In vivo targeting of a variant causing vanishing white matter using CRISPR/Cas9
| Autor: | Hillen, Anne E.J., Hruzova, Martina, Rothgangl, Tanja, Breur, Marjolein, Bugiani, Marianna, van der Knaap, Marjo S., Schwank, Gerald, Heine, Vivi M. |
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| Zdroj: | In Molecular Therapy - Methods & Clinical Development 9 June 2022 25:17-25 |
| Databáze: | ScienceDirect |
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