Writing in Molecular Therapy Nucleic Acids, researchers hypothesized that using poly(A) tail mimetics to enhance mRNA expression from haploinsufficiency-associated genes could be a disease-modifying ...

In a manuscript published today in the American Journal of Psychiatry titled Long-Read Genome Sequencing in Clinical Psychiatry: RFX3 Haploinsufficiency in a Hospitalized Adolescent With Autism, ...

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There are more than 300 such conditions (haploinsufficiency diseases) and some also lead to developmental delays. "Our research began as a way to help families find new treatment options for these ...