公开(公告)号：US20240294905A1

公开(公告)日：2024-09-05

申请号：US17921922

申请日：2021-04-26

Applicant: Universität Ulm

Inventor： Tobias Böckers ,  Stefanie Pfänder ,  Nadine Stirmlinger

IPC: C12N15/113 ,  G01N33/53

CPC classification number: C12N15/113 ,  G01N33/53 ,  C12N2310/11 ,  G01N2800/52

Abstract: The present invention relates to an oligonucleotide specifically binding to a polynucleotide comprising the nucleic acid sequence of SEQ ID NO:1. The present invention also relates to said oligonucleotide for use in medicine and for use in treatment and/or prevention of Shank3 deficiency, preferably for use in treatment and/or prevention of Phelan-McDermid syndrome. The present invention also relates to expression constructs, host cells, and methods related thereto.