Antisense oligonucleotides reverse rare neurodevelopmental disorder in preclinical models

Lucian Nemoiu

St. Jude Children's Research Hospital Apr 22 2026 Scientists at St. Jude Children's Research Hospital have found 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. Published in  Science Translational Medicine , the researchers showed that ASOs block production of the aberrant HNRNPH2 protein. This consequently boosts

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