JP2017519766A5 - - Google Patents

Download PDF

Info

Publication number
JP2017519766A5
JP2017519766A5 JP2016573805A JP2016573805A JP2017519766A5 JP 2017519766 A5 JP2017519766 A5 JP 2017519766A5 JP 2016573805 A JP2016573805 A JP 2016573805A JP 2016573805 A JP2016573805 A JP 2016573805A JP 2017519766 A5 JP2017519766 A5 JP 2017519766A5
Authority
JP
Japan
Prior art keywords
seq
pharmaceutical composition
item
acid polymer
polynucleic acid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
JP2016573805A
Other languages
English (en)
Japanese (ja)
Other versions
JP2017519766A (ja
JP7043169B2 (ja
Filing date
Publication date
Priority claimed from GBGB1410693.4A external-priority patent/GB201410693D0/en
Application filed filed Critical
Publication of JP2017519766A publication Critical patent/JP2017519766A/ja
Publication of JP2017519766A5 publication Critical patent/JP2017519766A5/ja
Priority to JP2020131686A priority Critical patent/JP7532136B2/ja
Application granted granted Critical
Publication of JP7043169B2 publication Critical patent/JP7043169B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2016573805A 2014-06-16 2015-06-16 イントロン保持の減少 Active JP7043169B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2020131686A JP7532136B2 (ja) 2014-06-16 2020-08-03 イントロン保持の減少

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1410693.4A GB201410693D0 (en) 2014-06-16 2014-06-16 Splicing modulation
GB1410693.4 2014-06-16
PCT/GB2015/051756 WO2015193651A1 (en) 2014-06-16 2015-06-16 Reducing intron retention

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2020131686A Division JP7532136B2 (ja) 2014-06-16 2020-08-03 イントロン保持の減少

Publications (3)

Publication Number Publication Date
JP2017519766A JP2017519766A (ja) 2017-07-20
JP2017519766A5 true JP2017519766A5 (cg-RX-API-DMAC7.html) 2018-08-02
JP7043169B2 JP7043169B2 (ja) 2022-03-29

Family

ID=51266659

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2016573805A Active JP7043169B2 (ja) 2014-06-16 2015-06-16 イントロン保持の減少
JP2020131686A Active JP7532136B2 (ja) 2014-06-16 2020-08-03 イントロン保持の減少
JP2022178219A Pending JP2023011885A (ja) 2014-06-16 2022-11-07 イントロン保持の減少

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2020131686A Active JP7532136B2 (ja) 2014-06-16 2020-08-03 イントロン保持の減少
JP2022178219A Pending JP2023011885A (ja) 2014-06-16 2022-11-07 イントロン保持の減少

Country Status (12)

Country Link
US (6) US9745577B2 (cg-RX-API-DMAC7.html)
EP (2) EP4015648B1 (cg-RX-API-DMAC7.html)
JP (3) JP7043169B2 (cg-RX-API-DMAC7.html)
KR (1) KR102638276B1 (cg-RX-API-DMAC7.html)
CN (1) CN107073137A (cg-RX-API-DMAC7.html)
AU (2) AU2015275870B2 (cg-RX-API-DMAC7.html)
CA (1) CA2951208A1 (cg-RX-API-DMAC7.html)
ES (1) ES2907033T3 (cg-RX-API-DMAC7.html)
GB (1) GB201410693D0 (cg-RX-API-DMAC7.html)
IL (2) IL295051B2 (cg-RX-API-DMAC7.html)
SG (2) SG10202112949PA (cg-RX-API-DMAC7.html)
WO (1) WO2015193651A1 (cg-RX-API-DMAC7.html)

