"Transfer of the Human Glucocerebrosidase (hGC) Gene Into Hematopoietic Stem Cells of Nonablated Mice."

According to an abstract submitted by the authors to the 36th Annual Meeting of the American Society of Hematology, held December 2-6, 1994, in Nashville, Tennessee, "Gaucher's disease (glucocerebrosidase [GC] deficiency) is a candidate for gene therapy since allogeneic bone marrow (BM) transplantation has been used successfully to treat this disorder. Maximum engraftment currently requires ablation of the endogenous bone marrow which, in humans, has significant morbidity. Previous reports have demonstrated that repeated injection of fresh BM can achieve up to 46% long-term engraftment. We investigated the possibility of gene transfer of hGC cDNA without BM ablation. Donor BM from...

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