Transcriptomic-guided compound prioritization and proteomics validation for HNRNPU deficiency identify signalling correction
Xuan Ye, Diana Tikhomirova, Marika Oksanen…·Kristiina Tammimies
The paper develops a transcriptomic-guided compound prioritization pipeline and validates it with proteomics, identifying two compounds that partially rescue HNRNPU deficiency phenotypes.