A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice
| Autor: | Sangster, Madison L., Bishop, Martha M., Yao, Yizheng, Feitor, Jessica F., Shahriar, Sanjid, Miller, Maxwell E., Chekuri, Anil K., Budnik, Bogdan, Bei, Fengfeng, Grishchuk, Yulia |
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| Zdroj: | In Molecular Therapy - Methods & Clinical Development 13 June 2024 32(2) |
| Databáze: | ScienceDirect |
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