Article

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

2026-02-04

Abstract excerpt

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 (S...

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Literature Corpus work: 94732ee3-171c-52a9-8bde-eaeb68a424b2
DOI: 10.64898/2026.01.28.26345012

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