Christopher Donnelly, PhD

Christopher Donnelly, Ph.D., is an Associate Professor of Neurology at the Peter O’Donnell Jr. Brain Institute at UT Southwestern and Neuromuscular Section Chief. He earned his Ph.D. from the University of Delaware, where he studied axonal RNA transport and local translation in nerve regeneration with Dr. Jeff Twiss, and completed postdoctoral training with Dr. Jeff Rothstein at Johns Hopkins School of Medicine, investigating cellular dysfunction in C9orf72-associated ALS and FTD. Prior to joining UT Southwestern in 2026, he was the founding Scientific Director of the LiveLikeLou Center for ALS Research at the University of Pittsburgh School of Medicine.
 
The Donnelly lab focuses on the molecular mechanisms underlying neurodegenerative disease, with an emphasis on how dysfunction of RNA-binding proteins, including TDP-43 and FUS, drives pathology and neuronal vulnerability in C9orf72 and sporadic ALS/FTLD. His laboratory investigates how RNA regulates protein function and phase behavior, and how defects in nuclear and axonal transport and pathogenic protein interactions drive ALS/FTLD. The goal of this work is to identify strategies to restore normal protein function in disease. The Donnelly lab integrates patient-derived neuronal models with RNA-based therapeutic approaches to target disease mechanisms. Dr. Donnelly’s prior research identified key disease pathways, including nucleocytoplasmic transport defects, and advanced therapeutic strategies targeting toxic C9orf72 gene products with antisense oligonucleotide (ASO) therapies. More recently, his lab developed short RNA oligonucleotides (bait/chaperone RNAs) approaches to reverse pathological protein aggregation and restore physiological function in ALS/FTLD.