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The in vitro application of RNA-targeting Cas13 devices enables the direct reduction of hazardous repetitive RNAs in patient cells, facilitating swift assessments of guide efficacy and collateral activity prior to in vivo experimentation

In nonclinical mouse studies, animals with genetic melanocortin pathway defects were more responsive to setmelanotide than wild-type mice ( Important discrepancies exist between setmelanotides exposure levels in nonclinical studies and clinical trials

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Borrelli et al