Flow Cytometry Assay To Monitor Effectiveness Of Gene Therapy Correction In Peripheral Blood Mononuclear Cells Of Gaucher Disease Type I Patients

The most common lysosomal storage disorder is Gaucher disease type I (GD-I) which is caused by a genetic defect in the gene encoding the enzyme beta-glucocerebrosidase (GCase). In GD-I, deficient GCase activity results in accumulation of glucocerebrosidases within macrophages of the reticuloendothelial system.