Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells
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|ClinicalTrials.gov Identifier: NCT00001234|
Recruitment Status : Completed
First Posted : November 4, 1999
Last Update Posted : March 4, 2008
National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by:
National Institutes of Health Clinical Center (CC)
No Study Results Posted on ClinicalTrials.gov for this Study
|Recruitment Status :||Completed|
|Primary Completion Date :||No date given|
|Study Completion Date :||April 2002|
Fink JK, Correll PH, Perry LK, Brady RO, Karlsson S. Correction of glucocerebrosidase deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from patients with Gaucher disease. Proc Natl Acad Sci U S A. 1990 Mar;87(6):2334-8.
Medin JA, Migita M, Pawliuk R, Jacobson S, Amiri M, Kluepfel-Stahl S, Brady RO, Humphries RK, Karlsson S. A bicistronic therapeutic retroviral vector enables sorting of transduced CD34+ cells and corrects the enzyme deficiency in cells from Gaucher patients. Blood. 1996 Mar 1;87(5):1754-62.