Meredith A Mortberg

Prevention of prion disease symptoms in mice with deep PrP lowering

This study demonstrates that deep PrP lowering with a potent siRNA prevents prion disease symptoms in mice and extends survival in symptomatic animals.

Cell type distribution of intrathecal antisense oligonucleotide activity in deep brain regions of non-human primates

The paper assesses the cell-type-specific activity of intrathecally delivered antisense oligonucleotides in deep brain regions of non-human primates, finding broad target engagement across neuronal and non-neuronal cells.

Zinc Finger Repressors mediate widespread PRNP lowering in the nonhuman primate brain and profoundly extend survival in prion disease mice

This study demonstrates that Zinc Finger Repressors (ZFRs) reduce prion protein (PrP) expression in nonhuman primates and extend survival in prion disease mice.

Modulation of prion protein expression through cryptic splice site manipulation

This study identifies a cryptic exon in the human PRNP gene that can reduce prion protein expression when included in mRNA, offering a new therapeutic target for prion disease.

A single-cell map of antisense oligonucleotide activity in the brain

The paper maps antisense oligonucleotide activity in the brain using single-cell transcriptomics in mice and macaques, showing widespread target suppression across cell types with varying durations.

Analysis of non-human primate models for evaluating prion disease therapeutic efficacy

The paper evaluates the feasibility of using non-human primate models to assess prion disease therapeutic efficacy by analyzing incubation times and model characteristics.