Nonviral approaches for neuronal delivery of nucleic acids

Jamie M. Bergen, In Kyu Park, Philip J. Horner, Suzie H. Pun

Research output: Contribution to journalReview article

93 Scopus citations

Abstract

The delivery of therapeutic nucleic acids to neurons has the potential to treat neurological disease and spinal cord injury. While select viral vectors have shown promise as gene carriers to neurons, their potential as therapeutic agents is limited by their toxicity and immunogenicity, their broad tropism, and the cost of large-scale formulation. Nonviral vectors are an attractive alternative in that they offer improved safety profiles compared to viruses, are less expensive to produce, and can be targeted to specific neuronal subpopulations. However, most nonviral vectors suffer from significantly lower transfection efficiencies than neurotropic viruses, severely limiting their utility in neuron-targeted delivery applications. To realize the potential of nonviral delivery technology in neurons, vectors must be designed to overcome a series of extra- and intracellular barriers. In this article, we describe the challenges preventing successful nonviral delivery of nucleic acids to neurons and review strategies aimed at overcoming these challenges.

Original languageEnglish (US)
Pages (from-to)983-998
Number of pages16
JournalPharmaceutical Research
Volume25
Issue number5
DOIs
StatePublished - May 2008

Keywords

  • CNS
  • Neuron targeting
  • Nonviral gene delivery
  • Nucleic acid delivery

ASJC Scopus subject areas

  • Biotechnology
  • Molecular Medicine
  • Pharmacology
  • Pharmaceutical Science
  • Organic Chemistry
  • Pharmacology (medical)

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