Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease 机翻标题: 暂无翻译,请尝试点击翻译按钮。

源语言标题
(JP2016027823) 疾患​の​処置​の​ため​の​トリシクロ−DNA​アンチセンスオリゴヌクレオチド、組成​物​及び​方法
公开号/公开日
JP2016027823 A 2016-02-25 [JP2016027823] / 2016-02-25
申请号/申请日
2015JP-0197054 / 2015-10-02
发明人
DANIEL SCHUEMPERLI;CHRISTIAN LEUMANN;DENIS FURLING;LUIS GARCIA;THOMAS VOIT;
申请人
ASSOCIATION INSTITUT DE MYOLOGIE;CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE CNRS;UNIVERSITE PIERRE & MARIE CURIE;UNIVERSITY OF BERN;
主分类号
IPC分类号
A61K-031/711A61K-048/00A61P-021/04A61P-025/00A61P-043/00C12N-015/09C12N-015/113
摘要
(JP2016027823) PROBLEM TO BE SOLVED: To provide synthetic antisense oligonucleotides (AON) and methods employing AON for modifying splicing events that occur during pre-mRNA processing or for down-regulating the expression of mutated mRNA, for the treatment of diseases such as Duchenne Muscular Dystrophy.SOLUTION: A tricyclo-DNA antisense oligonucleotide (tc-DNA AON) for facilitating the skipping of an exon during processing of a dystrophin pre-mRNA, said tc-DNA AON containing between 10 and 18 tricyclo nucleotides, wherein 8-16 or 6-14 nucleotides of said tc-DNA AON are complementary to a dystrophin pre-mRNA intronic splice donor site, wherein 2-8 nucleotides of said tc-DNA AON are complementary to a dystrophin pre-mRNA exonic region, and wherein said intronic splice donor site is contiguous with 5' side of said exonic region.SELECTED DRAWING: None
机翻摘要
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地址
代理人
代理机构
;
优先权号
2009US-61212384 2009-04-10
主权利要求

        
法律状态
(JP2016027823) LEGAL DETAILS FOR JP2016027823  Actual or expected expiration date=2030-04-09    Legal state=ALIVE    Status=PENDING     Event publication date=2015-10-02  Event code=JP/APP  Event indicator=Pos  Event type=Examination events  Application details  Application country=JP JP2015197054  Application date=2015-10-02  Standardized application number=2015JP-0197054     Event publication date=2015-11-30  Event code=JP/A521  Event type=Restitution or restoration  Written amendment  Effective date of the event=2015-11-30  JAPANESE INTERMEDIATE CODE: A523     Event publication date=2016-02-25  Event code=JP/A  Event indicator=Pos  Event type=Examination events  Published application  Publication country=JP  Publication number=JP2016027823  Publication stage Code=A  Publication date=2016-02-25  Standardized publication number=JP2016027823     Event publication date=2016-09-06  Event code=JP/A131  Event indicator=Neg  Event type=Examination events  Notification of reasons for refusal  Effective date of the event=2016-09-06  JAPANESE INTERMEDIATE CODE: A131     Event publication date=2016-12-06  Event code=JP/A601  Event type=Examination events  Written request for extension of term  Effective date of the event=2016-12-06  JAPANESE INTERMEDIATE CODE: A601     Event publication date=2017-01-26  Event code=JP/A601  Event type=Examination events  Written request for extension of term  Effective date of the event=2017-01-26  JAPANESE INTERMEDIATE CODE: A601     Event publication date=2017-03-03  Event code=JP/A521  Event type=Restitution or restoration  Written amendment  Effective date of the event=2017-03-03  JAPANESE INTERMEDIATE CODE: A523     Event publication date=2017-08-08  Event code=JP/A131  Event indicator=Neg  Event type=Examination events  Notification of reasons for refusal  Effective date of the event=2017-08-08  JAPANESE INTERMEDIATE CODE: A131
专利类型码
A
国别省市代码
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