A Rapid and Scalable Approach for Screening Personalized ASOs

Scott Younger, director of disease gene engineering at Children Mercy Kansas City’s Genomic Medicine Center, discusses a process he developed to rapidly and inexpensively create organoids from a person’s induced pluripotent stem cells, how the process was used to match three children with Duchenne muscular dystrophy to an antisense oligonucleotide therapy, and the potential impact this may have on developing customized therapies for people with rare genetic diseases.