(CA)n oligonucleotides correct RNA mis-splicing in TDP-43 pathology (II)

ABSTRACT: The occurrence of extensive cryptic splicing following the depletion of nuclear TDP-43 results in impaired neuronal function and degeneration. Here, we show that oligonucleotides containing CA-repeat sequences can mimic TDP-43 pre-mRNA binding and broadly repress cryptic splicing events, reverse protein loss, and restore neuronal activity in TDP-43-depleted neurons. Our results indicate that (CA)n oligonucleotides are potential therapeutic agents to address global mis-splicing in TDP-43 proteinopathies.

ORGANISM(S): Homo sapiens

PROVIDER: GSE250255 | GEO | 2023/12/23

REPOSITORIES: GEO