Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof

摘要

The present invention relates generally to therapeutic fusion proteins useful for treating lysosomal storage disorders and methods of treating such disorders. Exemplary therapeutic fusion proteins include lysosomal enzymes, lysosomal targeting moieties such as IGF-II peptides, and spacer peptides. Also compositions comprising a targeted therapeutic fusion protein comprising alpha-N-acetylglucosaminidase (Naglu), a lysosomal targeting moiety, such as an IGF-II peptide, and a spacer peptide, and mycopolysaccharidosis type IIIB ( Methods of treating San Filippo B syndrome) are provided.