Genetic & Genomic MedicineGenetics in MedicinePeer-reviewed
Screening rare genetic diagnoses for amenability to bespoke antisense oligonucleotide therapy development: a retrospective cohort study
David Cheerie, Marlen C. Lauffer, Logan Newton…·Evgueni A. Ivakine
The paper estimates the proportion of genetic diagnoses in a pediatric cohort that are amenable to antisense oligonucleotide therapy, finding 4.9% eligible or likely eligible.
6 citations