Retroviral-Mediated Transfer and Expression of Glucocerebrosidase and Ceramidtrihexosidase (a-Galactosidase A) cDNA's in Human Hematopoietic Progenitor Cells
This protocol was developed in order to obtain bone marrow stem cells for ex vivo
transduction with retroviruses containing the human glucocerebrosidase gene. We continue to
enter a small number of patients to this protocol each year. Studies with the bone marrow
hematopoietic progenitor cells have enabled us to identify the most effective retroviral
construct currently available in order to carry out gene therapy trials in patients with
Gaucher's disease. The data revealed that a comparatively simple retroviral construct
containing human glucocerebrosidase cDNA driven by the MoLV promoter is highly effective.
We have obtained approval and initiated a Phase I safety and gene marking investigation in
patients with Type I Gaucher's Disease.
Endpoint Classification: Safety Study, Primary Purpose: Treatment
United States: Federal Government
|National Institute of Neurological Disorders and Stroke (NINDS)||Bethesda, Maryland 20892|