Vallabh Lab
Principal Investigator: Sonia Minikel Vallabh
Institution: Broad Institute (Cambridge, MA)
Primary Subfield: Neuroscience
Recruitment Status: Inquire for rotation/fellowship opportunities
Research Focus
PROJECT SUMMARY Prion disease is a rapidly fatal neurodegenerative disease that arises from a single protein, the native prion protein (PrP), which is capable of post-translational conversion into a self-templating and deadly conformer termed a prion. The centrality of PrP to this process, as the essential substrate for disease, has long led to the therapeutic hypothesis of PrP reduction in the brain. However, despite strong genetic evidence and pharmacological proof of concept using antisense oligonucleotides, whole-brain PrP reduction has been difficult to achieve at scale with large gene, biologic or even oligonucleotide therapeutics. We have recently identified two small molecules with PrP-lowering properties that may point towards more therapeutically facile ways to achieve this end, while simultaneously illuminating new facets of PrP’s biology and basal regulation. Following their discovery through a high-throughput immunofluorence-based phenotypic screen, we have now confirmed both potency and considerable proteomic selectivity with PrP among the top two downregulated proteins in cells following treatment with either compound. We now seek to take the following steps to deepl…
Active NIH Awards
- 5R01NS131663-04: Mechanism of Action of Prion Protein-Lowering Small Molecules (2026)
- 5R01NS125255-05: How Substrate Dosage Drives Prion Disease Kinetics (2026)
- 4U19NS132315-04: Research Project 1 (2026)
- 4U19NS132315-04: Therapeutic Editing to Lower PrP in Prion Disease (2026)
- 4U19NS132315-04: Therapeutic editing to lower PrP in prion disease: Administrative Core (2026)
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