Validation of high-sensitivity assays to quantitate cerebrospinal fluid and serum β-galactosidase activity in patients with GM1-gangliosidosis
| Autor: | Quadrini, Karen J., Vrentas, Catherine, Duke, Christian, Wilson, Chris, Hinderer, Christian J., Weinstein, David A., Al-Zaidy, Samiah A., Browne, Susan E., Wilson, James M., Ni, Yan G. |
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| Zdroj: | In Molecular Therapy - Methods & Clinical Development 12 September 2024 32(3) |
| Databáze: | ScienceDirect |
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