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201410693D0 (en) * 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
KR102620328B1 (ko) * 2014-10-03 2024-01-02 콜드스프링하버러보러토리 핵 유전자 산출량의 표적화 증강
KR20220105174A (ko) 2015-10-09 2022-07-26 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
MX391850B (es) 2015-12-10 2025-03-19 Ptc Therapeutics Inc Compuestos para usarse en el tratamiento o mejoramiento de la enfermedad de huntington
WO2017106370A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of eye diseases
CN109312343B (zh) 2015-12-14 2022-09-27 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
CA3005131A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of tuberous sclerosis complex
EP3485015A4 (en) * 2016-07-15 2020-07-29 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS OF MODULATION OF DYSTROPHINE TRANSCRIPT
WO2018014043A1 (en) * 2016-07-15 2018-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of transcript processing
SG11201911615WA (en) 2017-06-05 2020-01-30 Ptc Therapeutics Inc Compounds for treating huntington's disease
WO2019005980A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. METHODS OF TREATING HUNTINGTON'S DISEASE
MX2019015580A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
CN111499615B (zh) 2017-08-04 2024-02-02 斯基霍克疗法公司 用于调节剪接的方法和组合物
SMT202400071T1 (it) 2017-08-25 2024-03-13 Stoke Therapeutics Inc Oligomeri antisenso per il trattamento di condizioni e malattie
WO2019111791A1 (ja) * 2017-12-07 2019-06-13 第一三共株式会社 ジストロフィン遺伝子のイントロンリテンションを解消するアンチセンスオリゴヌクレオチド
AU2019243048A1 (en) 2018-03-27 2020-10-15 Ptc Therapeutics, Inc. Compounds for treating Huntington's disease
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
BR112020026534A2 (pt) 2018-06-27 2021-03-23 Ptc Therapeutics, Inc. Compostos de heteroarila para o tratamento da doença de huntington
MX2020014098A (es) 2018-06-27 2021-05-27 Ptc Therapeutics Inc Compuestos heterocíclicos y de heteroarilo para tratar la enfermedad de huntington.
EP3840733A4 (en) 2018-08-20 2022-07-20 Rogcon, Inc. ANTISENSE OLIGONUCLEOTIDES DIRECTED TO SCN2A FOR THE TREATMENT OF SCN1A ENCEPHALOPATHIES
EP3920915A4 (en) 2019-02-05 2022-10-05 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
JP7603595B2 (ja) 2019-02-06 2024-12-20 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
CN110718265B (zh) * 2019-09-05 2021-02-26 复旦大学 靶向生物毒素的g-四联体式核酸适配体三级结构预测方法
TW202134438A (zh) * 2019-12-02 2021-09-16 日商第一三共股份有限公司 著色性乾皮症f群組治療藥
WO2021113755A2 (en) * 2019-12-04 2021-06-10 Pai Athma A Anti-slc6a1 oligonucleotides and related methods
AU2021225957A1 (en) 2020-02-28 2022-09-08 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating SMN2
CA3173647A1 (en) 2020-05-11 2021-11-18 Isabel AZNAREZ Opa1 antisense oligomers for treatment of conditions and diseases
EP4370680A4 (en) 2021-07-16 2025-08-13 Aptah Bio Inc POLYNUCLEOTIDE COMPOSITIONS AND METHODS FOR REGULATING GENE EXPRESSION
EP4652278A1 (en) * 2023-01-18 2025-11-26 Aptah Bio, Inc. Polynucleotide compositions and methods for treatment of cancer
CN121127586A (zh) * 2023-01-18 2025-12-12 阿普塔生物公司 用于治疗神经退行性疾病的多核苷酸组合物和方法
WO2024235283A1 (en) * 2023-05-16 2024-11-21 Peking University Method of modulating rna splicing

