Pharmacodynamic biomarker development for antisense oligonucleotide therapy

I am evaluating RAN-translated peptides as a potential biomarker for antisense oligonucleotide therapy in C9orf72-mediated ALS/FTD.

Recent Publications

Gendron, T. F.*, Chew, J.*, Stankowski, J. N.*, Hayes, L. R.*, Zhang, Y.-J., Prudencio, M., et al. (2017). Poly(GP) proteins are a useful pharmacodynamic marker for C9ORF72-associated amyotrophic lateral sclerosis. Science Translational Medicine, 9(383), eaai7866. http://doi.org/10.1126/scitranslmed.aai7866. *These authors contributed equally to this work.

Hayes, L. R., & Rothstein, J. D. (2016). C9ORF72-ALS/FTD: Transgenic Mice Make a Come-BAC. Neuron, 90(3), 427–431. http://doi.org/10.1016/j.neuron.2016.04.026