Hien Tran Zhao

The paper measures PrP half-life in vivo and shows it limits the time to effect of PrP-lowering therapies, with implications for drug development and clinical trial design.

This study shows that translational control in spinal cord neurons, particularly in inhibitory and PV+ interneurons, significantly influences gene expression and pain hypersensitivity during the chronic phase of neuropathic pain.

This paper demonstrates that antisense oligonucleotides targeting human ATN1 protect against DRPLA pathology in a fully humanized mouse model.

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.

This study shows that SARM1 activation causes mitochondrial dysfunction in human neurons, which can be reversed by nicotinamide or prevented by antisense oligonucleotides.

This study evaluates the analgesic effects of intrathecal NaV 1.7 antisense oligonucleotides in rats, showing reduced channel binding and pain behaviors with concentration-dependent knock-down of Scn9a mRNA.

The paper characterizes a transient neurobehavioral response to high-dose oligonucleotide delivery in the CNS and provides a framework to mitigate it through magnesium supplementation.

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.

The paper maps ASO distribution and activity in the CNS of rodents and NHP after central administration, showing widespread effects across cell types.

This study shows that lowering prion protein expression with ASOs is effective in treating prion disease across different strains, treatment times, and endpoints.

This paper shows that reducing PLP1 expression in a mouse model of PMD improves myelination, motor function, and lifespan, supporting PLP1 suppression as a therapeutic strategy.

This paper demonstrates that antisense oligonucleotides targeting SNCA reduce alpha-synuclein production, prevent pathology, and show translational potential in non-human primates.