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Abstract
TDP-43 nuclear depletion in amyotrophic lateral sclerosis (ALS) causes de-repression of cryptic exons in multiple transcripts.
- Mis-splicing associated with TDP-43 depletion disrupts synaptic transmission and neurite growth.
- A therapeutic U7 snRNA (tU7) approach may suppress TDP-43-dependent mis-splicing.
- tU7 has the potential to restore target gene expression in human iPSC-derived neurons.
- Neuronal functions can be rescued by the tU7-mediated splicing correction strategy.
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