Rescue of a familial dysautonomia mouse model by AAV9-Exon-specific U1 snRNA
| Autor: | Romano, Giulia, Riccardi, Federico, Bussani, Erica, Vodret, Simone, Licastro, Danilo, Ragone, Isabella, Ronzitti, Giuseppe, Morini, Elisabetta, Slaugenhaupt, Susan A., Pagani, Franco |
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| Zdroj: | In The American Journal of Human Genetics 4 August 2022 109(8):1534-1548 |
| Databáze: | ScienceDirect |
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