Author

Claire E. Williams

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Open access Sep 2026

RNA trans-splicing treatment for Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a fatal disorder caused by dystrophin mutations, leading to progressive muscle degeneration and cardiac failure. Although AAV-based DMD therapies with innovative designs to overcome the cargo limits of the virus are effective in animal models, these current systems may face clinical...

Ryan H. Hsu, Claire E. Williams, Geraldine Maier et al. · bioRxiv · 0 citations

Open access Sep 2026

An intersectional expression platform for gene complementation using RNA-fragment end joining (REJ)

Gene complementation is a powerful tool for genetic selection and protein functional studies but typically requires extensive screening for complementary components. Here we engineered a platform for gene complementation based on RNA end-joining (REJ) that precisely and efficiently splices separate RNA units into funct...

L. Bachmann, Ryan H. Hsu, K. Hermann et al. · bioRxiv · 0 citations