Figure 3. Acid α-glucosidase (GAA) activity in HEK-293H cells expressing mutant forms of the enzyme treated with a pharmacological chaperone (NB-DNJ or DNJ) and a combination consisting of NB-DNJ/ABX and DNJ/ABX. Upper: PC-responsive mutations of GAA were treated with 40 μmol/l ABX,...
we tested if they would reveal altered functional responses to stress in the form of chloroquine treatment, glucose starvation, or glycogen phosphorylase inhibition applied for 24 h at day 13 of differentiation (Fig.3a). Chloroquine inhibits autophagosome-lysosome fusion and disrupts lysosomal membrane...
The final construct was named pMCK-hTFEB-3Xflag. The vectors were then packaged into AAV9 capsids and were produced by triple transfection of HEK293 cells followed by two rounds of CsCl2 purification according to published procedures. Vector titers were determined by DNA dot blot at ~1 ×...