[HTML][HTML] Antisense oligonucleotides in therapy for neurodegenerative disorders

MM Evers, LJA Toonen… - Advanced drug delivery …, 2015 - Elsevier
Antisense oligonucleotides are synthetic single stranded strings of nucleic acids that bind to
RNA and thereby alter or reduce expression of the target RNA. They can not only reduce …

Development of phosphorothioate DNA and DNA thioaptamers

DE Volk, GLR Lokesh - Biomedicines, 2017 - mdpi.com
Nucleic acid aptamers are short RNA-or DNA-based affinity reagents typically selected from
combinatorial libraries to bind to a specific target such as a protein, a small molecule, whole …

PrP is a central player in toxicity mediated by soluble aggregates of neurodegeneration-causing proteins

GT Corbett, Z Wang, W Hong, M Colom-Cadena… - Acta …, 2020 - Springer
Neurodegenerative diseases are an enormous public health problem, affecting tens of
millions of people worldwide. Nearly all of these diseases are characterized by …

Prion protein lowering is a disease-modifying therapy across prion disease stages, strains and endpoints

EV Minikel, HT Zhao, J Le, J O'Moore… - Nucleic acids …, 2020 - academic.oup.com
Lowering of prion protein (PrP) expression in the brain is a genetically validated therapeutic
hypothesis in prion disease. We recently showed that antisense oligonucleotide (ASO) …

[HTML][HTML] Antisense oligonucleotides extend survival of prion-infected mice

GJ Raymond, HT Zhao, B Race, LD Raymond… - JCI insight, 2019 - ncbi.nlm.nih.gov
Prion disease is a fatal, incurable neurodegenerative disease of humans and other
mammals caused by conversion of cellular prion protein (PrP C) into a self-propagating …

Intriguing nucleic-acid-binding features of mammalian prion protein

JL Silva, LMTR Lima, D Foguel, Y Cordeiro - Trends in biochemical …, 2008 - cell.com
In transmissible spongiform encephalopathies, the infectious material consists chiefly of a
protein, the scrapie prion protein PrP Sc, that carries no genetic coding material; however …

Developing therapeutics for PrP prion diseases

K Giles, SH Olson, SB Prusiner - Cold Spring …, 2017 - perspectivesinmedicine.cshlp.org
The prototypical PrP prion diseases are invariably fatal, and the search for agents to treat
them spans more than 30 years, with limited success. However, in the last few years, the …

Unraveling prion protein interactions with aptamers and other PrP-binding nucleic acids

B Macedo, Y Cordeiro - International journal of molecular sciences, 2017 - mdpi.com
Transmissible spongiform encephalopathies (TSEs) are a group of neurodegenerative
disorders that affect humans and other mammals. The etiologic agents common to these …

Nonspecific prion protein–nucleic acid interactions lead to different aggregates and cytotoxic species

B Macedo, TA Millen, CACA Braga, MPB Gomes… - Biochemistry, 2012 - ACS Publications
A misfolded form of the prion protein (PrP) is the primary culprit in mammalian prion
diseases. It has been shown that nucleic acids catalyze the misfolding of cellular PrP into a …

Intracerebral infusion of antisense oligonucleotides into prion-infected mice

KN Friberg, G Hung, E Wancewicz, K Giles… - … Therapy-Nucleic Acids, 2012 - cell.com
Mice deficient for the cellular prion protein (PrP C) do not develop prion disease;
accordingly, gene-based strategies to diminish PrP C expression are of interest. We …