Efficient transduction of human neurons with an adeno-associated virus vector

B. Du, Ping Wu, D. M. Boldt-Houle, E. F. Terwilliger

Research output: Contribution to journalArticle

66 Citations (Scopus)

Abstract

Anadeno-associated virus vector containing a lacZ gene driven by a CMV immediate-early promoter (AAVβ-gal) was evaluated with respect to its transduction efficiency and integration ability in nondividing human NT neurons. A dose-dependent pattern in transduction efficiency of the AAVβ-gal was demonstrated immunocytochemically, with up to 100% of the neurons expressing the gene product. No neurotoxic effects of the vector were detected. Quantitative PCR analyses of high molecular weight cellular DNA from the transduced neurons indicated that the copy number of the AAVβ-gal genome increased gradually in a time-dependent manner, suggesting a slow but progressive rate of vector integration over a period of approximately 1 week following transduction. Equal or greater transduction efficiency of the AAVβ-gal into NT neurons than into a standard target cell line indicated that the NT neurons were readily susceptible to AAV-mediated gene transfer. This study demonstrates that AAV-based vectors can efficiently transduce and stably express a foreign gene in post-mitotic human neurons.

Original languageEnglish (US)
Pages (from-to)254-261
Number of pages8
JournalGene Therapy
Volume3
Issue number3
StatePublished - Mar 1996
Externally publishedYes

Fingerprint

Dependovirus
Neurons
Genes
Satellite Viruses
Lac Operon
Molecular Weight
Genome
Cell Line
Polymerase Chain Reaction
DNA

Keywords

  • β-galactosidase
  • AAV
  • Gene transfer
  • Human NT neuron

ASJC Scopus subject areas

  • Genetics

Cite this

Du, B., Wu, P., Boldt-Houle, D. M., & Terwilliger, E. F. (1996). Efficient transduction of human neurons with an adeno-associated virus vector. Gene Therapy, 3(3), 254-261.

Efficient transduction of human neurons with an adeno-associated virus vector. / Du, B.; Wu, Ping; Boldt-Houle, D. M.; Terwilliger, E. F.

In: Gene Therapy, Vol. 3, No. 3, 03.1996, p. 254-261.

Research output: Contribution to journalArticle

Du, B, Wu, P, Boldt-Houle, DM & Terwilliger, EF 1996, 'Efficient transduction of human neurons with an adeno-associated virus vector', Gene Therapy, vol. 3, no. 3, pp. 254-261.
Du B, Wu P, Boldt-Houle DM, Terwilliger EF. Efficient transduction of human neurons with an adeno-associated virus vector. Gene Therapy. 1996 Mar;3(3):254-261.
Du, B. ; Wu, Ping ; Boldt-Houle, D. M. ; Terwilliger, E. F. / Efficient transduction of human neurons with an adeno-associated virus vector. In: Gene Therapy. 1996 ; Vol. 3, No. 3. pp. 254-261.
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