Therapeutic Targeting of the GSK3β-CUGBP1 Pathway in Myotonic Dystrophy

Therapeutic Targeting of the GSK3β-CUGBP1 Pathway in Myotonic Dystrophy

Myotonic Dystrophy type 1 (DM1) is a neuromuscular disease associated with toxic RNA containing expanded CUG repeats. The developing therapeutic approaches to DM1 target mutant RNA or correct early toxic events downstream of the mutant RNA. We have previously described the benefits of the correction...

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Bibliographic Details
Main Authors: Maggie Lutz, Miranda Levanti, Rebekah Karns, Genevieve Gourdon, Diana Lindquist, Nikolai A. Timchenko, Lubov Timchenko
Format: Article
Language:English
Published: MDPI AG 2023-06-01
Series:International Journal of Molecular Sciences
Subjects:
Myotonic Dystrophy (DM1)
congenital Myotonic Dystrophy
development of therapy
myotonia
brain atrophy
GSK3β
Online Access:https://www.mdpi.com/1422-0067/24/13/10650

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https://www.mdpi.com/1422-0067/24/13/10650

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