CLINICAL TRIAL OF GENE THERAPY OF GAUCHER DISEASE

$3,034M01FY2000RRNIH

University Of Pittsburgh At Pittsburgh, Pittsburgh PA

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Abstract

The hypothesis of this study is that genetically corrected peripheral blood stem cells (PBSC) will result in a sustained reversal of the phenotype in patients with Gaucher disease. Specific aims to be achieved are the transfer of the human GC gene into PBSC obtained from patients with Gaucher disease, the transplantation of transduced PBSC autogously to patients and the measurement of carriage and expression of transferred gene and its duration in peripheral blood luekocytes (PBL) and the assessment of clinical effects of transplanting genetically corrected PBSC in patients with Gaucher disease.

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