METHODS AND COMPOSITIONS FOR INCREASING N-ACETYLGLUCOSAMINIDASE ACTIVITY IN THE CNS

摘要

Provided herein are methods and compositions for treating a subject suffering from an enzyme deficiency in the central nervous system (CNS). The bifunctional fusion antibody provided herein comprise an antibody to an endogenous blood brain barrier (BBB) receptor and an enzyme deficient in mucopolysaccharidosis IIIB (MPS-IIIB). The fusion antibodies provided herein comprise alpha-N-acetylgulcosaminidase (NAGLU). The methods of treating an enzyme deficiency in the CNS comprise systemic administration of a fusion antibody provided herein.

主权项

1. A method for treating an alpha-N-acetylgulcosaminidase (NAGLU) deficiency in the central nervous system of a subject in need thereof, comprising systemically administering to the subject a therapeutically effective dose of a fusion antibody having NAGLU activity, wherein the fusion antibody comprises: (a) a fusion protein comprising the amino acid sequences of an immunoglobulin heavy chain and a NAGLU, and (b) an immunoglobulin light chain; wherein the fusion antibody crosses the blood brain barrier (BBB).