American journal of human genetics2026ResearchNon-viral Gene Delivery
1. Scaling haplospecific antisense oligonucleotides from N-of-1 to broad use in genetic disease populations by diplotyping
Kim-Mcmanus O, Goddard P, Olsson S, Protopsaltis L, Gleeson J, Zhang Q, Et Al.
Antisense oligonucleotides (ASOs) are versatile disease-modifying therapies for genetic diseases. An accelerated US Food and Drug Administration (FDA) pathway enables ASO treatment trial initiation in an individual within a year. However, this rapid N-of-1 pathway lacks extensibility to broad use necessary for…
- N-of-1 therapy
- TruPath
- antisense oligonucleotide
- autism
- basket trial
- causal variant
- disease modifying therapy
- epilepsy
- genetic disease
- genetic therapy