Immune response to enzyme replacement therapy in lysosomal storage disorder patients and animal models

DA Brooks - Molecular genetics and metabolism, 1999 - Elsevier
The lysosomal storage disorders (LSD) are a group of severe multiple pathology disorders
characterized by enzyme deficiencies which cause the lysosomal accumulation of
undegraded or partially degraded macromolecules. Enzyme replacement therapy (ERT) has
been developed as a therapy for LSD patients. However, immune responses to ERT have
been reported in some individuals from LSD animal model and LSD human patient studies.
Antibodies can have adverse effects during ERT, which include hypersensitivity …