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Intellia and Regeneron made history in June 2021 with NTLA-2001 when they announced positive early data from the first-ever patients to have their DNA edited with an in vivo CRISPR/Cas9 therapy delivered systemically

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Hippocampal network hyperactivity after selective reduction of tonic inhibition in GABA A receptor alpha5 subunit-deficient mice

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Study characteristics such as sample size (17% of 2 explained, p = 0.01) and ethnicity (31% of 2 , p = 0.02) explained some of the heterogeneity, but source of controls (0% of 2 , p = 0.02) and cancer type ( 2 3 = 5.55, p = 0.14) did not explain much of it