| 发明名称 |
DISABLING AUTOPHAGY AS A TREATMENT FOR LYSOSOMAL STORAGE DISEASES |
| 摘要 |
Provided herein are methods of treating lysosomal storage disease, for instance Pompe disease, through inhibition of autophagy. Optionally, treatment is administered as an adjunct to enzyme replacement therapy (ERT). |
| 申请公布号 |
US2013331309(A1) |
申请公布日期 |
2013.12.12 |
| 申请号 |
US201313970265 |
申请日期 |
2013.08.19 |
| 申请人 |
THE U.S.A., AS REPRESENTED BY THE SECRETARY, DEPT. OF HEALTH AND HUMAN SERVICE |
发明人 |
RABEN NINA;XIE TAO;SCHREINER CYNTHIA;BAUM REBECCA;TAKIKITA SHOICHI;PLOTZ PAUL |
| 分类号 |
C12N15/113 |
主分类号 |
C12N15/113 |
| 代理机构 |
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代理人 |
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