Time-controlled and muscle-specific CRISPR/Cas9-mediated deletion of CTG-repeat expansion in the DMPK gene
CRISPR/Cas9-mediated therapeutic gene editing is a promising technology for durable treatment of incurable monogenic diseases such as myotonic dystrophies. Gene-editing approaches have been recently applied to in vitro and in vivo models of myotonic dystrophy type 1 (DM1) to delete the pathogenic CT...
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Asıl Yazarlar: | , , , , , , , , , , , , , , , , |
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Materyal Türü: | Kitap |
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Elsevier,
2022-03-01T00:00:00Z.
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Yer Numarası: |
A1234.567 |
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Kopya Bilgisi 1 | Kütüphanede |