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

Antisense oligonucleotides (ASOs) made for one individual’s extremely rare causal variant usually cannot help others. We show that by designing ASOs against common haplotypes rather than causal variants, a small set of pre-designed ASOs could treat most patients with a genetic disease, turning costly personalized medicine into scalable, population-wide treatment.