Family Cites Families (254)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4476116A (en) 1982-12-10 1984-10-09 Syntex (U.S.A.) Inc. Polypeptides/chelating agent nasal compositions having enhanced peptide absorption
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
AU5698186A (en) 1985-03-15 1986-10-13 Summerton, J. Polynucleotide assay reagent and method
US4866042A (en) 1987-11-18 1989-09-12 Neuwelt Edward A Method for the delivery of genetic material across the blood brain barrier
US6294520B1 (en) 1989-03-27 2001-09-25 Albert T. Naito Material for passage through the blood-brain barrier
US5914396A (en) 1990-01-11 1999-06-22 Isis Pharmaceuticals, Inc. 2'-O-modified nucleosides and phosphoramidites
US7101993B1 (en) 1990-01-11 2006-09-05 Isis Pharmaceuticals, Inc. Oligonucleotides containing 2′-O-modified purines
US5151510A (en) 1990-04-20 1992-09-29 Applied Biosystems, Inc. Method of synethesizing sulfurized oligonucleotide analogs
DE69133511T2 (de) 1990-08-13 2006-09-28 Isis Pharmaceuticals, Inc., Carlsbad Zuckermodifizierte oligonukleotide zum erkennen und steuern der genexpression
US7015315B1 (en) 1991-12-24 2006-03-21 Isis Pharmaceuticals, Inc. Gapped oligonucleotides
DE69333842T2 (de) 1992-07-23 2006-04-27 Isis Pharmaceuticals, Inc., Carlsbad Neue 2'-O-Alkyl-Nukleoside und -Phosphoramidite, Verfahren zu ihrer Herstellung und ihre Verwendungen
AU692423B2 (en) 1992-09-25 1998-06-11 Institut National De La Sante Et De La Recherche Medicale Adenovirus vectors for the transfer of foreign genes into cells of the central nervous system, particularly in brain
EP0698092B1 (en) 1993-05-11 2007-07-25 The University Of North Carolina At Chapel Hill Antisense oligonucleotides which combat aberrant splicing and methods of using the same
EP0736094A1 (en) 1993-12-24 1996-10-09 Medical Research Council Polycystic kidney disease 1 gene and uses thereof
US5656612A (en) 1994-05-31 1997-08-12 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of raf gene expression
FR2727867B1 (fr) 1994-12-13 1997-01-31 Rhone Poulenc Rorer Sa Transfert de genes dans les motoneurones medullaires au moyen de vecteurs adenoviraux
US6166197A (en) 1995-03-06 2000-12-26 Isis Pharmaceuticals, Inc. Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions
CA2246229C (en) 1996-02-14 2011-08-23 Isis Pharmaceuticals Inc. Sugar-modified gapped oligonucleotides
GB9605808D0 (en) 1996-03-20 1996-05-22 Isis Innovation CFTR gene regulator
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
USRE44779E1 (en) 1997-03-07 2014-02-25 Santaris Pharma A/S Bicyclonucleoside and oligonucleotide analogues
ATE312935T1 (de) 1997-06-03 2005-12-15 Regulatorische sequenzen für die in-vivo- expression einer heterologen dns-sequenz in endothelzellen und ihre verwendungen.
US6963589B1 (en) 1997-07-03 2005-11-08 Canon Kabushiki Kaisha Information processing apparatus for and method of transmitting and/or receiving broadcast signal
US6391452B1 (en) 1997-07-18 2002-05-21 Bayer Corporation Compositions for nasal drug delivery, methods of making same, and methods of removing residual solvent from pharmaceutical preparations
GB9716231D0 (en) 1997-07-31 1997-10-08 Amersham Int Ltd Base analogues
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ/エス オリゴヌクレオチド類似体
US7045610B2 (en) 1998-04-03 2006-05-16 Epoch Biosciences, Inc. Modified oligonucleotides for mismatch discrimination
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
US7071324B2 (en) 1998-10-13 2006-07-04 Brown University Research Foundation Systems and methods for sequencing by hybridization
US20030224514A1 (en) 2002-05-31 2003-12-04 Isis Pharmaceuticals Inc. Antisense modulation of PPAR-delta expression
US6436657B1 (en) 1998-12-18 2002-08-20 E. I. Du Pont De Nemours And Company Polynucleotides encoding aminomethyltransferases
ATE287897T2 (de) 1999-02-12 2005-02-15 Sankyo Co Analoga von nukleosiden und oligonukleotiden
EP1161439B1 (en) 1999-03-18 2010-04-21 Exiqon A/S Xylo-lna analogues
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US6734291B2 (en) 1999-03-24 2004-05-11 Exiqon A/S Synthesis of [2.2.1]bicyclo nucleosides
AU3418800A (en) 1999-03-24 2000-10-09 Exiqon A/S Improved synthesis of (2.2.1)bicyclo nucleosides
US6383752B1 (en) 1999-03-31 2002-05-07 Hybridon, Inc. Pseudo-cyclic oligonucleobases
CN102180924A (zh) 1999-05-04 2011-09-14 桑塔里斯制药公司 L-核糖-lna类似物
US6083482A (en) 1999-05-11 2000-07-04 Icn Pharmaceuticals, Inc. Conformationally locked nucleosides and oligonucleotides
US6531591B1 (en) 1999-07-07 2003-03-11 Exiqon A/S Synthesis of stable quinone and photoreactive ketone phosphoramidite reagents for solid phase synthesis of photoreactive-oligomer conjugates
JP4151751B2 (ja) 1999-07-22 2008-09-17 第一三共株式会社 新規ビシクロヌクレオシド類縁体
US6677445B1 (en) 1999-08-27 2004-01-13 Chiron Corporation Chimeric antisense oligonucleotides and cell transfecting formulations thereof
US6187586B1 (en) 1999-12-29 2001-02-13 Isis Pharmaceuticals, Inc. Antisense modulation of AKT-3 expression
KR20020097241A (ko) 2000-05-04 2002-12-31 에이브이아이 바이오파마 인코포레이티드 스플라이스-영역 안티센스 조성물 및 방법
US6809194B1 (en) 2000-05-10 2004-10-26 Chiron Corporation Akt3 inhibitors
CA2697031C (en) 2000-07-13 2017-10-31 The Johns Hopkins University School Of Medicine Detection and treatment of polycystic kidney disease
AU2001282522A1 (en) 2000-08-29 2002-03-13 Takeshi Imanishi Novel nucleoside analogs and oligonucleotide derivatives containing these analogs
HUP0301805A3 (en) 2000-09-02 2005-12-28 Gruenenthal Gmbh Antisense oligonucleotides against vanilloid receptor 1
AU2001293687A1 (en) 2000-10-04 2002-04-15 Cureon A/S Improved synthesis of purine locked nucleic acid analogues
