Novel Methods for Addressing Immunogenicity of Therapeutic Enzymes

摘要: While the biotechnology revolution has contributed to development of life-saving replacement proteins for blood factor and enzyme such as hemophilia Pompe disease, these therapies can induce immune responses leading treatment failure. The degree response is dependent on whether patient established tolerance missing protein. For example, patients that do not produce any natural lysosomal acid alpha-glucosidase (GAA) are categorized cross-reactive immunologic material (CRIM)-negative, those with partial GAA protein expression, CRIM-positive. interdependent relationship between incidence ADA CRIM-status in leads following dilemma: less expressed, more severe greater dependence protein, but risk severity ADA. Thus, spite having an effective there remains a critical unmet medical need immunogenic GAA, especially CRIM-negative babies. New methods inducing have been developed recent years, which reviewed here. They include drug-induced tolerance, deimmunization (removal T cell epitope triggers response) antigen-specific induction using Tregitope, novel regulatory T-cell intervention.