Table 3.

Effect of IFG and NB-DNJ on GC levels in Gaucher patient-derived cells homozygous for the L444P mutation.

Cell ID	Cell Type	GC Levels
		Baseline	Compound/Regimen (% decrease)
		μg/mg protein	IFG ‘7 on/3 off’	IFG ‘10 on’	NB-DNJ ‘10 on’
GM07968	F	14±0.5	23±3^**	-	66±1^***
GM10915	F	2.5±0.1	32±4^**	-	76±1^***
GS0501	L	2.2±0.3	50±2^*	-	77±1^**
GS0505	L	2.7±0.2	26±1^*	-	75±1^***

GC levels in Gaucher fibroblasts and LCLs homozygous for L444P GCase were determined after 7-day incubation in the absence or presence of 30 μM IFG followed by a 3-day washout (‘7 on/3 off’). For comparison, cells were also incubated for 10 days (‘10 on’) with 30 μM IFG or 500 μM NB-DNJ. The data for each cell line have been normalized to the GC levels in untreated cells and are expressed as the mean±SEM from three flasks for each condition tested. Differences in GC levels between treated and untreated cells were determined using a two-tailed, unpaired student’s t-test (^*p<0.05, ^**p<0.01, ^***p<0.001). While incubation with 30 μM IFG for 10 days did not significantly reduce GC levels in any cell line tested, significant increases were seen in fibroblast cell lines 07968 and 10915 (16% and 35%, respectively; p<0.05 compared to untreated). GC levels in fibroblasts and LCLs derived from healthy volunteers (CRL2076 and WT0003, respectively) were 1.2±0.02 and 0.85±0.2 μg/mg protein, respectively (see Fig. 3). ‘F’, fibroblast; ‘L’, lymphoblastoid cell line; ‘-‘, no decrease.