JP2002345489A (ja) 2000-11-03 2002-12-03 Astrazeneca Ab 化学物質
DK1409497T3 (da) 2001-07-12 2005-05-30 Santaris Pharma As Fremgangsmåde til fremstilling af LNA-phosphoramiditer
WO2003016492A2 (en) 2001-08-16 2003-02-27 The Regents Of The University Of Michigan Adamts13 genes and proteins and variants, and uses thereof
US7060809B2 (en) 2001-09-04 2006-06-13 Exiqon A/S LNA compositions and uses thereof
US6936589B2 (en) 2001-09-28 2005-08-30 Albert T. Naito Parenteral delivery systems
JP2005508196A (ja) 2001-11-07 2005-03-31 アプレラ コーポレイション 核酸分析の汎用ヌクレオチド
US7615619B2 (en) 2002-02-13 2009-11-10 Takeshi Imanishi Nucleoside analogues and oligonucleotide derivative comprising nucleotide analogue thereof
AU2003222743B2 (en) 2002-05-08 2008-12-11 Roche Innovation Center Copenhagen A/S Synthesis of locked nucleic acid derivatives
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
WO2004001010A2 (en) 2002-06-21 2003-12-31 Ptc Therapeutics, Inc. METHODS FOR IDENTIFYING SMALL MOLECULES THAT MODULATE PREMATURE TRANSLATION TERMINATION AND NONSENSE MEDIATED mRNA DECAY
US20040023220A1 (en) 2002-07-23 2004-02-05 Lawrence Greenfield Integrated method for PCR cleanup and oligonucleotide removal
US20060058253A1 (en) 2002-08-12 2006-03-16 Benoit Chabot Methods to reprogram splice site selection in pre-messenger rnas
WO2004014222A2 (en) 2002-08-12 2004-02-19 The Regents Of The University Of Michigan Diagnosis and treatment of tuberous sclerosis
EP1562971B1 (en) 2002-11-05 2014-02-12 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US8604183B2 (en) 2002-11-05 2013-12-10 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
ATE442152T1 (de) 2002-11-18 2009-09-15 Santaris Pharma As Antisense-entwurf
US7217807B2 (en) * 2002-11-26 2007-05-15 Rosetta Genomics Ltd Bioinformatically detectable group of novel HIV regulatory genes and uses thereof
ATE479752T1 (de) 2003-03-07 2010-09-15 Alnylam Pharmaceuticals Inc Therapeutische zusammensetzungen
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
EP2141234B1 (en) 2003-03-21 2016-04-27 Roche Innovation Center Copenhagen A/S Short Interfering RNA (siRNA) Analogues
KR20060015505A (ko) 2003-04-13 2006-02-17 엔존 파마슈티컬즈, 인코포레이티드 올리고뉴클레오타이드 전구약물
US20050053981A1 (en) 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
US20070009899A1 (en) * 2003-10-02 2007-01-11 Mounts William M Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases
GB0326578D0 (en) 2003-11-14 2003-12-17 Univ Belfast Cancer diagnosis and therapy
US20050221354A1 (en) 2004-02-18 2005-10-06 Wyeth Nucleic acid arrays for monitoring expression profiles of drug target genes
WO2005111211A2 (en) 2004-05-14 2005-11-24 Rosetta Genomics Ltd. Micronas and uses thereof
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
US20070087376A1 (en) 2004-08-30 2007-04-19 Potashkin Judith A Splice variants of pre-mRNA transcripts as biomarkers in idiopathic neurodegenerative diseases
EP1812798A2 (en) 2004-11-19 2007-08-01 Acadia Pharmaceuticals Inc. Enabling tools to identify ligands for hormone nuclear receptors
WO2006081311A2 (en) 2005-01-26 2006-08-03 Medical College Of Georgia Research Institute Compositions and methods for the intracellular disruption of vegf and vegfr-2 by intraceptors
US20100150839A1 (en) 2005-02-04 2010-06-17 Massachusetts Institute Of Technology Compositions and Methods for Modulating Cognitive Function
WO2006107846A2 (en) 2005-04-01 2006-10-12 University Of Florida Research Foundation, Inc. Biomakers of liver injury
US7557198B2 (en) * 2005-05-04 2009-07-07 Exagen Diagnostics, Inc. Acute myelogenous leukemia biomarkers
AU2006257073B2 (en) 2005-06-17 2011-08-04 Children's Medical Center Corporation ADAMTS13-comprising compositions having thrombolytic activity
PT3308788T (pt) 2005-06-23 2019-01-30 Cold Spring Harbor Laboratory Composições e métodos para a modulação do splicing de smn2
EP4174179B1 (en) 2005-08-23 2025-05-07 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
WO2007028065A2 (en) 2005-08-30 2007-03-08 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
US8258109B2 (en) 2005-10-20 2012-09-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of LMNA expression
US20080280848A1 (en) 2005-10-28 2008-11-13 Volker Patzel Structures of Active Guide Rna Molecules and Method of Selection
WO2007048629A2 (en) 2005-10-28 2007-05-03 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Modulation of rna silencing efficiency by argonaute proteins
EP1954254A4 (en) 2005-11-01 2010-12-22 Harvard College MODULATION OF THE LOADING OF THE ENDOPLASMIC RETICULUM IN THE TREATMENT OF TUBEROUS SCLEROSIS
US7785834B2 (en) 2005-11-10 2010-08-31 Ercole Biotech, Inc. Soluble TNF receptors and their use in treatment of disease
EP1792981A1 (en) 2005-12-02 2007-06-06 Humboldt-Universität zu Berlin Microginin producing proteins and nucleic acids encoding a microginin gene cluster as well as methods for creating microginins
PT2161038E (pt) 2006-01-26 2014-03-10 Isis Pharmaceuticals Inc Composições e suas utilizações dirigidas à huntingtina
CN102908630B (zh) 2006-01-27 2014-11-19 Isis制药公司 6-修饰的双环核酸类似物
EP1984499B1 (en) 2006-01-27 2015-05-27 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
US8007790B2 (en) 2006-04-03 2011-08-30 Stowers Institute For Medical Research Methods for treating polycystic kidney disease (PKD) or other cyst forming diseases
EP2015758B1 (en) 2006-05-05 2014-04-02 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression apob
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
EP2066684B1 (en) 2006-05-11 2012-07-18 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
