Introduction to Gene Therapy

  • Mauro Giacca


The idea to use “genes” as “drugs” for human therapy was originally conceived in the United States around the 1970s. It was the logical consequence of at least two major advancements that were occurring in those years, namely the exponentially growing knowledge of human gene function and the impact of their mutations, and the development of progressively more effective technologies for the delivery of DNA into mammalian cells.


Gene Therapy Gene Transfer Muscular Dystrophy Viral Vector Adenoviral Vector 
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Further Reading

  1. Couzin-Frankel J (2009) Genetics. The promise of a cure: 20 years and counting. Science 324:1504–1507CrossRefPubMedGoogle Scholar
  2. Ledley FD (1995) Nonviral gene therapy: the promise of genes as pharmaceutical products. Hum Gene Ther 6:1129–1144CrossRefPubMedGoogle Scholar
  3. Miller DA (1992) Human gene therapy comes of age. Nature 357:455–460CrossRefPubMedGoogle Scholar
  4. Pearson H (2009) Human genetics: one gene, twenty years. Nature 460:164–169CrossRefPubMedGoogle Scholar
  5. Ross G, Erickson R, Knorr D et al (1996) Gene therapy in the United States: a five-year status report. Hum Gene Ther 7:1781–1790CrossRefPubMedGoogle Scholar
  6. Thomas CE, Ehrhardt A, Kay MA (2003) Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet 4:346–358CrossRefPubMedGoogle Scholar
  7. Zaldumbide A, Hoeben RC (2007) How not to be seen: immune-evasion strategies in gene therapy. Gene Ther 15:239–246CrossRefPubMedGoogle Scholar

Copyright information

© Springer-Verlag Italia 2010

Authors and Affiliations

  • Mauro Giacca
    • 1
  1. 1.International Centre for Genetic Engineering and Biotechnology (ICGEB)TriesteItaly

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