Abstract:
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
Abstract:
The invention relates to novel polyalkylene glycol compounds and methods of using them. In particular, compounds comprising a novel polyethylene glycol conjugate are used alone, or in combination with antiviral agents to treat a viral infection, such as chronic hepatitis C.
Abstract:
The present invention relates to improved methods in the separation recombinant polypeptides with post-translational modifications from complex mixtures through the use of a cation exchange medium.
Abstract:
In some embodiments, aspects of the disclosure relate to methods of evaluating cell culture materials, for example, nutrient materials, or other materials that can be used in cell culture media.
Abstract:
A controlled release dosage form containing monomethyl fumarate, a compound that can be metabolized into monomethyl fumarate in vivo, or a pharmaceutically acceptable salt thereof or combinations thereof, wherein the monomethyl fumarate, a compound that can be metabolized into monomethyl fumarate in vivo, or a pharmaceutically acceptable salt thereof or combinations thereof is delivered to the subject. Also provided is a method of treating a disease or disorder (e.g., multiple sclerosis) by orally administering a controlled release dosage form containing monomethyl fumarate, a compound that can be metabolized into monomethyl fumarate in vivo, or a pharmaceutically acceptable salt thereof or combinations thereof, wherein the monomethyl fumarate, a compound that can be metabolized into monomethyl fumarate in vivo, or a pharmaceutically acceptable salt thereof or combinations thereof.
Abstract:
The present invention pertains to a cell culture medium comprising media supplements that are shown to control recombinant protein glycosylation and/or cell culture in a controlled or modulated (shifted) temperature to control recombinant protein glycosylation and/or cell culture with controlled or modulated seed density to control recombinant protein glycosylation, and methods of using thereof. The present invention further pertains to a method of controlling or manipulating glycosylation of a recombinant protein of interest in a large scale cell culture.