Preclinical evaluation of antisense oligonucleotide therapy in a mouse model of HNRNPH2-related neurodevelopmental disorder

Ane Korff, Xiaojing Yang, Ozan Ozdemir, Ananya Samanta, Yong-Dong Wang, Tushar Patni, Alfonso J. Lav / science - Science Translational Medicine, Volume 18, Issue 846, April 2026.

AI Summary: Researchers report that antisense oligonucleotide therapy reversed neurological deficits in mouse models of HNRNPH2‑related neurodevelopmental disorder. The preclinical results provide a targeted mechanism to correct pathogenic RNA processing, moving a once‑untreatable condition toward a plausible therapeutic path — pending the usual caution about translating mice to humans.