Antisense oligonucleotides targeting ACTL6B

The present invention relates to antisense oligonucleotide splicing modulators of actin-like 6B (ACTL6B). These antisense oligonucleotide splice modulators are complementary, such as fully complementary, to ACTL6B precursor mRNA, and are capable of increasing or restoring expression of ACTL6B in TDP-43 depleted cells, such as for TDP-43 functional depleted conditions and medical indications. 本发明涉及肌动蛋白样6B(ACTL6B)的反义寡核苷酸剪接调节剂。这些反义寡核苷酸剪接调节剂与ACTL6B前体mRNA互补，诸如完全互补，并且能够在TDP-43耗竭的细胞中增加或恢复ACTL6B的表达，诸如用于TDP-43功能耗竭的病况和医学适应症。