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Gene therapy progress and prospects: RNA aptamers.

Publication ,  Journal Article
Que-Gewirth, NS; Sullenger, BA
Published in: Gene Ther
February 2007

Aptamers are oligonucleotides evolved in vitro or in nature to bind target ligands with high affinity and specificity. They are emerging as powerful tools in the fields of therapeutics, drug development, target validation and diagnostics. Aptamers are attractive alternatives to antibody- and small-molecule-based therapeutics owing to their stability, low toxicity, low immunogenicity and improved safety. With the recent approval of the first aptamer drug Macugen by the US FDA, there is great impetus to develop therapeutic aptamers that can target a wide array of disease states. The recent demonstration that aptamer activity can be reversed by the administration of a simple antidote greatly enhances the potential value of aptamers as therapeutic agents.

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Published In

Gene Ther

DOI

ISSN

0969-7128

Publication Date

February 2007

Volume

14

Issue

4

Start / End Page

283 / 291

Location

England

Related Subject Headings

  • Stem Cell Transplantation
  • Humans
  • Genetic Therapy
  • Gene Transfer Techniques
  • Forecasting
  • Biotechnology
  • Aptamers, Nucleotide
  • 42 Health sciences
  • 32 Biomedical and clinical sciences
  • 31 Biological sciences
 

Citation

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Chicago
ICMJE
MLA
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Que-Gewirth, N. S., & Sullenger, B. A. (2007). Gene therapy progress and prospects: RNA aptamers. Gene Ther, 14(4), 283–291. https://doi.org/10.1038/sj.gt.3302900
Que-Gewirth, N. S., and B. A. Sullenger. “Gene therapy progress and prospects: RNA aptamers.Gene Ther 14, no. 4 (February 2007): 283–91. https://doi.org/10.1038/sj.gt.3302900.
Que-Gewirth NS, Sullenger BA. Gene therapy progress and prospects: RNA aptamers. Gene Ther. 2007 Feb;14(4):283–91.
Que-Gewirth, N. S., and B. A. Sullenger. “Gene therapy progress and prospects: RNA aptamers.Gene Ther, vol. 14, no. 4, Feb. 2007, pp. 283–91. Pubmed, doi:10.1038/sj.gt.3302900.
Que-Gewirth NS, Sullenger BA. Gene therapy progress and prospects: RNA aptamers. Gene Ther. 2007 Feb;14(4):283–291.

Published In

Gene Ther

DOI

ISSN

0969-7128

Publication Date

February 2007

Volume

14

Issue

4

Start / End Page

283 / 291

Location

England

Related Subject Headings

  • Stem Cell Transplantation
  • Humans
  • Genetic Therapy
  • Gene Transfer Techniques
  • Forecasting
  • Biotechnology
  • Aptamers, Nucleotide
  • 42 Health sciences
  • 32 Biomedical and clinical sciences
  • 31 Biological sciences