OLIGONUCLEOTIDES TARGETING THE SPLICE ACCEPTOR SITE DOWNSTREAM OF A MICROEXON AS AN INNOVATIVE THERAPY FOR AUTISM
Ainhoa Martínez‐Pizarro, Sara Picó, Mar Álvarez, Julia Pose‐Utrilla, Lise Lolle Holm, Thomas Koed Doktor, Brage Storstein Andresen, José J. Lucas, Lourdes R Desviat
The paper demonstrates that splice-switching antisense oligonucleotides (SSOs) targeting the downstream splice acceptor site of a microexon can modulate microexon inclusion and restore protein levels of ASD risk genes.
NAR Molecular Medicine · Oct 1, 2025Peer-reviewedNeuroscience