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Specific DMPK-promoter targeting by CRISPRi reverses myotonic dystrophy type 1-associated defects in patient muscle cells

Document Type

Article

Author

Porquet, F.; Weidong, L.; Gazon, H.; Willems, L.; Jehasse, K.; Massotte, L.; Seutin, V.; Kondili, M.; Furling, D.; Klein, A.F.; Di Valentin, E.; Blacher, S.; Gillet, N.A.

Source

In: Molecular Therapy Nucleic Acids. (Molecular Therapy Nucleic Acids, 13 June 2023, 32:857-871)

Subject

Language

English

ISSN

21622531

Online Access

Full Text (ScienceDirect 종량제) Full Text (ScienceDirect O/A) Open Access (EBSCO) Web of Science JCR 저널정보 Scopus Find it@PNU

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