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When a tissue-specific gene is totally inactivated by a mutation, it may cause severe disease but will not necessarily have interfered with embryonic development

JAMA 323(12):11751183

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This offers an additional large potential revenue stream IF IND application is successful AND additional clinical trials show significant benefits

In total, 5 studies included patients with T2DM (2427, 29) while another 6 studies evaluated patients with overweight or obesity without T2DM (10, 28, 30, 31, 33, 34)