Article

Creation of de novo cryptic splicing for ALS/FTD precision medicine

2023-11-15

Abstract excerpt

A system enabling the expression of therapeutic proteins specifically in diseased cells would be transformative, providing greatly increased safety and the possibility of pre-emptive treatment. Here we describe “TDP-REG”, a precision medicine approach primarily for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), which exploits the cryptic splicing events that occur in cells with TDP-43 loss-...

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Literature Corpus work: 3b53c417-acb0-584d-a89f-f29f6c4e0453
DOI: 10.1101/2023.11.15.565967

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