Article

ASO-enhancement of <i>TARDBP</i> exitron splicing mitigates TDP-43 proteinopathies

2024-07-22

Abstract excerpt

Amyotrophic lateral sclerosis and frontotemporal lobar degeneration are fatal neurodegenerative diseases characterized by pathological aggregation and nuclear functional loss of TDP-43 1,2 . Current therapies inadequately address this core pathology 3,4 , necessitating innovative approaches that target aggregation while preserving TDP-43’s essential functions. Here we demonstrate that enhancing the splicing of t...

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Literature Corpus work: 6024ce9b-4398-5084-a8ed-17807c4027f5
DOI: 10.1101/2024.07.22.604579

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