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专利权人:
发明人:
RAMASWAMI MANI
申请号:
IN1524/DELNP/2006
公开号:
IN2006DN01524A
申请日:
2006.03.21
申请国别(地区):
IN
年份:
2012
代理人:
摘要:
The invention relates to various methods of treating lysosomal storage disorders using somatic cells and methods of delivering therapeutic enzymes. More particularly, gain-of-function or loss-of-function mutations in components of the intracellular Golgi to lysosome sorting pathway are used to enhance secretion of one or more lysosomal enzymes in somatic cells, thereby providing treatment for lysosomal storage disorders, particularly in neuronal cells. in addition, homoiogous recombination may be used to engineer therapeutically useful dells, for example, somatic cells, such as, glial progenitor cells, mesenchymal stem cells and astrocyte precursor cells, to enhance secretion of one or more Iysosomal enzymes.
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