Fig. 3.

Functional significance of HOXB-AS3 expression in AML patient blasts. a Relative HOXB-AS3 RNA expression in AML blasts from three patients with NPM1 mutations (NPM1mut), treated with scramble versus anti-HOXB-AS3 gapmers (HOXB-AS3 KD). b Number of colonies formed by scramble versus HOXB-AS3 KD AML patient blasts in colony-forming unit assays. Experiments were conducted in triplicates. c Relative HOXB-AS3 RNA expression in AML blasts from a NPM1mut AML patient treated in vivo with scramble or anti-HOXB-AS3 gapmers. d Schematic representation of the study design testing therapeutic value of in vivo HOXB-AS3 KD. e Kaplan–Meier curves depicting survival of NSG mice xeno-transplanted with blasts of NPM1mut AML patients treated with scramble (n = 11) or anti-HOXB-AS3 gapmers (n = 11). f, g Frequency of BFP-expressing cells transduced with either anti-Hoxb5os gRNAs or non-targeting controls over a 15-day period of competitive co-culture with non-transduced cells of the f Rosa26^Cas9/+;Npm1^cA/+;Flt3^ITD/+ or the g Rosa26^Cas9/+;Npm1^cA/+;Nras^G12D/+ murine AML cell lines. gRNAs targeting the Aurkb gene were used as a pan-essential positive controls. In (a–c), P values were calculated using paired two-sided t-tests. Heights of boxplots indicate mean values with standard deviation. Error bars indicate highest and lowest values in each population. In (e–g), P values were calculated using the log-rank test. Source data are provided as a Source Data file.