Patent classifications
C12Y204/01224
DEPLETION OF EXT1 EXPRESSION AND/OR ACTIVITY IMPROVES CELLULAR PRODUCTION OF BIOLOGICAL ENTITIES
The use of an inhibitor of EXT1 expression and/or activity for the production of a biological entity in a cell. Also, the use of a cell having at least depleted EXT1 expression and/or activity for the production of a biological entity. Further, evidence is provided about the role of glycosylation in rapid dynamism of ER shaping and function. In particular, depletion of EXT1 results in a recomposed ER shaping, which could benefit production of recombinant proteins.
METHODS OF TREATING NEURODEGENERATIVE DISEASE BY INHIBITING N-DEACETYLASE N-SULFOTRANSFERASE
A method of treating a subject suffering from a neurodegenerative disease characterized by insufficient autophagy is provided, the method comprising administering to the subject an effective amount of a composition that inhibits N-deacetylase N-sulfotransferase (NDST), Further provided is a method of identifying a modulator of autophagy.
HEPARIN SULFATE BIOSYNTHESIS PATHWAY ENZYME IRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a heparan sulfate biosynthesis pathway enzyme gene (HSBPE) gene, e.g., Exostosin Glycosyltransferase 1 (EXT1), Exostosin Glycosyltransferase 2 (EXT2), and/or N-Deacetylase And N-Sulfotransferase 2, (NDST2 gene), as well as methods of inhibiting expression of an HSBPE gene and methods of treating subjects having Mucopolysaccaridosis type III (MPS III), e.g., MPS IIIA, MPS IIIB, MPS IIIC, or MPS IIID, using such dsRNAi agents and compositions.
ANTICOAGULANT HEPARIN-CHONDROITIN CHIMERIC SACCHARIDE MOLECULE AS WELL AS PREPARATION METHOD AND APPLICATION THEREOF
An anticoagulant heparin-chondroitin chimeric saccharide molecule as well as a preparation method and application thereof are disclosed. The anticoagulant heparin-chondroitin chimeric saccharide molecule has a structure as shown in formula I. The heparin-chondroitin chimeric saccharide molecule of the present disclosure has potent activities against an Xa factor and IIa, and the activity of the heparin-chondroitin chimeric saccharide molecule can be effectively neutralized by protamine, with a neutralization rate of greater than or equal to 70%. The risk of causing adverse reactions such as fatal HIT is obviously lower than that of enoxaparin and other low-molecular-weight heparins. The heparin-chondroitin chimeric saccharide molecule disclosed by the present disclosure is suitable for the preparation of a safer potent anticoagulant and antithrombotic new drug.