic Aneurysm / pathology
  • Aortic Aneurysm / therapy*
  • Clinical Trials as Topic
  • Disease Models, Animal
  • Gene Expression Regulation
  • Genetic Therapy / methods*
  • Genetic Vectors / chemistry*
  • Genetic Vectors / immunology
  • Humans
  • Immunity, Innate
  • Lentivirus / genetics*
  • Lentivirus / immunology
  • Mice
  • Patient Safety
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / metabolism
  • Transduction, Genetic / methods
  • Transgenes

Substances

  • RNA, Small Interfering
  • ADAMTS1 Protein
  • Adamts1 protein, mouse