CN101460624B (zh) 2006-06-08 2013-06-05 阿明诺化学株式会社 具有iNOS的表达控制作用的正义寡核苷酸以及含有其的组合物
WO2008001778A1 (fr) 2006-06-27 2008-01-03 Takeda Pharmaceutical Company Limited Animal génétiquement modifié et son utilisation
CA2658421C (en) 2006-07-24 2018-03-20 Athena Diagnostics, Inc. Pkd mutations and evaluation of same
ES2620472T5 (es) 2006-10-18 2020-07-09 Ionis Pharmaceuticals Inc Compuestos antisentido
US20090264353A1 (en) 2007-10-19 2009-10-22 Santaris Pharma A/S Splice Switching Oligomers for TNF Superfamily Receptors and their Use in Treatment of Disease
DK2099461T3 (da) 2006-11-13 2012-07-02 Santaris Pharma As LNA Nukleoside Phosphoramidates
US8293684B2 (en) 2006-11-29 2012-10-23 Exiqon Locked nucleic acid reagents for labelling nucleic acids
EP2118118B1 (en) 2007-01-19 2017-09-27 Exiqon A/S Mediated cellular delivery of lna oligonucleotides
US20100093836A1 (en) 2007-01-29 2010-04-15 Isis Pharmaceuticals, Inc Compounds and methods for modulating protein expression
WO2008111485A1 (ja) 2007-03-09 2008-09-18 Riken モノヌクレオシドまたはモノヌクレオチドから誘導される構造を有する化合物、核酸、標識物質、核酸検出方法およびキット
AR065648A1 (es) 2007-03-09 2009-06-24 Pioneer Hi Bred Int Identificacion de transportadores de amonio (amts) para la regulacion del metabolismo de nitrogeno en plantas
WO2008111908A1 (en) 2007-03-15 2008-09-18 Jyoti Chattopadhyaya Five- and six-membered conformationally locked 2',4'- carbocyclic ribo-thymidines for the treatment of infections and cancer
EP2149605B1 (en) 2007-03-22 2013-07-03 Santaris Pharma A/S Short RNA antagonist compounds for the modulation of target mRNA
ES2388590T3 (es) 2007-05-30 2012-10-16 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos con puente aminometileno N-sustituido.
WO2008154401A2 (en) 2007-06-08 2008-12-18 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
WO2009006478A2 (en) 2007-07-05 2009-01-08 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
KR101654007B1 (ko) 2007-08-15 2016-09-05 아이오니스 파마수티컬즈, 인코포레이티드 테트라하이드로피란 핵산 유사체
ES2639852T3 (es) 2007-10-26 2017-10-30 Academisch Ziekenhuis Leiden Medios y métodos para contrarrestar los trastornos musculares
WO2009064920A2 (en) 2007-11-13 2009-05-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
CA2910760C (en) 2007-12-04 2019-07-09 Muthiah Manoharan Targeting lipids
US8846386B2 (en) 2007-12-18 2014-09-30 University Of Kentucky Research Foundation sVEGFR-2 and its role in lymphangiogenesis modulation
JP5608863B2 (ja) 2007-12-28 2014-10-15 公立大学法人横浜市立大学 新生児期〜乳児期発症の難治性てんかんの検出方法
WO2009099942A2 (en) 2008-01-31 2009-08-13 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides and uses thereof
US8530640B2 (en) 2008-02-07 2013-09-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
EP2276855B1 (en) 2008-03-13 2016-05-11 Celera Corporation Genetic polymorphisms associated wiith venous thrombosis, methods of detection and uses thereof
EP2274423A2 (en) 2008-04-04 2011-01-19 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
DK2285819T3 (da) 2008-04-04 2013-12-02 Isis Pharmaceuticals Inc Oligomere forbindelser omfattende neutralt bundne, terminale bicykliske nukleosider
EP2119783A1 (en) * 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
US8710021B2 (en) 2008-06-11 2014-04-29 Bionucleon S.R.L. Inhibition of HRP-3 using modified oligonucleotides
EP2151248A1 (en) 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
US8501805B2 (en) 2008-09-24 2013-08-06 Isis Pharmaceuticals, Inc. Substituted alpha-L-bicyclic nucleosides
WO2010051632A1 (en) 2008-11-07 2010-05-14 Centre Hospitalier Universitaire Sainte-Justine Syngap1 dysfunctions and uses thereof in diagnostic and therapeutic applications for mental retardation
EP2370581B1 (en) 2008-12-04 2016-08-03 CuRNA, Inc. Treatment of vascular endothelial growth factor (vegf) related diseases by inhibition of natural antisense transcript to vegf
US9006194B2 (en) 2008-12-19 2015-04-14 Drexel University Compositions and methods for diminishing viral infection and inflammation associated with viral infection
US20100166784A1 (en) * 2008-12-30 2010-07-01 The Washington University Method and compositions for modulating th17 cell development
US8680254B2 (en) 2009-01-14 2014-03-25 Philadelphia Health & Education Corporation Modulation of pre-mRNA using splice modulating oligonucleotides as therapeutic agents in the treatment of disease
CN102459597B (zh) 2009-05-08 2020-06-30 库尔纳公司 通过针对dmd家族的天然反义转录物的抑制治疗肌营养蛋白家族相关疾病
AU2010262862C1 (en) 2009-06-17 2020-04-30 Biogen Ma Inc. Compositions and methods for modulation of SMN2 splicing in a subject
EP2275545A1 (en) 2009-07-16 2011-01-19 Julius-Maximilians-Universität Würzburg Use of microRNA-24 and/or its targets for the treatment and prevention of ischemia and induction of angiogenesis
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
WO2011020118A1 (en) 2009-08-14 2011-02-17 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex
EP3135772B1 (en) 2009-09-08 2019-10-23 Laboratory Corporation of America Holdings Method for diagnosing autism spectrum disorders
WO2011032034A2 (en) 2009-09-10 2011-03-17 University Of Idaho Nucleobase-functionalized conformationally restricted nucleotides and oligonucleotides for targeting nucleic acids
JPWO2011052436A1 (ja) 2009-10-29 2013-03-21 国立大学法人大阪大学 架橋型人工ヌクレオシドおよびヌクレオチド
PL2499249T3 (pl) * 2009-11-12 2019-03-29 Univ Western Australia Cząsteczki antysensowne i sposoby leczenia patologii
US9334495B2 (en) 2009-11-25 2016-05-10 Elitechgroup B.V. Minor groove binder (MGB)-oligonucleotide miRNA antagonists
US8779118B2 (en) 2010-01-11 2014-07-15 Isis Pharmaceuticals, Inc. Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom
