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AAV9-NPC1 significantly ameliorates Purkinje cell death and behavioral abnormalities in mouse NPC disease |
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| Institution: | 2. Institute of Biochemistry and Cell Biology, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai 200031, China |
| Abstract: | |
| Keywords: | cholesterol gene therapy Niemann-Pick type C disease lysosomal storage diseases adeno-associated virus serotype 9 Niemann-Pick type C1 blood-brain barrier |
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