Enhancing In Vitro SMN Protein Expression and Cell Viability through Xeno-Nucleic Acid-Based ASOs in Spinal Muscular Atrophy
Ozum Kilic, Hale Ahsen Babar, Cemre Can Inci, Sibel Pınar Odabaş, Gamze Yelgen, Sevgi Oltan, Sila Kulac, Cihan Taştan
This study evaluates XNA-DNA-ASO sequences for enhancing SMN protein expression and cell viability in SMA fibroblasts, showing improved stability and reduced toxicity compared to conventional ASOs.
bioRxiv · Aug 21, 2023 · PreprintGenetics