Modified G6PC (glucose-6-phosphatase, catalytic subunit) nucleic acids and glucose-6-phosphatase-α (G6Pase-α) enzymes with increased phosphohydrolase activity are described. Also described are vectors, such as adeno-associated virus (AAV) vectors, and recombinant AAV expressing modified G6Pase-α. The disclosed AAV vectors and rAAV can be used for gene therapy applications in the treatment of glycogen storage disease, particularly glycogen storage disease type Ia (GSD-Ia), and complications thereof.

Disclosed herein are compositions and methods of enhancing stem cell differentiation into beta cells with use of one or more epigenetic modification compounds. The present disclosure also relates to compositions and methods of sorting and enriching the differentiated beta cells. The present disclosure also relates to compositions and methods of irradiating cell population for reducing proliferation.

A method of treating a disease or condition may be characterized by a metabolic disorder in a subject in need thereof, that can include administering to the subject a therapeutically effective amount of avocatin B.

A method of treating a disease or condition may be characterized by a metabolic disorder in a subject in need thereof, that can include administering to the subject a therapeutically effective amount of avocatin B.

A compound having a KHK inhibitory effect, or pharmaceutically acceptable salts thereof, and use thereof in the preparation of a medicament for treating a disease associated with KHK kinase abnormal expression. Provided is a compound as represented by formula (III) or a pharmaceutically acceptable salt thereof.

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A compound having a KHK inhibitory effect, or pharmaceutically acceptable salts thereof, and use thereof in the preparation of a medicament for treating a disease associated with KHK kinase abnormal expression. Provided is a compound as represented by formula (III) or a pharmaceutically acceptable salt thereof.

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Recombinant human alpha glucosidase (rhGAA) composition derived from CHO cells that contains a more optimized glycan composition consisting of a higher amount of rhGAA containing N-glycans carrying mannose-6-phosphate (M6P) or bis-M6P than conventional rhGAAs, along with low amount of non-phosphorylated high mannose glycans, and low amount of terminal galactose on complex oligosaccharides. Compositions containing the rhGAA, and methods of use are described.

Provided are novel human-derived antibodies specific for Fibroblast Activation Protein (FAP), preferably capable of selectively inhibiting the enzymatic activity of FAP, as well as methods related thereto. In addition, methods of diagnosing and/or monitoring diseases and treatments thereof which are associated with FAP are provided. Assays and kits related to antibodies specific for FAP are also disclosed. The novel anti-FAP antibodies can be used in pharmaceutical and diagnostic compositions for FAP-targeted immunotherapy and diagnostics.

The present disclosure relates to compositions comprising a non-viable fecal bacterial preparation for treating various gastrointestinal-related conditions and disorders. The present disclosure also relates to stool-based compositions comprising adjuvants to facilitate the efficacy of immunomodulators such as vaccines and immunotherapies.

Methods of inhibiting release of one or more cytokines, or inhibiting a cytokine storm, or preventing cytokine release syndrome or a cytokines storm, in a subject infected with severe acquired respiratory syndrome coronavirus 2. The method involves administering to the subject an effective amount of one or more NAD+ precursors, one or more inhibitors of stimulator of interferon genes (STING), one or more famesoid X receptor (FXR) agonists, or a combination thereof. In addition, treatment regimens involving the use of one or more NAD+ precursors, one or more STING inhibitors, one or more FXR agonists, or a combination thereof; and kits comprising pharmaceutical compositions of one or more NAD+ precursors, one or more inhibitors of stimulator of interferon genes, one or more farnesoid X receptor agonists, or combinations thereof.