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Quantitative analysis of lanosterol levels in lenses via LC-MS Wistar rats aged 3 or 8 weeks were anesthetized and intracamerally injected with 1.5 L of mLNPs according to the above protocol

While a number of promising therapies for SMA are emerging, defining therapeutic windows and developing sensitive and relevant biomarkers are critical to facilitate potential success in clinical trials

doi: 10.1128/JVI.79.10.6349-6357.2005

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