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METHODES ET COMPOSITIONS POUR LE TRAITEMENT DE LA MALADIE DE POMPE
专利权人:
VALERION THERAPEUTICS; LLC
发明人:
ARMSTRONG, DUSTIN D.,WAY, JEFFREY C.
申请号:
CA2901978
公开号:
CA2901978A1
申请日:
2014.02.20
申请国别(地区):
CA
年份:
2014
代理人:
摘要:
Provided herein are chimeric polypeptides comprising a mature acid-alpha glucosidase (GAA) polypeptide and an internalizing moiety. An exemplary internalizing moiety of the application is the 3E10 antibody which is capable of transiting a cellular membrane via an equilibrative nucleoside transporter 2 (ENT2) and binding DNA. Further provided are methods and uses of the said chimeric polypetides for treating Pompe disease, increasing acid alpha-glucosidase enzyme activity in a cell and decreasing glycogen accumulation in the cytoplasm, lysosomes and autophagic vacuoles of cells.
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中国工程科技知识中心
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