Retinitis pigmentosa (RP) is an inherited retinal dystrophy that causes loss of vision. Pathogenic variants in proteins involved in RNA splicing are the second most common cause of autosomal dominant RP, with mutations in PRPF31 being the most prevalent. Additionally, mutations in spliceosomal small nuclear RNAs (snRNAs) U4 and U6 have recently been linked to RP.

Researchers from McGill University and collaborating institutions aimed to investigate whether oligonucleotides are a viable drug class to prevent hydrocephalus.

Three decades of trial-and-error, and the resulting safety data, in the oligonucleotide-based therapeutic space have paved way for the present-day innovations and the promise of “programmable,” precision medicine for patients, speakers at Bio Korea 2026 said April 28.