CellPro Inc. announced Thursday that the Recombinant DNAAdvisory Committee (RAC) of the National Institutes of Health(NIH) has approved two gene therapy protocols that use thecompany's Ceprate stem cell concentration system for treatingGaucher's disease.
The two separate studies will be conducted by Stefan Karlssonand Cynthia Dunbar of the NIH, Donald Kohn of ChildrensHospital Los Angeles and John Barranger of the University ofPittsburgh School of Medicine.
In preclinical studies, it has been demonstrated that the genethat produces glucocerebrosidase, the enzyme absent inindividuals who suffer from Gaucher's disease, can besuccessfully inserted into stem cells. The clinical investigatorswill use CellPro's device to isolate stem cells from an individualpatient, after which they will transfect the stem cells with thegene and then return them to that patient.
"These trials demonstrate the increasing role of stem cells asvehicles for the delivery and functional integration of missinggenes in inherited diseases or genes with therapeuticpotential," commented Ronald Berenson, vice president ofbiological research and medical affairs at the Bothell, Wash.,company (NASDAQ:CPRO).
-- Jennifer Van Brunt Senior Editor
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