Armand Soriano

TL;DR: This work used two independent approaches to test whether decreasing ataxin-2 levels could mitigate disease in a mouse model of TDP-43 proteinopathy, and presented a promising alternative therapeutic strategy for ALS that involves targeting ataxIn-2.

TL;DR: The identification of a simplified tris-based GalNAc cluster (THA-GN3), which can be efficiently assembled using readily available starting materials and conjugated to ASOs using a solution phase conjugation strategy, represents a viable approach for enhancing potency of ASO drugs in the clinic without adding significant complexity or cost to existing protocols for manufacturing oligonucleotide drugs.

TL;DR: In this article, the authors present an atlas of ASO distribution and activity in the central nervous system (CNS) of mice, rats, and non-human primates (NHP), species commonly used in preclinical therapeutic development.

TL;DR: The results demonstrate that ASO drugs are well suited for treating a wide range of neurological diseases for which no effective treatments are available, and for the first time the atlas of ASO distribution and activity in the CNS of mice, rats, and non-human primates is presented.

TL;DR: A mechanism for CHMP2B-mediated neuronal dysfunction and a potential therapeutic strategy targeting the TMEM106B gene, a major risk factor for FTD, are reported and targeting TMEM 106B may be relevant to a broad range of FTDs.