Molecular BiologySciencePeer-reviewed
Creation of de novo cryptic splicing for ALS/FTD precision medicine
Oscar G. Wilkins, Max Z. Y. J. Chien, Josette J Wlaschin…·Pietro Fratta
The paper develops TDP-REG, a system using cryptic splicing to enable precise gene therapy activation in diseased neurons for ALS/FTD.
39 citationstop 1%