DT
HealthMore in Health→
Preclinical RNA therapy points to a treatment path for an ultrarare neurodevelopmental disorder
Key Takeaways
- St. Jude researchers used an antisense oligonucleotide to suppress aberrant HNRNPH2 in preclinical models.
- The treatment also boosted HNRNPH1 expression and reduced multiple symptoms of the disorder.
- The work offers mechanistic evidence to support advancement toward clinical studies for an ultrarare disease.
DE
DT Editorial Team··via medicalxpress.com
