Raymond, G. J et al. JCI Insight 4, e131175 (2019)
To date, no treatment is available for transmissible spongiform encephalopathies (TSEs), a group of fatal neurodegenerative diseases caused by a conformational change of the cellular prion protein (PrP) in the central nervous system of humans and other mammals. Previous work has shown that PrP depletion in mice is protective against prion disease, leading researchers to investigate the potential of PrP-lowering therapies. However, limitations such as poor tolerability of drug delivery by osmotic pumps have hindered the development of RNA interference therapy. By showing that intracerebroventricular bolus injection of antisense oligonucleotides (ASO)s complementary to Prnp mRNA could extend the survival of prion-infected mice, a study brings new hope for ASO therapy for prion disease.
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Le Bras, A. Antisense therapy for prion disease. Lab Anim 48, 330 (2019). https://doi.org/10.1038/s41684-019-0429-0
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DOI: https://doi.org/10.1038/s41684-019-0429-0