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1. AR cooperates with SMAD4 to maintain skeletal muscle homeostasis

3. Control of backbone chemistry and chirality boost oligonucleotide splice switching activity.

5. Variants in ATP6V0A1 cause progressive myoclonus epilepsy and developmental and epileptic encephalopathy.

6. Modelling Neuromuscular Diseases in the Age of Precision Medicine.

7. Gene therapy with AR isoform 2 rescues spinal and bulbar muscular atrophy phenotype by modulating AR transcriptional activity.

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