1 / 7

T-Lymphocyte Gene Transfer for a Patient with ADA-SCID

T-Lymphocyte Gene Transfer for a Patient with ADA-SCID. Onodera, M., T. Ariga, N. Kawamura, I. Koboyashi, M. Ohtsu, M. Yamada, A. Tame, H. Furuta, M. Okano, S. Matsumoto, H. Kotani, G.J. McGarrity, R. M. Blaese, and Y. Sakiyama. 1998. . What is ADA-SCID?. ADA is an enzyme

wilford
Télécharger la présentation

T-Lymphocyte Gene Transfer for a Patient with ADA-SCID

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript

  1. T-Lymphocyte Gene Transfer for a Patient with ADA-SCID Onodera, M., T. Ariga, N. Kawamura, I. Koboyashi, M. Ohtsu, M. Yamada, A. Tame, H. Furuta, M. Okano, S. Matsumoto, H. Kotani, G.J. McGarrity, R. M. Blaese, and Y. Sakiyama. 1998.

  2. What is ADA-SCID? • ADA is an enzyme • Deficiency impairs immunity responses • Resulting in SCID (Severe Combined Immunodeficiency) • Lack of enzyme of ADA coded for by a gene on chromosome 20 • Causes B and T lymphocytes to function improperly

  3. Symptoms of ADA-SCID • Productive cough and Nasal Discharge • Lymphopenia • Very little mature T and B lymphocytes • Low Ig serum levels • Undetectable isohemagglutinins • No T-cell response to phytohemagglutinins, Concanavalin A, and pokeweed mitogen.

  4. Mutations responsible for ADA deficiency • G632 to A transition (Arginine replaced by Histidine) • RsaI digestion same as father Paternal missense mutation(1c)

  5. Clinical Course After Gene Therapy • 10 infusions and patient had an increase in lymphocyte numbers. Eventually returned to basal levels • PBL count remained in a normal range • Increase in CD8+ count • ADA enzyme activity increased • Immune functions improved • Ig levels increased

  6. Conclusion • ADA-SCID patients had positive outcomes with the genetic therapy • Genetic therapy has established a new form of treatment for people with inherited diseases and acquired diseases

More Related