A recent study published in Science, “Creation of de novo cryptic splicing for ALS and FTD precision medicine,” describes TDP-REG, an elegant method of reverse-engineering cryptic splicing to achieve precise and targeted protein expression by leveraging #TDP43 loss of function, a hallmark of #ALS and #FTD. This strategy induces expression of specific genes at the first signs of pathology. Thus, TDP-REG could enable therapeutic delivery in the presymptomatic stage of individuals carrying genetic variants of ALS. Congratulations to the team of Oscar G. Wilkins, Max Z. Y. K. Chien, Josette J. Wlaschin, Simone Barattucci, Peter Harley, Francesca Mattedi, Puja R. Mehta, Maria Pisliakova, Eugeni Ryadnov, Matthew J. Keuss, David Thompson, Holly Digby, Lea Knez, Rebecca Simkin, Juan Antinao Diaz, Matteo Zanovello, Anna-Leigh Brown, Annalucia Darbey, Rajvinder Karda, Elizabeth M.C. Fisher, Thomas J. Cunningham, Claire E. Le Pichon, Jernej Ule, and Petro Fratta on their research which further validates QurAlis’ approach in developing precision medicines for ALS, FTD, and other #neurodegenerative and #neurologicaldiseases. https://bit.ly/3XQFlkW