| 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 |
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| DOI | |
| 其它 |
期刊:Hum Gene Ther 作者:Liu W, Geard AF, Massaro G, Hughes MP, Aristorena M, Coombe-Tennant O, Xu L, Semenyuk O, Bush R, Te Vruchte D, Priestman D, Laban R, Veleva E, Heslegrave AJ, Zetterberg H, Platt FM, Smith AJ, Mole SE, Ali RR, Rahim AA 出版日期:2026-02-08 |
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| 下载 |
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(2025-6-4)