What are aptamers used for?

Molecular Biotechnology pp 455-474 | Cite as

  • David P. Clark
  • Nanette J. Pazdernik


An organism is permanently changed by genetic engineering so that these changes are passed on in a stable manner. In the case of multicellular organisms, this requires the conscious modification of the DNA in the cells of the germline. A Gene therapy is less permanent: genes are only changed in part of a patient's body. For example, partial cure in patients with cystic fibrosis can be achieved by introducing the wild-type gene into the lungs. However, these modifications are not passed on - the alleles in the germ line cells remain defective.

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further reading

  1. Aartsma-Rus A, van Ommen GJ (2007) Antisense-mediated exon skipping: A versatile tool with therapeutic and research applications. RNA 13: 1609-1624 CrossRefGoogle Scholar
  2. Athanasopoulos T, Graham IR, Foster H, Dickson G (2004) Recombinant adeno-associated viral (rAAV) vectors as therapeutic tools for Duchenne muscular dystrophy (DMD). Gene Ther 11 (Suppl. 1): 109-121 CrossRefGoogle Scholar
  3. Bagheri S, Kashani-Sabet M (2004) Ribozymes in the age of molecular therapeutics. Curr Mol Med 4: 489-506 CrossRefGoogle Scholar
  4. Conese M, Boyd AC, Di Gioia S, Auriche C, Ascenzioni F (2007) Genomic context vectors and artificial chromosomes for cystic fibrosis gene therapy. Curr Gene Ther 7: 175-187 CrossRefGoogle Scholar
  5. Devi GR (2006) siRNA-based approaches in cancer therapy. Cancer Gene Ther 13: 819-829 CrossRefGoogle Scholar
  6. Duan D (2006) Challenges and opportunities in dystrophindeficient cardiomyopathy gene therapy. Hum Mol Genet 15: R253 – R261CrossRefGoogle Scholar
  7. Fichou Y, Férec C (2006) The potential of oligonucleotides for therapeutic applications. Trends Biotechnol 24: 563-570 CrossRefGoogle Scholar
  8. Foster K, Foster H, Dickson JG (2006) Gene therapy progress and prospects: Duchenne muscular dystrophy. Gene Ther 13: 1677-1685 CrossRefGoogle Scholar
  9. Fritz JJ, Gorbatyuk M, Lewin AS, Hauswirth WW (2004) Design and validation of therapeutic hammerhead ribozymes for autosomal dominant diseases. Methods Mol Biol 252: 221-236 Google Scholar
  10. Gleave ME, Monia BP (2005) Antisense therapy for cancer. Nat Rev Cancer 5: 468-479 CrossRefGoogle Scholar
  11. Jang JH, Lim KI, Schaffer DV (2007) Library selection and directed evolution approaches to engineering targeted viral vectors. Biotechnol Bioeng 98: 515-524 CrossRefGoogle Scholar
  12. Lee JE, Choi JH, Lee MG (2005) Gene SNPs and mutations in clinical genetic testing: Haplotype-based testing and analysis. Mutat Res 573: 195-204 CrossRefGoogle Scholar
  13. Li SD, Huang L (2006) Gene therapy progress and prospects: Non-viral gene therapy by systemic delivery. Gene Ther 13: 1313-1319 CrossRefGoogle Scholar
  14. Loewen N, Poeschla EM (2005) Lentiviral vectors. Adv Biochem Eng Biotechnol 99: 169-191 Google Scholar
  15. Lu PY, Xie F, Woodle MC (2005) In vivo Application of RNA interference: From functional genomics to therapeutics. Adv Genet 54: 117-142 Google Scholar
  16. Pelletier R, Caron SO, Puymirat J (2006) RNA based gene therapy for dominantly inherited diseases. Curr Gene Ther 6: 131-146 CrossRefGoogle Scholar
  17. Warrington KH Jr, Herzog RW (2006) Treatment of human disease by adeno-associated viral gene transfer. Human Genet 119: 571-603 CrossRefGoogle Scholar
  18. Wilton SD, Fletcher S (2006) Modification of pre-mRNA processing: Application to dystrophin expression. Curr Opin Mol Ther 8: 130-135 Google Scholar
  19. Wu Z, Asokan A, Samulski RJ (2006) Adeno associated virus serotypes: Vector toolkit for human gene therapy. Mol Ther 14: 316-327 CrossRefGoogle Scholar

Copyright information

© Spectrum Academic Publishers Heidelberg 2009

Authors and Affiliations

  • David P. Clark
  • Nanette J. Pazdernik

There are no affiliations available