Antisense oligonucleotide strategy reverses HNRNPH2-related neurodevelopmental disorder

MedicalXpress | Apr 26, 2026

Scientists at St. Jude Children's Research Hospital have found that they can reverse the effects of HNRNPH2-related neurodevelopmental disorder using antisense oligonucleotides (ASOs) in preclinical models. ASOs are short synthetic nucleic acid strands that target specific messenger RNA. In work published in Science Translational Medicine, the researchers show that ASOs block production of the aberrant HNRNPH2 protein. This consequently boosts expression of the closely related HNRNPH1 protein, reducing multiple symptoms of the disorder. The work provides vital mechanistic data to support the advancement of this promising therapy to clinical studies.