公开(公告)号：WO2022094320A2

公开(公告)日：2022-05-05

申请号：PCT/US2021/057419

申请日：2021-10-29

Applicant: GARCIA-BLANCO, Mariano A. ,  GALARZA-MUNOZ, Gaddiel

Inventor： GARCIA-BLANCO, Mariano A. ,  GALARZA-MUNOZ, Gaddiel

IPC: C07K14/715 ,  C12N15/113 ,  C12P19/34 ,  A61K31/713 ,  C07H21/04 ,  A61P35/00

Abstract: The present invention includes compositions and methods for treating an autoimmune disorder or a cancer in a subject in need thereof, the method comprising: administering an effective amount of a composition comprising an oligonucleotide that specifically binds a complementary sequence of the Interleukin-7 receptor (IL7R) pre-mRNA that influences splicing of exon 6, wherein the SM-ASO decreases or increases exclusion of exon 6 in IL7R pre-mRNAs and respectively decreases or increases expression of the soluble isoform of IL7R (sIL7R). In certain embodiments, the oligonucleotide is an antisense oligonucleotide (ASO), or a splice-modulating antisense oligonucleotide (SM-ASO).