Scaling haplospecific antisense oligonucleotides from N-of-1 to broad use in genetic disease populations by diplotyping

2026-02-04 genetic and genomic medicine Title + abstract only

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Antisense oligonucleotides (ASO) are versatile disease modifying therapies for genetic diseases. An accelerated FD) pathway enables ASO treatment trial initiation in single patients within a year. However, this rapid N-of-1 pathway lacks extensibility to broad use necessary for sustainability. Individualized ASOs bind pre-mRNAs encompassing an entire locus. Thus, ASOs targeting common heterozygous polymorphisms (SNPs) are potentially haplospecific in many patients with dominant disorders. We dev...

Scaling haplospecific antisense oligonucleotides from N-of-1 to broad use in genetic disease populations by diplotyping

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