Repeat-associated non-AUG translation in neuromuscular diseases: mechanisms and therapeutic insights

Expanded short tandem repeats cause more than 50 monogenic diseases, which are mostly neuromuscular diseases. In the non-coding repeat expansion diseases, in which the expanded repeat sequence is located outside of the coding region, the toxicity of the transcribed repeat-containing RNAs had been th...

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Veröffentlicht in:	Journal of biochemistry (Tokyo) 2023-04, Vol.173 (4), p.273-281
Hauptverfasser:	Fujino, Yuzo, Mori, Kohji, Nagai, Yoshitaka
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Neuromuscular Diseases - genetics Neuromuscular Diseases - therapy Open Reading Frames Peptides RNA
Online-Zugang:	Volltext
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