Preclinical results in rodents strongly support clinical evaluation of scAAV9/MFSD8 as a potential gene therapy for CLN7 patients
| Autor: | Chen, Xin, Shaffo, Frances, Dong, Thomas, Hu, Yuhui, Belur, Nandkishore R., Mazzulli, Joseph R., Gray, Steven J. |
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| Zdroj: | In Molecular Genetics and Metabolism February 2021 132(2):S24-S24 |
| Databáze: | ScienceDirect |
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