COMPOSITIONS AND METHODS FOR INTERNALIZING ENZYMES

Abstract: compositions and methods for treating enzyme-deficiency diseases are disclosed. multidomain therapeutic proteins containing an internalization effector binding domain and a lysosomal replacement enzyme activity are disclosed. the multidomain therapeutic proteins are capable of entering cells, segregating to the lysosome, and delivering the replacement enzyme activity to the lysosome.

Inventor(s): Andrew Baik, Katherine Cygnar, Christopher Schoenherr, Christos Kyratsous, Cheng Wang

CPC Classification: C07K14/70596 (PEPTIDES (peptides in foodstuffs ; obtaining protein compositions for foodstuffs, working-up proteins for foodstuffs ; preparations for medicinal purposes ; peptides containing beta-lactam rings ; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, ; ergot alkaloids of the cyclic peptide type ; macromolecular compounds having statistically distributed amino acid units in their molecules, i.e. when the preparation does not provide for a specific; but for a random sequence of the amino acid units, homopolyamides and block copolyamides derived from amino acids ; macromolecular products derived from proteins ; preparation of glue or gelatine ; single cell proteins, enzymes ; genetic engineering processes for obtaining peptides ; compositions for measuring or testing processes involving enzymes ; investigation or analysis of biological material ))

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