ANDERSSON Claes,ANDERSSON Claes,FOGH Jens,FOGH Jens,WEIGELT Cecilia,WEIGELT Cecilia,ROCES Diego Prieto,ROCES Diego Prieto,VON FIGURA Kurt,VON FIGURA Kurt,IRANI Meher,IRANI Meher
申请号：
SI200532209
公开号：
SI1740204T1
申请日:
2005.04.01
申请国别(地区):
SI
年份:
2018
代理人:
摘要：
The present invention provides means and strategies for treating the lysosomal storage disorder alpha-mannosidosis by enzyme replacement therapy. In particular, the invention pertains to the reduction of stored neutral mannose-rich oligosaccharides in cells within the central nervous system. Accordingly, the present invention provides the use of a lysosomal alpha-mannosidase for the preparation of a medicament for reducing the intracellular levels of neutral mannose-rich oligosaccharides in cells within one or more regions of the central nervous system. The invention further provides a formulation of lysosomal alpha-mannosidase for use as a medicament for reducing the intracellular levels of neutral mannose-rich oligosaccharides in cells within one or more regions of the central nervous system. Also, the invention provides a method of reducing the intracellular levels of neutral mannose-rich oligosaccharides in cells within one or more regions of the central nervous system of a subject, said method comprising administering to said subject a preparation of lysosomal alpha-mannosidase. Finally, the invention pertains to methods of producing, isolating, and purifying an alpha-mannosidase as well as the use of an alpha-mannosidase, which is obtainable according to these methods.
