Preclinical Development of a Platform for Enzyme Therapy in the CNS of MPS I and MPS II Patients Based on Intrathecal AAV Delivery

Monolithic silica in MonoSpin for solid-phase extraction of drugs from whole blood samples was developed to facilitate high-throughput analysis. Monolithic silica of various pore sizes and octadecyl contents were synthesized, and their effects on recovery rates were evaluated. The silica monolith M18-200 (20 μm through-pore size, 10.4 nm mesopore size, and 17.3% carbon content) achieved the best recovery of the target analytes in whole blood samples. The extraction proceeded with centrifugal force at 1000 rpm for 2 min, and the eluate was directly injected into the liquid chromatography–mass spectrometry system without any tedious steps such as evaporation of extraction solvents. Under the optimized condition, low detection limits of 0.5–2.0 ng mL−1 and calibration ranges up to 1000 ng mL−1 were obtained. The recoveries of the target drugs in the whole blood were 76–108% with relative standard deviation of less than 14.3%. These results indicate that the developed method based on monolithic silica is convenient, highly efficient, and applicable for detecting drugs in whole blood samples.