ABSTRACT

Enzymes have tremendous potential to serve as highly efficacious drugs with limited off-target effects in a plethora of disease conditions, including ischemia, bleeding, thrombosis, inflammatory disorders, cancer, and lysosomal storage disorders (LSDs), among others. Compared to traditional small-molecule drugs, these biotherapeutics influence their molecular targets with exceptional specificity and with high potency and efficacy due to their catalytic nature. Enzymes, however, are costly, labile, often have unfavorable pharmacokinetics (PK) and usually require delivery to specific compartments in the body, sometimes on a subcellular level, to be efficacious. For these reasons, nanocarriers (NCs) are being explored as enzyme drug delivery vehicles.