Herpesvirus-mediated systemic delivery of nerve growth factor

Mol Ther. 2001 Jan;3(1):61-9. doi: 10.1006/mthe.2000.0225.

Abstract

Sustained systemic dissemination of therapeutic proteins from peripheral sites is an attractive prospect for gene therapy applications. Replication-defective genomic herpes simplex virus type 1 (HSV-1) vectors were evaluated for their ability to express nerve growth factor (NGF) as a model gene product both locally and systemically. Intra-articular inoculation of NGF expression vectors in rabbits resulted in significant increases in joint lavage and blood plasma NGF that persisted for 1 year. A rhesus macaque injected intra-articularly displayed a comparable increase in plasma NGF for at least 6 months, at which time the serum NGF levels of this animal were sufficient to cause differentiation of PC12 cells in culture, but not to increase footpad epidermis innervation. Long-term reporter transgene expression was observed primarily in ligaments, a finding confirmed by direct inoculation of patellar ligament. Patellar ligament inoculation with a NGF vector resulted in elevated levels of circulating NGF similar to those observed following intra-articular vector delivery. These results represent the first demonstration of sustained systemic release of a transgene product using HSV vectors, raising the prospect of new applications for HSV-1 vectors in the treatment of systemic disease.

Publication types

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

MeSH terms

  • Animals
  • Epidermis / innervation
  • Gene Deletion
  • Gene Transfer Techniques*
  • Genetic Vectors*
  • Herpesvirus 1, Human / genetics*
  • Lac Operon
  • Ligaments / metabolism
  • Macaca mulatta
  • Nerve Growth Factor / blood
  • Nerve Growth Factor / genetics*
  • PC12 Cells
  • Rabbits
  • Rats
  • Reverse Transcriptase Polymerase Chain Reaction
  • Time Factors
  • Transgenes

Substances

  • Nerve Growth Factor