Exon7 Targeted CRISPR-Prime Editing Approaches for SMN2 Gene Editing in Spinal Muscular Atrophy (SMA) Disease Can Increase In Vitro SMN Expression
Sibel Pınar Odabaş, Enes Bal, Gamze Yelgen, Ayse Simay Metin, Ebrar Karakaya, Gamze Gülden, Berranur Sert, Tarık Teymur, Yasin Ay, Nulifer Neslihan Tiryaki, Hasret Araz, Ilayda Cavrar, Cihan Taştan
This study uses CRISPR-Prime Editing to correct two point mutations in the SMN2 gene, increasing SMN expression in SMA-related cells.
bioRxiv · Mar 21, 2022 · PreprintGenetics