A recent study published in PLOS Pathogens by Dr. Rodrigo Morales and Rebeca Benavente sheds light on the evolving landscape of therapeutic strategies for prion diseases in humans and animals. Despite decades of research, prion diseases remain fatal with no significant curative treatments. This comprehensive review discusses promising approaches targeting the misfolded prion protein (PrP^Sc) and its cellular counterpart (PrP^C), as well as associated pathological pathways.
The study highlights strategies such as reducing PrP^C expression through antisense oligonucleotides, which has shown potential in increasing survival rates in experimental models. Additionally, the use of antibodies, small molecules targeting PrP^Sc, and even unconventional methods like prion-interference and immunotherapy are explored. However, challenges remain, including the variability of prion strains and the risk of drug-resistant prion variants.
While significant hurdles persist, the research emphasizes the potential of combinatory therapies targeting both PrP^C and PrP^Sc, along with downstream mechanisms like cellular toxicity. These findings open new avenues for future research aimed at developing effective treatments for these devastating neurodegenerative diseases.
