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T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates.

Publication ,  Journal Article
Kohn, DB; Hershfield, MS; Carbonaro, D; Shigeoka, A; Brooks, J; Smogorzewska, EM; Barsky, LW; Chan, R; Burotto, F; Annett, G; Nolta, JA ...
Published in: Nat Med
July 1998

Adenosine deaminase-deficient severe combined immunodeficiency was the first disease investigated for gene therapy because of a postulated production or survival advantage for gene-corrected T lymphocytes, which may overcome inefficient gene transfer. Four years after three newborns with this disease were given infusions of transduced autologous umbilical cord blood CD34+ cells, the frequency of gene-containing T lymphocytes has risen to 1-10%, whereas the frequencies of other hematopoietic and lymphoid cells containing the gene remain at 0.01-0.1%. Cessation of polyethylene glycol-conjugated adenosine deaminase enzyme replacement in one subject led to a decline in immune function, despite the persistence of gene-containing T lymphocytes. Thus, despite the long-term engraftment of transduced stem cells and selective accumulation of gene-containing T lymphocytes, improved gene transfer and expression will be needed to attain a therapeutic effect.

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Published In

Nat Med

DOI

ISSN

1078-8956

Publication Date

July 1998

Volume

4

Issue

7

Start / End Page

775 / 780

Location

United States

Related Subject Headings

  • Umbilical Cord
  • Transplantation, Autologous
  • Transplantation Immunology
  • Transformation, Genetic
  • T-Lymphocytes
  • Polyethylene Glycols
  • Mice, SCID
  • Mice
  • Lymphocyte Count
  • Leukocytes, Mononuclear
 

Citation

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Kohn, D. B., Hershfield, M. S., Carbonaro, D., Shigeoka, A., Brooks, J., Smogorzewska, E. M., … Parkman, R. (1998). T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates. Nat Med, 4(7), 775–780. https://doi.org/10.1038/nm0798-775
Kohn, D. B., M. S. Hershfield, D. Carbonaro, A. Shigeoka, J. Brooks, E. M. Smogorzewska, L. W. Barsky, 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.Nat Med 4, no. 7 (July 1998): 775–80. https://doi.org/10.1038/nm0798-775.
Kohn DB, Hershfield MS, Carbonaro D, Shigeoka A, Brooks J, Smogorzewska EM, 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. Nat Med. 1998 Jul;4(7):775–80.
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.Nat Med, vol. 4, no. 7, July 1998, pp. 775–80. Pubmed, doi:10.1038/nm0798-775.
Kohn DB, Hershfield MS, Carbonaro D, Shigeoka A, Brooks J, Smogorzewska EM, Barsky LW, Chan R, Burotto F, Annett G, Nolta JA, Crooks G, Kapoor N, Elder M, Wara D, Bowen T, Madsen E, Snyder FF, Bastian J, Muul L, Blaese RM, Weinberg K, Parkman R. T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates. Nat Med. 1998 Jul;4(7):775–780.

Published In

Nat Med

DOI

ISSN

1078-8956

Publication Date

July 1998

Volume

4

Issue

7

Start / End Page

775 / 780

Location

United States

Related Subject Headings

  • Umbilical Cord
  • Transplantation, Autologous
  • Transplantation Immunology
  • Transformation, Genetic
  • T-Lymphocytes
  • Polyethylene Glycols
  • Mice, SCID
  • Mice
  • Lymphocyte Count
  • Leukocytes, Mononuclear