We have written about the promise of gene therapy to correct genetic disease many times. An exciting development happened ...

Although heart cells and skin cells contain identical instructions for creating proteins encoded in their DNA, they're able ...

Obtained ex-US Regulatory Clearance for DT-818, a Potentially Best-in-Disease Treatment for Myotonic Dystrophy Type-1 (DM1) Trials of DT-216P2 ...

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered ...

A new Northwestern University study using patient nervous tissue and lab-grown human neurons has uncovered how a key disease ...

Subscribe to our newsletter for the latest sci-tech news updates. Published in Nature Communications, scientists from the Wellcome Sanger Institute reveal insights into how our genes influence the ...

Northwestern researchers were able to calm overactive neurons and restore neuronal activity, which could potentially slow the progression of disease.