专利检索 ap:("Hoffmann-La Roche Inc.") AND inv:"Bettina NORDBO" 第 1 页

1.

发明公开
ANTISENSE OLIGONUCLEOTIDES TARGETING ACTL6B 审中-公开

公开(公告)号：US20240318180A1

公开(公告)日：2024-09-26

申请号：US18735668

申请日：2024-06-06

申请人： Hoffmann-La Roche Inc.

发明人： Lars JOENSON , Bettina NORDBO , Jonas VIKESAA

IPC分类号： C12N15/113

CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/3233 , C12N2320/33

摘要： The present invention relates to antisense oligonucleotide splice modulators of actin-like 6B (ACTL6B). These antisense oligonucleotide splice modulators are complementary, such as fully complementary, to the ACTL6B precursor-mRNA, and are capable of increasing or restoring expression of ACTL6B in TDP-43 depleted cells, such as for use in conditions and medical indications where TDP-43 is functionally depleted.