Thu Huyen Nguyen

Antisense oligonucleotide selection scheme for rare Duchenne muscular dystrophy mutations: Application to DMD exon 16 skipping

Over the last decades, antisense oligonucleotides (AONs) have emerged as powerful tools to modulate alternative splicing, allowing exclusion of a specific exon from a target mRNA to bypass mutations or restore the reading frame, enabling production of a functional protein. In Duchenne muscular dystrophy (DMD), a lethal...