Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • News & Views
  • Published:

Gene therapy: Progress, problems, prospects.

A recent meeting highlights the progress and the challenges of gene therapy.

This is a preview of subscription content, access via your institution

Relevant articles

Open Access articles citing this article.

Access options

Buy this article

Prices may be subject to local taxes which are calculated during checkout

References

  1. Fisher, K.J. et al. Recombinant adeno-associated virus for muscle directed gene therapy. Nature Med. 3, 306–312 (1997).

    Article  CAS  Google Scholar 

  2. Kay, M.A. et al. Long-term adenovirus-medicated gene expression in mice following CTLA41g administration. Nature Genet. 11, 191–197 (1995).

    Article  CAS  Google Scholar 

  3. Koeberl, D.D., Alexander, I.E., Halbert, C.L., Russell, D.W. & Miller, A.D. Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virous vectors. PNAS USA 94, 1426–1431 (1997).

    Article  CAS  Google Scholar 

  4. Ilan, Y. et al Insertion of the adenoviral E3 region into a recombinant viral vector prevents ativiral humoral and cellular immune responses and permits long-term gene expression. PNAS USA 94, 2587–2592 (1997).

    Article  CAS  Google Scholar 

  5. Fasbender, A. et al. Complexes of adenovirus with polycationic polymers and cationic lipids increase the efficiency of gene transfer in vitro and in vivo. I. Biol. Chem. 272, 6479–6489 (1997).

    Article  CAS  Google Scholar 

  6. Naldini, L., Blomer, U., Gage, F.H., Trono, D. & Verma, I.M. Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. PNAS USA 93, 11382–11388 (1996).

    Article  CAS  Google Scholar 

  7. Tinsley, J.M. et al Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene. Nature 384, 349–353 (1996).

    Article  CAS  Google Scholar 

  8. Bohl, D., Naffakh, N. & Heard, J.M. Long-term control of erythropoietin secretion by doxycycline in mice transplanted with engineered primary myoblasts. Nature Med. 3, 299–305 (1997).

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Rights and permissions

Reprints and permissions

About this article

Cite this article

Blau, H., Khavari, P. Gene therapy: Progress, problems, prospects.. Nat Med 3, 612–613 (1997). https://doi.org/10.1038/nm0697-612

Download citation

  • Issue Date:

  • DOI: https://doi.org/10.1038/nm0697-612

This article is cited by

Search

Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing