GeneticsbioRxivPreprint
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…·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.
DOI 10.1101/2023.08.21.552929