Structural basis of a small molecule targeting RNA for a specific splicing correction

UCSF Chimera—A visualization system for exploratory research and analysis

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Computing, Bioinformatics, Visualization +17

The Amber biomolecular simulation programs

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Natural Sciences, Biophysics, Chemistry +17

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

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Automated NMR Structure Calculation With CYANA

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Natural Sciences, Chemistry, Computational Chemistry +15

Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model

Yimin Hua, Kentaro Sahashi, Frank Rigo et al. · Nature · 2011 · 743 citations · Full text

Medicine, Neurobiology, Neuroscience +9