| 标题 |
 Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration |
| 网址 | |
| DOI | |
| 其它 |
期刊：Human Gene Therapy 作者：Wenfei Liu; Amy F. Geard; Giulia Massaro; Michael P. Hughes; Mikel Aristorena; et al 出版日期：2026-02-08 |
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