Muscle-directed AAV gene therapy rescues the maple syrup urine disease phenotype in a mouse model
| Autor: | Greig, Jenny A., Jennis, Matthew, Dandekar, Aditya, Chorazeczewski, Joanna K., Smith, Melanie K., Ashley, Scott N., Yan, Hanying, Wilson, James M. |
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| Zdroj: | In Molecular Genetics and Metabolism September-October 2021 134(1-2):139-146 |
| Databáze: | ScienceDirect |
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