M-31850 White solid. PROTECT FROM LIGHT. PACKAGED UNDER INERT GAS. A cell-permeable bisnaphthalimide compound that acts as a substrate-competitive and selective inhibitor against human b-N-acetyl hexosaminidase (IC50 = 6.0 & 3.1 µM for hHex A & hHex B, respectively), while exhibiting much reduced potency against Jack Bean or bacterial Hex (IC50 = 280 & >500 µM for JBHex & SpHex, respectively) and little or no activity against SmHex or hOGN. Shown to function as a pharmacological chaperone and effectively enhance the transport of mutant Hex alpha subunit from the ER to lysosome in cells derived from ISD and ATSD patients. Purity: ≥95% by HPLC. Ref.: Tropak, M.B., et al. 2007. Chem. Biol. 14, 153. |
