4.5 Review

Gene therapy progress and prospects: non-viral gene therapy by systemic delivery

Journal

GENE THERAPY
Volume 13, Issue 18, Pages 1313-1319

Publisher

NATURE PUBLISHING GROUP
DOI: 10.1038/sj.gt.3302838

Keywords

non-viral vectors; siRNA; targeted delivery; systemic delivery

Funding

  1. NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES [R56AI048851, R01AI048851] Funding Source: NIH RePORTER
  2. NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES [R01DK068556] Funding Source: NIH RePORTER
  3. NIAID NIH HHS [AI48851] Funding Source: Medline
  4. NIDDK NIH HHS [DK68556] Funding Source: Medline

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Non-viral vectors continue to be an attractive alternative to viral vectors due to their safety, versatility and ease of preparation and scale-up. Over the past few years, investigators have been successful in developing gene carriers that can be targeted to the disease site. Several different delivery vectors for systemic use have been developed by different groups for plasmid DNA and oligonucleotide. Most of them are designed for targeted tumor therapy. The mechanism of inflammatory toxicity, the major toxicity of cationic lipoplex, has been studied and managed. In this review, we focus on the progress made over the last 2 years. We also discuss some future prospects for gene delivery.

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