Gemma Bachmann

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.

Reversable Acute Sedation Response of Phosphorothioate Antisense Oligonucleotides Following Local Delivery to the Central Nervous System

This study characterizes an acute sedation response caused by phosphorothioate ASOs after CNS delivery, showing it is sequence-, dose-, and chemistry-dependent and can be mitigated.

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 therapy in a new rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment.

This paper introduces a new rat model of Alexander disease that exhibits key pathological features and motor impairment, demonstrating that antisense oligonucleotide therapy can reverse GFAP pathology and white matter deficits.