Work

From cryptic exons to neuronal dysfunction: combinatorial antisense makes sense in TAR DNA-binding protein-43 (TDP-43) pathology

Year: 2026

Type: article

Abstract: In a recent study published in Science Translational Medicine , Guo et al. 1 shed further light on TDP-43 pathology by demonstrating that several transcripts involved in synaptic function and membrane excitability are aberrantly spliced upon depletion of TDP-43. Simultaneous treatment with antisense oligonucleotides targeting these transcripts corrected the splicing abnormalities and restored neuronal function, suggesting that combination therapy could be a promising approach in TDP-43 proteinopathies. 1

Source: Signal Transduction and Targeted Therapy

DOI: 10.1038/s41392-026-03002-x

Authors: Gianluca Figlia, Juliane Bremer

Institution: Universitätsklinikum Aachen

Language: English

Topic: Amyotrophic Lateral Sclerosis Research

Subfield: Neurology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,790