Systemic Gene Delivery by Single-Dose Intracardiac Administration of scAAV2/9 and scAAV2/rh10 Variants in Newborn Rats

Hum Gene Ther Methods. 2018 Aug;29(4):189-199. doi: 10.1089/hgtb.2017.192.r3.

Abstract

Recombinant adeno-associated virus serotype 9 (rAAV2/9) and pseudotype rhesus-10 (rAAV2/rh10) are used for gene delivery, especially into the central nervous system. Both serotypes cross the blood-brain barrier and mediate stable long-term transduction in dividing and nondividing cells. Among possible routes of administration, intracardiac injection holds the potential for widespread vector diffusion associated with a relatively simple approach. In this study adopting the intracardiac route, we compare the cell-specific tropism and transfection efficacy of a panel of engineered rAAV2/9 and rAAV2/rh10 vectors encoding the enhanced green fluorescent protein. We observed transduction in the brain and peripherally, with a predominant neuronal tropism while the various serotypes achieved different expression patterns.

Keywords: AAV; CNS; animal model; intracardiac injection; rat.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Blood-Brain Barrier / metabolism
  • Dependovirus / genetics
  • Dependovirus / metabolism
  • Gene Transfer Techniques / adverse effects
  • Gene Transfer Techniques / standards*
  • Genetic Therapy / methods*
  • Genetic Vectors / genetics*
  • Genetic Vectors / metabolism
  • HEK293 Cells
  • Humans
  • Myocardium / metabolism*
  • Rats
  • Rats, Sprague-Dawley
  • Serogroup