Karen Ling

Enhanced splicing modulation by NMA-modified antisense oligonucleotides

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

NMNAT2-SARM1 Axis Drives Redox Failure and Disrupts APP Processing in Neurons

This study shows that NMNAT2 loss disrupts APP processing in neurons by causing redox failure and proteostasis disruption, with SARM1 playing a key role in this process.

TDP-43 pathology triggers SRRM4-dependent cryptic splicing of G3BP1 in ALS/FTD

The paper shows that TDP-43 pathology causes a cryptic exon in G3BP1 to be included in neurons, leading to a protein that disrupts stress granule dynamics in ALS/FTD.

Reduction of RAD23A extends lifespan and mitigates pathology in TDP-43 mice

This study shows that reducing RAD23A in TDP-43 mice improves survival, behavior, and reduces TDP-43 pathology and transcriptomic alterations.

Antisense oligonucleotides targeting the miR-29b binding site in the GRN mRNA increase progranulin translation

This study shows that antisense oligonucleotides can increase progranulin protein levels by blocking miR-29b binding in GRN mRNA, offering a potential therapy for FTD.

Early activation of cellular stress and death pathways caused by cytoplasmic TDP-43 in the rNLS8 mouse model of ALS/FTD

This study identifies early activation of stress and apoptotic pathways in a mouse model of ALS/FTD and shows that suppressing Chop has limited effects on disease progression.

NAD homeostasis maintained by NMNAT2 supports vesicular glycolysis and fuels fast axonal transport in distal axons of cortical glutamatergic neurons in mice

This study shows NMNAT2 maintains NAD redox potential in axons to support glycolysis and fast transport, preventing axonal degeneration in mice.