EP3321361B1 (en) 2010-02-08 2019-03-27 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
US9193752B2 (en) 2010-03-17 2015-11-24 Isis Pharmaceuticals, Inc. 5′-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
EP2550361B1 (en) 2010-03-25 2017-02-08 The J. David Gladstone Institutes Compositions and methods for treating neurological disorders
WO2011127210A1 (en) 2010-04-06 2011-10-13 Massachusetts Institute Of Technology Targeted delivery of nucleic acids
US20110269735A1 (en) 2010-04-19 2011-11-03 Celera Corporation Genetic polymorphisms associated with statin response and cardiovascular diseases, methods of detection and uses thereof
KR20130103662A (ko) 2010-04-19 2013-09-24 엔라이프 떼라퓨틱스, 에스.엘. 올리고뉴클레오티드 분자들의 특정 뉴런 유형들로의 선택적인 전달을 위한 조성물들 및 방법
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
EP2606057B1 (en) 2010-04-28 2016-06-15 Ionis Pharmaceuticals, Inc. Modified 5' diphosphate nucleosides and oligomeric compounds prepared therefrom
CA2799944A1 (en) 2010-05-20 2011-11-24 University Of Rochester Methods and compositions related to modulating autophagy
US8957200B2 (en) 2010-06-07 2015-02-17 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011156202A1 (en) 2010-06-08 2011-12-15 Isis Pharmaceuticals, Inc. Substituted 2 '-amino and 2 '-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
US9518259B2 (en) 2010-06-15 2016-12-13 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating interaction between proteins and target nucleic acids
PL2585596T3 (pl) 2010-06-23 2021-06-28 Curna, Inc. Leczenie chorób związanych z podjednostką alfa kanału sodowego bramkowanego napięciem (SCNA) poprzez hamowanie naturalnego transkryptu antysensownego SCNA
WO2012054723A2 (en) 2010-10-22 2012-04-26 Opko Curna Llc Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
WO2012106529A1 (en) 2011-02-02 2012-08-09 The Trustees Of Princeton University Jagged1 as a marker and therapeutic target for breast cancer bone metastasis
WO2012138487A2 (en) 2011-04-07 2012-10-11 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
US9644035B2 (en) 2011-04-08 2017-05-09 Omeros Corporation Methods for treating conditions associated with MASP-2 dependent complement activation
US9957558B2 (en) 2011-04-28 2018-05-01 Life Technologies Corporation Methods and compositions for multiplex PCR
US20140357558A1 (en) 2011-06-24 2014-12-04 Cold Spring Harbor Laboratory Compositions and methods for treatment of spinal muscular atrophy
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
US8592156B2 (en) 2011-08-08 2013-11-26 Roche Molecular Systems, Inc. Predicting response to anti-CD20 therapy in DLBCL patients
EP3640332A1 (en) 2011-08-29 2020-04-22 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
WO2013036403A1 (en) 2011-09-06 2013-03-14 Curna, Inc. TREATMENT OF DISEASES RELATED TO ALPHA SUBUNITS OF SODIUM CHANNELS, VOLTAGE-GATED (SCNxA) WITH SMALL MOLECULES
US9453261B2 (en) 2011-09-20 2016-09-27 The George Washington University Alternative splicing variants of genes associated with prostate cancer risk and survival
BR112014011018A2 (pt) 2011-11-11 2017-05-02 Santaris Pharma As compostos para modulação de splining de smn2
US9534222B2 (en) 2011-11-15 2017-01-03 University Of Utah Research Foundation Morpholinos, morpholino upregulating, and associated methods
CN104053786B (zh) 2011-11-29 2017-06-06 生命技术公司 用于多重pcr的方法和组合物
US20150025231A1 (en) 2012-01-11 2015-01-22 Cold Spring Harbor Laboratory Compositions and methods for modulation of ikbkap splicing
EP2812342B1 (en) 2012-02-08 2017-11-15 Ionis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
BR112014019750B1 (pt) 2012-02-10 2020-03-03 F. Hoffmann-La Roche Ag. Composto, composição farmacêutica e seus usos
US8846885B2 (en) 2012-02-17 2014-09-30 Ajinomoto Co., Inc. Oligonucleotide with protected base
US9221864B2 (en) 2012-04-09 2015-12-29 Isis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
WO2013159108A2 (en) 2012-04-20 2013-10-24 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
US20140378526A1 (en) 2012-05-11 2014-12-25 City Of Hope Design of nucleic acid binding molecules with non-watson crick and non-canonical pairing based on artificial mutation consensus sequences to counter escape mutations
WO2013177188A1 (en) 2012-05-22 2013-11-28 Idenix Pharmaceuticals, Inc. 3',5'-cyclic phosphoramidate prodrugs for hcv infection
WO2013177195A1 (en) 2012-05-22 2013-11-28 Idenix Pharmaceuticals, Inc. 3',5'-cyclic phosphate prodrugs for hcv infection
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
SG10201912895PA (en) 2012-07-13 2020-02-27 Wave Life Sciences Ltd Chiral control
US8729263B2 (en) 2012-08-13 2014-05-20 Novartis Ag 1,4-disubstituted pyridazine analogs there of and methods for treating SMN-deficiency-related conditions
CA2880869A1 (en) 2012-08-20 2014-02-27 The Regents Of The University Of California Polynucleotides having bioreversible groups
EP2898072A1 (en) 2012-09-24 2015-07-29 Yissum Research Development Company of the Hebrew University of Jerusalem Ltd. Restoration of the cftr function by splicing modulation
WO2014049536A2 (en) 2012-09-25 2014-04-03 Universidade De Lisboa Drug targets for cystic fibrosis and other conditions
US9192621B2 (en) 2012-09-27 2015-11-24 Idenix Pharmaceuticals Llc Esters and malonates of SATE prodrugs
UA116639C2 (uk) 2012-10-09 2018-04-25 Рег'Юлес Терап'Ютікс Інк. Способи лікування синдрому альпорта
US9029335B2 (en) 2012-10-16 2015-05-12 Isis Pharmaceuticals, Inc. Substituted 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
US20150291957A1 (en) 2012-10-26 2015-10-15 Larry J. Smith METHODS AND COMPOSITIONS TO PRODUCE ss-RNAi ACTIVITY WITH ENHANCED POTENCY
