Overall selectivity of KT777 knockdown ( KCNT1 downregulated at an adjusted P value ( P adj ) = 1.7 × 10 −34 ; 11 additional downregulated genes nominally significant at P adj < 0.05; Supplementary Table 5 ) was similar to that of RNase H-activating ASOs BIIB067 and HTTRx (targeting SOD1 and HTT , respectively) (Supplementary Figs. 1 and 2 ).
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Antisense oligonucleotide-mediated knockdown therapy in two infants with severe KCNT1 epileptic encephalopathy.
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