Figure 4. Prenylation activity in fibroblasts (CPF1) (i) and iPSCs (CPS1, CPS2) (ii) cultured from CHM individuals is restored following infection with AAV2.

hCHM. Prenylation assay was performed using the cytosolic fraction of cells transduced with AAV2. hCHM and from untreated affected cells (Control). Cell lysates were incubated with RabGGTase, RAB27 and [3H]-labeled GGPP. A significant increase (P<0.02) in the prenylation activity of exogenous REP-1 was observed in both CPF1 (2 fold) and CPS1, CPS2 cells (∼3 fold).