公开(公告)号：US20240360443A1

公开(公告)日：2024-10-31

申请号：US18609277

申请日：2024-03-19

Applicant: Dystrogen Gene Therapies Inc.

Inventor： Marta OLEJNICZAK ,  Anna KOTOWSKA-ZIMMER ,  Marianna PEWINSKA ,  Paul LEWICKI ,  Krzysztof SIEMIONOW

IPC: C12N15/113 ,  A61P25/28 ,  C12N15/86

CPC classification number: C12N15/113 ,  A61P25/28 ,  C12N15/86 ,  C12N2310/141 ,  C12N2310/531 ,  C12N2750/14143

Abstract: A nucleic acid molecule composed of a duplex and loop, in which one of the duplex strands, the guide strand, comprises a sequence chosen from SEQ ID NO. 1-4, and the other strand of the duplex, the passenger strand, is at least 80% complementary to the guide strand, wherein the nucleic acid molecule forms a hairpin structure in a cell.