T Lymphocyte-Directed Gene Therapy for ADA− SCID: Initial Trial Results After 4 Years

@article{Blaese1995TLG,
  title={T Lymphocyte-Directed Gene Therapy for ADA− SCID: Initial Trial Results After 4 Years},
  author={R. Michael Blaese and Kenneth W. Culver and A Dusty Miller and Charles S. Carter and Thomas A. Fleisher and Mario Clerici and Gene M. Shearer and Lauren Chang and Yawen L. Chiang and Paul Tolstoshev and Jay J. Greenblatt and Steven A. Rosenberg and H Klein and Mel Berger and Craig A. Mullen and W. J. Ramsey and Linda Mesler Muul and Richard A. Morgan and W. French Anderson},
  journal={Science},
  year={1995},
  volume={270},
  pages={475 - 480}
}
In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase (ADA) gene into the T cells of two children with severe combined immunodeficiency (ADA− SCID). The number of blood T cells normalized as did many cellular and humoral immune responses. Gene treatment ended after 2 years, but integrated vector and ADA gene expression in T cells persisted. Although many components remain to be perfected, it is concluded here that gene therapy can be a safe and… Expand

Paper Mentions

Interventional Clinical Trial
Primary Objective: - To determine if there is significant toxicity associated with the administration of CD34-TK75 transduced donor lymphocytes after allogeneic BMT for… Expand
ConditionsHodgkin Disease, Leukemia, Lymphoma, Non-Hodgkin, (+2 more)
InterventionGenetic, Radiation
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The developments achieved in over two decades of clinical and laboratory research that led to the establishment of a protocol for the autologous transplant of retroviral vector-mediated gene-modified hematopoietic stem cells are discussed. Expand
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This chapter reviews several crucial problems inherent in the current retroviral technology based on the clinical data observed in these pioneering ADA gene therapy trials and presents the new Retroviral vector system for the next stem cell gene therapy. Expand
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Stem cell gene therapy combined with appropriate conditioning regimens might be extended to other genetic disorders of the hematopoietic system. Expand
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TLDR
The results obtained in this trial are in agreement with previously published observations and support the usefulness of T lymphocyte-directed gene transfer in the treatment of ADA-SCID. Expand
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TLDR
This review reflects the progresses made in specific disorders, including adenosine deaminase deficiency, X-linked severe combined immunodeficiency, chronic granulomatous disease, and Wiskott-Aldrich syndrome. Expand
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In comparison with SCID-X1, ADA-SCID gene therapy presents a better safety profile and engraftment of multilineage transduced stem/progenitor cells, thanks to the use of nonmyeloablative preconditioning. Expand
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