Skip to main content

Advertisement

SpringerLink
Log in

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

  • Letters to the Editor
  • Published:

From Nature Medicine

View current issue Submit your manuscript

This is a preview of subscription content, log in via an institution to check access.

Access this article

Log in via an institution

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Institutional subscriptions

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

  1. San Raffaele Telethon Institute for Gene Therapy(HSR-TIGET), Milan, Italy

    Alessandro Aiuti, Sergio Vai, Alessandra Mortellaro, Francesca Ficara, Grazia Andolfi, Giuliana Ferrari, Maria Grazia Roncarolo & Claudio Bordignon

  2. Cancer Immunotherapy and Gene Therapy Program, Scientific Institute H.S. Raffaele, Milan, Italy

    Giulia Casorati

  3. Istituto di Biochimica ed Enzimologia, Università di Siena, Siena, Italy

    Antonella Tabucchi & Filippo Carlucci

  4. Department of Pediatrics, University of Washington School of Medicine, Seattle, Washington, USA

    Hans D. Ochs

  5. Clinica Pediatrica Brescia, Brescia, Italy

    Luigi D. Notarangelo

  6. Università Vita-Salute San Raffaele, Milan, Italy

    Claudio Bordignon

Authors
  1. Alessandro Aiuti
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Sergio Vai
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. Alessandra Mortellaro
    View author publications

    You can also search for this author in PubMed Google Scholar

  4. Giulia Casorati
    View author publications

    You can also search for this author in PubMed Google Scholar

  5. Francesca Ficara
    View author publications

    You can also search for this author in PubMed Google Scholar

  6. Grazia Andolfi
    View author publications

    You can also search for this author in PubMed Google Scholar

  7. Giuliana Ferrari
    View author publications

    You can also search for this author in PubMed Google Scholar

  8. Antonella Tabucchi
    View author publications

    You can also search for this author in PubMed Google Scholar

  9. Filippo Carlucci
    View author publications

    You can also search for this author in PubMed Google Scholar

  10. Hans D. Ochs
    View author publications

    You can also search for this author in PubMed Google Scholar

  11. Luigi D. Notarangelo
    View author publications

    You can also search for this author in PubMed Google Scholar

  12. Maria Grazia Roncarolo
    View author publications

    You can also search for this author in PubMed Google Scholar

  13. Claudio Bordignon
    View author publications

    You can also search for this author in PubMed Google Scholar

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

  • Springer Nature America, Inc.

This article is cited by

Search

Navigation