Systemic Evaluation of Chimeric LNA/2′-O-Methyl Steric Blockers for Myotonic Dystrophy Type 1 Therapy

Myotonic dystrophy type 1 (DM1) is a dominantly inherited, multisystemic disorder characterized clinically by delayed muscle relaxation and weakness. The disease is caused by a CTG repeat expansion in the 3′ untranslated region (3′ UTR) of the DMPK gene, which leads to the expression of a toxic gain...

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Detalles Bibliográficos
Autores principales: Christou, Melina, Wengel, Jesper, Sokratous, Kleitos, Kyriacou, Kyriacos, Nikolaou, Georgios, Phylactou, Leonidas A., Mastroyiannopoulos, Nikolaos P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2020
Materias:
Original Research Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7133450/
https://www.ncbi.nlm.nih.gov/pubmed/31873063
http://dx.doi.org/10.1089/nat.2019.0811

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7133450/
https://www.ncbi.nlm.nih.gov/pubmed/31873063
http://dx.doi.org/10.1089/nat.2019.0811

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