Peter Harley

This study uses hPSCs to model MED with MATN3 mutations, revealing altered matrix organisation and upregulated cholesterol biosynthesis pathway.

The paper develops U7 snRNA therapeutics to correct TDP-43-dependent mis-splicing in ALS, restoring gene expression and neuronal function in human iPSC-derived neurons.

The paper develops TDP-REG, a system using cryptic splicing to enable precise gene therapy activation in diseased neurons for ALS/FTD.

This study shows that loss of TDP-43 causes synaptic dysfunction through a cryptic exon in UNC13A, which is rescued by antisense oligonucleotides.

This study shows that TDP-43G298S mutations in human iPSC-derived motor neurons alter axon initial segment structure and neuronal excitability at different developmental stages.

The paper characterizes immune responses in a neuropathic pain mouse model and develops a web-based tool for transcriptional data-mining of peripheral neuroimmune interactions.