Daniel Urrutia-Cabrera

Mar 9, 2024 | biorxiv.org | Roxanne Liu |Daniel Urrutia-Cabrera |Ida Westin |Irina Golovleva

AbstractDysregulation of the alternative splicing process results in aberrant mRNA transcripts, leading to dysfunctional proteins or nonsense-mediated decay that cause a wide range of mis-splicing diseases. Development of therapeutic strategies to target the alternative splicing process could potentially shift the mRNA splicing from disease isoforms to a normal isoform and restore functional protein.