Targeted blocking of gene splicing can dysregulate intron-embedded primary microRNAs (opens in new tab)

Addressing whether antisense oligonucleotides (ASOs)-based targeting of genes embedded with intronic noncoding RNAs (ncRNAs) affects the expression and function of intronic ncRNAs is crucial to the success of ASOs in clinical trials. While studying zebrafish posterior pituitary development—an important neuroendocrine interface—we found that an ASO targeting the slit3 splice site, but not the translation start site, disrupts pituitary axonal morphogenesis. In addition to altered slit3 splicing...