These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.
Pubmed for Handhelds
PUBMED FOR HANDHELDS
Search MEDLINE/PubMed
Title: [Gene therapy for severe combined immunodeficiency]. Author: Petersen LB, Jensen TG. Journal: Ugeskr Laeger; 2010 Jun 07; 172(23):1756-8. PubMed ID: 20534204. Abstract: New results with gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency are promising. We review a clinical project in which ten children were treated with gene modified autologous haematopoietic stem cells. After treatment, eight patients were able to do without enzyme-replacement therapy, and nine patients showed improved immune function and sustained low concentration of toxic metabolites. No clonal outgrow was observed indicating a limited risk for future malignant development. Despite these promising results, the safety of gene therapy can still be improved.[Abstract] [Full Text] [Related] [New Search]