Lucia Yang

This study demonstrates that exon-skipping antisense oligonucleotides targeting H3.3K27M-altered DMG reduce proliferation and extend survival in a mouse model.

This study develops a cocktail of ASOs to specifically increase CFTR W1282X mRNA and protein levels by inhibiting NMD, improving chloride current in human bronchial epithelial cells.

This paper develops an exon-skipping antisense oligonucleotide (ASO) strategy to increase CFTR protein levels in patients with the W1282X mutation by bypassing nonsense-mediated mRNA decay.