A capsid-modified adenovirus vector devoid of all viral genes: assessment of transduction and toxicity in human hematopoietic cells

Mol Ther. 2001 Jul;4(1):36-44. doi: 10.1006/mthe.2000.0410.

Abstract

Inefficient gene transfer has limited the success of gene therapy in the hematopoietic system. Here we develop a novel chimeric adenovirus (Ad) vector containing Ad serotype 11 fiber-modified capsids and E1/E3 deleted viral genomes (Ad5/11) or genomes devoid of all viral genes (DeltaAd5/11). The capsid-modified vectors transduced human hematopoietic cells more efficiently than the unmodified Ad5-based vector. The absence of viral genes from the DeltaAd5/11 vector allowed for transduction without the associated toxicity seen with the first-generation E1/E3 deleted vector. Chimeric vectors were used for transient expression of the ecotropic retrovirus receptor (ecoR) in Mo7e cells (a CD34-positive, c-Kit-positive, growth-factor-dependent human cell line) as a model for human hematopoietic progenitor cells. Expression of ecoR conferred susceptibility to subsequent retroviral transduction. The DeltaAd5/11 vector used to express ecoR allowed for expansion of retrovirally transduced cells, whereas transduction with the first-generation Ad5/11 vector resulted in cytotoxicity and, over time, loss of cells expressing the retrovirus-vector-derived transgene.

Publication types

  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adenoviridae* / genetics*
  • Animals
  • Antigens, CD34 / biosynthesis
  • Apoptosis
  • Capsid / genetics
  • Capsid / metabolism
  • Capsid / physiology
  • Cell Line
  • Cell Survival
  • Cells, Cultured
  • Gene Deletion
  • Gene Transfer Techniques*
  • Genetic Therapy / methods
  • Genetic Vectors* / toxicity
  • Genome, Viral*
  • Hematopoietic Stem Cells*
  • Humans
  • Rats
  • Transduction, Genetic
  • Transgenes

Substances

  • Antigens, CD34