Minikel Lab
Principal Investigator: Eric Vallabh Minikel
Institution: Broad Institute (Cambridge, MA)
Primary Subfield: Neuroscience
Recruitment Status: Inquire for rotation/fellowship opportunities
Research Focus
PROJECT SUMMARY Prion disease is an incurable and uniformly fatal neurodegenerative disease of humans and other mammals. PrP lowering is a pharmacologically validated therapeutic strategy for prion disease, and antisense oligonucleotides (ASOs) with this mechanism of action are currently advancing toward the clinic. The promise of this progress, and indeed our ability to advance any therapy for prion disease, will critically depend on our capacity to leverage molecular biomarkers as windows into the brain. To date the molecular states of the brain during lifelong genetic risk prior to disease, first prion formation, and early phases of prion replication all remain elusive, and yet, it is precisely these upstream disease stages that have shown themselves most amenable to PrP-lowering intervention. To become a clinical reality, early treatment paradigms will require biomarkers to report on drug safety and efficacy but also the earliest molecular stages of risk and disease; critical decisions about trial eligibility, timing of treatment, and advancement or termination of whole experimental drug programs will rest on our ability to accurately interpret and mechanistically contextualize…
Active NIH Awards
- 5R01NS132022-03: Biomarkers and Mechanisms of PrP Misfolding, Mutation, and Deficiency (2026)
- 4U19NS132315-04: Research Project 2 (2026)
- 4U19NS132315-04: Research Project 3 (2026)
- 1R21NS144519-01: Prion Clearance and Treatment of Recalcitrant Prion Strains Through Deep PrP Suppression (2025)
- 5R01NS132022-02: Biomarkers and Mechanisms of PrP Misfolding, Mutation, and Deficiency (2025)
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