First in-human, intracisternal dosing of RGX-111, an investigational AAV gene therapy, for a 21-month-old child with mucopolysaccharidosis type I (MPS I): 3.5 year follow-up
| Autor: | Wang, Raymond Y., Movsesyan, Nina, Kan, Shih-hsin, Beydoun, Tammam, Taylor, Mery, Chang, Richard C., Phillips, Dawn, Burke, Jenna, Gilmor, Michelle, Cho, Yoonjin, Falabella, Paulo, Pisani, Laura |
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| Zdroj: | In Molecular Genetics and Metabolism February 2024 141(2) |
| Databáze: | ScienceDirect |
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