US9909128B2 (en) 2012-11-15 2018-03-06 The Regents Of The University Of California Splice modulating oligonucleotides that inhibit cancer
WO2014099941A1 (en) 2012-12-19 2014-06-26 Idenix Pharmaceuticals, Inc. 4'-fluoro nucleosides for the treatment of hcv
CN104004826B (zh) 2013-01-07 2016-03-02 赵晨 突变的基因prpf4在制备遗传性视网膜疾病诊断试剂中的应用
US20150361497A1 (en) 2013-01-18 2015-12-17 Anna Mary ROSE Therapeutics and diagnostics based on minisatellite repeat element 1 (msr1)
EP3778618A1 (en) 2013-02-04 2021-02-17 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
WO2014126229A1 (ja) 2013-02-18 2014-08-21 塩野義製薬株式会社 含窒素複素環構造を有するヌクレオシド及びヌクレオチド
WO2014137926A1 (en) 2013-03-04 2014-09-12 Idenix Pharmaceuticals, Inc. 3'-deoxy nucleosides for the treatment of hcv
US9339541B2 (en) 2013-03-04 2016-05-17 Merck Sharp & Dohme Corp. Thiophosphate nucleosides for the treatment of HCV
WO2014165542A1 (en) 2013-04-01 2014-10-09 Idenix Pharmaceuticals, Inc. 2',4'-fluoro nucleosides for the treatment of hcv
US10590412B2 (en) 2013-04-19 2020-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
KR102138781B1 (ko) 2013-05-01 2020-07-28 아이오니스 파마수티컬즈, 인코포레이티드 아포지질단백질 c-iii 발현을 조절하는 조성물 및 방법
WO2014198890A1 (en) 2013-06-13 2014-12-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for the treatment of erythropoietic protoporphyria
WO2014201413A1 (en) 2013-06-14 2014-12-18 Isis Pharmaceuticals, Inc. Compounds and methods for modulating non-coding rna
WO2014209841A2 (en) 2013-06-25 2014-12-31 F. Hoffmann-La Roche Ag Compounds for treating spinal muscular atrophy
AU2014306416B2 (en) 2013-08-16 2021-02-25 Translate Bio Ma, Inc. Compositions and methods for modulating RNA
WO2015023941A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Oligonucleotides targeting euchromatin regions of genes
EP3033423A4 (en) 2013-08-16 2017-04-26 Rana Therapeutics Inc. Epigenetic regulators of frataxin
WO2015023939A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Compositions and methods for modulating expression of frataxin
KR20160045063A (ko) 2013-08-19 2016-04-26 에프. 호프만-라 로슈 아게 스크리닝 방법
PL3041958T3 (pl) 2013-09-04 2020-11-02 Cold Spring Harbor Laboratory Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi
WO2015035231A1 (en) 2013-09-05 2015-03-12 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
EP3480312A1 (en) 2013-09-11 2019-05-08 Synthena AG Nucleic acids and methods for the treatment of pompe disease
AU2015272128B2 (en) 2014-06-10 2021-10-28 Erasmus University Medical Center Rotterdam Antisense oligonucleotides useful in treatment of Pompe disease
GB201410693D0 (en) * 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
GB201411468D0 (en) 2014-06-27 2014-08-13 Univ Edinburgh Novel treatment for endometriosis
GB2547586A (en) 2014-08-20 2017-08-23 Lifesplice Pharma Llc Splice modulating oligonucleotides and methods of use thereof(In the PCT request)
KR102620328B1 (ko) 2014-10-03 2024-01-02 콜드스프링하버러보러토리 핵 유전자 산출량의 표적화 증강
EP3207048A4 (en) 2014-10-17 2018-05-30 The Broad Institute, Inc. Compositions and methods of treating muscular dystrophy
WO2016077837A1 (en) 2014-11-14 2016-05-19 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of proteins
GB201421379D0 (en) 2014-12-02 2015-01-14 Isis Innovation Ltd And Medical Res Council Molecule
CN114642735A (zh) 2015-01-21 2022-06-21 菲泽尔克斯公司 用于将治疗剂和诊断剂递送到细胞中的方法、组合物和系统
JP6884102B2 (ja) 2015-02-09 2021-06-09 エフ・ホフマン−ラ・ロシュ・アクチェンゲゼルシャフト がんの治療のための化合物
US9840709B2 (en) 2015-02-20 2017-12-12 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting genes associated with cystic fibrosis
EP3262056A4 (en) 2015-02-27 2018-09-19 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
HK1249140A1 (zh) 2015-04-03 2018-10-26 Ionis Pharmaceuticals, Inc. 用於调节tmprss6表达的化合物和方法
EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
EP3352872A4 (en) 2015-09-24 2019-07-10 The Trustees of the University of Pennsylvania TRIPTYENE DERIVATIVES FOR STABILIZING A NUCLEIC ACID COMPOUND
GB2546719B (en) 2015-10-09 2021-07-14 Univ Southampton Modulation of gene expression and screening for deregulated protein expression
KR20220105174A (ko) 2015-10-09 2022-07-26 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
EP3183347A4 (en) 2015-10-17 2018-04-18 Lifesplice Pharma LLC Splice modulating oligonucleotides and methods of use thereof
CA3005090A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of liver diseases
CA3005247A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of polycystic kidney disease
CA3005246A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of central nervous system diseases
CA3005131A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of tuberous sclerosis complex
WO2017106292A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of kidney diseases
WO2017106370A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of eye diseases
ES2903394T3 (es) 2015-12-14 2022-04-01 Cold Spring Harbor Laboratory Composiciones para el tratamiento de la retinitis pigmentosa 13
CN109312343B (zh) 2015-12-14 2022-09-27 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
EP3389671A4 (en) 2015-12-14 2019-07-17 Cold Spring Harbor Laboratory ANTISENSE OLIGOMERS FOR THE TREATMENT OF ALAGILLE SYNDROME
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
CN117821509A (zh) 2017-04-03 2024-04-05 编码治疗公司 组织选择性转基因表达
SMT202400071T1 (it) 2017-08-25 2024-03-13 Stoke Therapeutics Inc Oligomeri antisenso per il trattamento di condizioni e malattie
DK3700570T3 (da) 2017-10-23 2025-03-31 Stoke Therapeutics Inc Antisense-oligomerer til behandling af ikke-sense medieret rna henfald baserede tilstande og sygdomme

