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.

  • Letters to the Editor
  • Published:

Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement

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

Relevant articles

Open Access articles citing this article.

Access options

Buy this article

Purchase on Springer Link

Instant access to full article PDF

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

Figure 1: Selective advantage of genetically modified PBL with restoration of ADA activity and T-cell functions following PEG-ADA discontinuation.

References

  1. Bordignon, C. et al. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients. Science 270, 470–475 (1995).

    Article  CAS  Google Scholar 

  2. Blaese, R.M. et al. T lymphocyte–directed gene therapy for ADA-SCID: Initial trial results after 4 years. Science 270, 475–480 (1995).

    Article  CAS  Google Scholar 

  3. Kohn, D.B. et al. Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nature Med. 1, 1017–1023 (1995).

    Article  CAS  Google Scholar 

  4. Hoogerbrugge, P.M. et al. Bone marrow gene transfer in three patients with adenosine deaminase deficiency. Gene Ther. 3, 179–183 (1996).

    CAS  PubMed  Google Scholar 

  5. Onodera, M. et al. Successful peripheral T-lymphocyte-directed gene transfer for a patient with severe combined immune deficiency caused by adenosine deaminase deficiency. Blood 91, 30–36 (1998).

    CAS  PubMed  Google Scholar 

  6. Ochs, H.D. et al. Antibody responses to bacteriophage phi X174 in patients with adenosine deaminase deficiency. Blood 80, 1163–1171 (1992).

    CAS  PubMed  Google Scholar 

  7. Parkman, R., Gelfand, E.W., Rosen, F.S., Sanderson, A. & Hirschhorn, R. Severe combined immunodeficiency and adenosine deaminase deficiency. N. Engl. J. Med. 292, 714–719 (1975).

    Article  CAS  Google Scholar 

  8. Hirschhorn, R., Roegner, M.V., Kuritsky, L. & Rosen, F.S. Bone marrow transplantation only partially restores purine metabolites to normal in adenosine deaminase-deficient patients. J. Clin. Invest. 68, 1387–1393 (1981).

    Article  CAS  Google Scholar 

  9. Kohn, D.B. et al. T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates. Nature Med. 4, 775–780 (1998).

    Article  CAS  Google Scholar 

  10. Arredondo, V.F., Santisteban, I., Daniels, S., Toutain, S. & Hershfield, M.S. Adenosine deaminase deficiency: Genotype-phenotype correlations based on expressed activity of 29 mutant alleles. Am J Hum Genet 63, 1049–1059 (1998).

    Article  Google Scholar 

  11. Bordignon, C. et al. Transfer of the ADA gene into bone marrow cells and peripheral blood lymphocytes for the treatment of patients affected by ADA-deficient SCID. Hum. Gene Ther. 4, 513–520 (1993).

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Ethics declarations

Competing interests

The authors declare no competing financial interests.

Rights and permissions

Reprints and permissions

About this article

Cite this article

Aiuti, A., Vai, S., Mortellaro, A. et al. Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement. Nat Med 8, 423–425 (2002). https://doi.org/10.1038/nm0502-423

Download citation

  • Issue Date:

  • DOI: https://doi.org/10.1038/nm0502-423

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