Regeneron Pharmaceuticals, Inc.
METHODS AND COMPOSITIONS FOR THERAPEUTIC PROTEIN DELIVERY

Abstract:

Compositions and methods for delivering a therapeutic protein to the central nervous system (CNS), in order to treat diseases and disorders that impair the CNS, such as treating lysosomal storage diseases are disclosed. Therapeutic proteins delivered via a therapeutically effective amount of a nucleotide composition encoding the therapeutic protein conjugated to a cell surface receptor-binding protein that crosses the blood brain barrier (BBB) are provided.