公开(公告)号：US20230193281A1

公开(公告)日：2023-06-22

申请号：US17982993

申请日：2022-11-08

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Anastasia Khvorova ,  James W. Gilbert ,  Chantal Ferguson ,  Robert Brown ,  Alexandra Weiss

IPC: C12N15/113 ,  A61P25/28

CPC classification number: C12N15/1137 ,  A61P25/28 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2320/32

Abstract: This disclosure relates to novel SOD1 targeting sequences. Novel SOD1 targeting oligonucleotides for the treatment of SOD1-related familial amyotrophic lateral sclerosis (ALS).