Thursday, July 17, 2014
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Macrophage model of Gaucher's
A macrophage model of
Gaucher's disease could help identify new therapies to treat the condition,
which is caused by a genetic deficiency in glucocerebrosidase
Induced pluripotent stem (iPS) cells and monocytes derived from patients with
Gaucher's disease or healthy subjects were used to generate macrophages.
Patient-derived macrophages showed impaired bacteria-induced reactive oxygen
species production, decreased GBA activity and increased lysosomal storage of
GBA substrates compared with macrophages generated from healthy subjects. In
the patient-derived macrophages, a chaperone protein that restores mutant GBA
folding increased GBA activity with potency comparable to that of recombinant
GBA. Next steps could include developing screening assays that use the
macrophages to identify new therapeutic candidates.
Published online July 17, 2014
Patent and licensing status
Aflaki, E. et al. Sci.
Transl. Med.; published online June 11, 2014;
Contact: Ellen Sidransky, National Institutes of Health, Bethesda,
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