Recombinant AAV (rAAV) vectors comprising a rAVV genome comprising a heterologous nucleic acid encoding a lysosomal protein, e.g., acid alpha-glucosidase (GAA) polypeptide, and optionally a signal peptide and/or optionally a targeting sequence, e.g., IGF2 targeting peptide, operatively linked to a liver-specific promoter (LSP), enabling the GAA polypeptide to be secreted from the liver and targeted to the lysosomes. Particular embodiments relate to a recombinant AAV (rAAV) vector encoding an alpha-glucosidase (GAA) polypeptide, having a liver secretory signal peptide and a IGF2 targeting peptide that binds human cation-independent mannose-6-phosphate receptor (CI-MPR) or to the IGF2 receptor, permitting proper subcellular localization of the GAA polypeptide to lysosomes. Also encompassed are cells, and methods to treat a lysosomal disease, for example, a glycogen storage disease type II (GSD II) disease and/or Pompe Disease with the rAAV vector.

The invention provides compositions and methods for effective lysosomal targeting mediated by PCSK9. In particular, the compositions and methods provided by the invention may be used to treat lysosomal storage diseases such as Pompe Disease and Sanfilippo Syndrome Type B, and they may be used for targeting lysosomal enzymes to the various muscles of the human body.

Genetically programmed microorganisms, such as bacteria or virus, pharmaceutical compositions thereof, and methods of modulating and treating cancers are disclosed.

The present disclosure generally relates to methods and compositions comprising a protein having hyaluronidase activity to diagnose the etiology of, minimize, and treat peri-orbital hollowness with or without associated thyroid disease and to treat tear trough deformity.

Methods for the continuous production, capturing and purification of biologics such as recombinant proteins are described. Also described are pharmaceutical compositions comprising such biologics, as well as methods of treatment and uses of such biologics.

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, e.g., anti-TfRCscfv:GAA, that crosses the blood brain barrier (BBB) are provided.

A process of stabilizing the activity of an enzyme against inactivation by water weathering are provided including associating one or more polymeric moieties of a polyoxyethylene having a molecular weight of 10,000 Daltons or greater with an enzyme to form a chemically modified enzyme; and dispersing said chemically modified enzyme in a base to form a water-stabilized active coating material.

Bioactive coatings suitable for facilitating removal of a fingerprint when contacting the coating are provided including a base associated with a chemically modified enzyme, and, optionally a first polyoxyethylene present in the base and independent of the enzyme. The coatings are optionally overlayered onto a substrate. Also provided are processes of facilitating fingerprint removal.

A method for extracting functional ingredients of mulberry leaves using enzyme is provided. A method for preparing crude cellulase enzyme solution and crude pectinase enzyme solution includes: preparing PDA slant culture mediums; preparing fermentation mediums for producing cellulase and pectinase; preparing solid bacteria; fermentation; preparing crude cellulase enzyme solution and crude pectinase enzyme solution. A preparation method of mulberry leaf concentrate is provided and includes: enzymatic hydrolysis, enzyme inactivation and concentration. A method for preparing mulberry leaf products from mulberry leaf concentrate is provided. Cellulase and pectinase prepared by the stock culture of agaric are safer and more reliable; the content and yield of protein and other functional ingredients in mulberry leaves are improved; the mulberry leaf product can be directly applied to processing and production of food, health care products and cosmetics which improves comprehensive utilization rate of mulberry leaf raw materials.

Provided are pharmaceutical formulations comprising a recombinant acid α-glucosidase, wherein the recombinant acid α-glucosidase is expressed in Chinese hamster ovary (CHO) cells and comprises an increased content of N-glycan units bearing one or two mannose-6-phosphate residues when compared to a content of N-glycan units bearing one or two mannose-6-phosphate residues of alglucosidase alfa; at least one buffer selected from the group consisting of a citrate, a phosphate and combinations thereof; and at least one excipient selected from the group consisting of mannitol, polysorbate 80, and combinations thereof, wherein the formulation has a pH of from about 5.0 to about 7.0. Also provided are methods of treating Pompe disease using these pharmaceutical formulations.