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Gene Therapy

Genetic Medicines Mitochondrial Health Down's Syndrome Treatments Non-coding RNA Therapeutics mRNA Vaccines Extracellular Vesicles

Toronto Team Synthesizes Functional lncRNAs, Shows Preclinical Anti-Inflammatory Effects

The study outlines a tunable RNA-based approach that mirrors mRNA therapeutics to target inflammation in early preclinical tests.

Excess HMGN1 Identified as Driver of Down Syndrome Heart Defects

Nature Study Flags ABHD18 Inhibition as a Preclinical Path to Treating Barth Syndrome