King, Robert M.

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

Alterman, J. F., Godinho, B. M. D. C., Hassler, M. R., Ferguson, C. M., Echeverria, D., Sapp, E., Haraszti, R. A., Coles, A. H., Conroy, F., Miller, R., et al. (2019) · Nature Biotechnology

RNA Interference and Gene DeliveryDNA and Nucleic Acid Chemistry

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Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide

Tran, H., Moazami, M. P., Yang, H., McKenna-Yasek, D., Douthwright, C. L., Pinto, C., Metterville, J., Shin, M., Sanil, N., Dooley, C., et al. (2022) · Nature Medicine

Amyotrophic Lateral Sclerosis ResearchRNA Interference and Gene Delivery

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Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep

Ferguson, C. M., Godinho, B. M. D. C., Alterman, J. F., Coles, A. H., Hassler, M., Echeverria, D., Gilbert, J. W., Knox, E. G., Caiazzi, J., Haraszti, R. A., et al. (2021) · JCI Insight

RNA Interference and Gene DeliveryBarrier Structure and Function Studies

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