公开(公告)号：US20230407301A1

公开(公告)日：2023-12-21

申请号：US18145604

申请日：2022-12-22

申请人： Sarepta Therapeutics, Inc.

发明人： Frederick J. SCHNELL ,  Baozhong CAI ,  Jason GATLIN ,  Patrick L. IVERSEN

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/3513

摘要： A modified antisense oligonucleotide of about 10 to about 40 nucleobases is disclosed. The oligonucleotide comprises a targeting sequence having a region complementary to at least one string of three or more identical contiguous nucleobases in a target sequence, wherein the target sequence comprises at least one additional nucleobase compared to the region of the targeting sequence and the at least one additional nucleobase has no complementary nucleobase in the region of the targeting sequence, and wherein the targeting region complementary to the at least one string of three or more identical contiguous nucleobases is internal to the targeting sequence.

公开(公告)号：US20210130822A1

公开(公告)日：2021-05-06

申请号：US16754805

申请日：2018-10-18

申请人： SAREPTA THERAPEUTICS, INC.

发明人： Frederick J. SCHNELL ,  Bao CAI ,  Jason GATLIN ,  Patrick L. IVERSEN

IPC分类号： C12N15/113

摘要： A modified antisense oligonucleotide of about 10 to about 40 nucleobases is disclosed. The oligonucleotide comprises a targeting sequence having a region complementary to at least one string of three or more identical contiguous nucleobases in a target sequence, wherein the target sequence comprises at least one additional nucleobase compared to the region of the targeting sequence and the at least one additional nucleobase has no complementary nucleobase in the region of the targeting sequence, and wherein the targeting region complementary to the at least one string of three or more identical contiguous nucleobases is internal to the targeting sequence.

公开(公告)号：US20190241890A1

公开(公告)日：2019-08-08

申请号：US16127600

申请日：2018-09-11

申请人： Sarepta Therapeutics, Inc.

发明人： Patrick L. IVERSEN

IPC分类号： C12N15/113 ,  C12N15/11

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/3233 ,  C12N2310/3513 ,  C12N2320/12 ,  C12N2320/34

摘要： Provided herein are antisense oligonucleotides, compositions comprising antisense oligonucleotides, and methods for the use of antisense oligonucleotides in manipulating translation. Expression of isoforms of proteins expressed from different start codons of the same transcript are inhibited by antisense oligonucleotides, which may also enhance expression of non-target isoforms.