C. Frank Bennett

This study identifies LINE-1 retrotransposons as drivers of neuronal senescence in Alzheimer's disease through cytoplasmic DNA sensing and the cGAS-STING pathway.

The paper introduces an automated AI pipeline for correlating chemical and ultrastructural images to enable organelle-specific molecular imaging in cells and tissues.

This paper introduces NMA-modified antisense oligonucleotides that show improved potency compared to MOE-modified SSOs in modulating splicing for SMA and Dravet syndrome.

This study tests antisense oligonucleotides targeting Hnrnph2 in a mouse model, showing reduced Hnrnph2 levels, increased Hnrnph1, and improved phenotypes like seizures and motor function.

This study investigates how different HTT-lowering therapies affect somatic instability in HD mice, finding that transcriptional repression and CAG repeat binding reduce SI without lowering HTT levels.

This study identifies splice-switching antisense oligonucleotides that correct splicing defects in SCN8A, reducing seizures and improving survival in a mouse model of genetic epilepsy.

This paper demonstrates that transient suppression of PTBP1 in aged glial cells can re-activate neurogenesis in murine and human brains, generating new neurons through canonical pathways.

This study shows that reducing huntingtin protein levels decreases somatic instability in CAG-expanded loci in both mouse models and human neurons.

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

This study demonstrates that GalNAc-conjugated ASOs can target cancer cells expressing ectopic ASGP-R, improving splice-switching ASO potency in vitro and in tumor models.

This study shows that reducing huntingtin in peripheral organs of HD mice does not improve central nervous system disease signs.

The paper identifies and characterizes long non-coding RNAs (lncRNAs) as potential therapeutic targets in mammary tumor progression by analyzing RNA-Seq data from mouse models and validating their function in tumor cells.