The gene editing treatment targets the relatively common 1717-1G>A splicing mutation, which does not respond to many current CF therapies.
A novel chemo-enzymatic approach to making synthetic 100-unit oligonucleotide single guide RNA (sgRNA) increases both purity and yield, according to its developers at F. Hoffmann-La Roche and ...
This article and associated images are based on a poster originally authored by Donato Tedesco, Tianbing Liu, Dongfang Hu, Nadya Isachenko, Debbie Deng, Paul Diehl and Alex Chenchik and presented at ...