Intravenous delivery of AAV9-GLB1 gene therapy for GM1 gangliosidosis: An interim analysis
| Autor: | D’Souza, Precilla, Johnston, Jean M., Thurm, Audrey, Farmer, Cristan, Gross, Amanda L., Acosta, Maria T., Tifft, Cynthia |
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| Zdroj: | In Molecular Genetics and Metabolism February 2024 141(2) |
| Databáze: | ScienceDirect |
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