Similar Documents

Publication Publication Date Title
JP2017519766A5 (cg-RX-API-DMAC7.html)
JP7420679B2 (ja) 状態および疾患の処置のためのアンチセンスオリゴマー
WO2015193651A4 (en) Reducing intron retention
JP2022062143A (ja) 腎臓病の処置のための組成物と方法
US20090082297A1 (en) Compositions and Methods for Regulating Gene Expression
JP2019500347A5 (cg-RX-API-DMAC7.html)
JP2019500347A (ja) 網膜色素変性症18と網膜色素変性症13の処置のための組成物と方法
EP3389671A1 (en) Antisense oligomers for treatment of alagille syndrome
JP2011510678A (ja) Dna反復不安定性関連遺伝性障害を治療するための方法及び手段
JP2019500350A (ja) 結節性硬化症の処置のためのアンチセンスオリゴマー
EP3353301A1 (en) Antisense oligonucleotides and uses thereof
AU2018375807A1 (en) Angelman syndrome antisense treatment
WO2017192662A2 (en) Methods for identifying treatment targets based on multiomics data
EP4058152A1 (en) Oligonucleotide interference treatments of prostate cancer
JP2021523227A (ja) コレステリルエステル蓄積症の処置のための方法及び組成物
WO2020136125A2 (en) Antisense oligonucleotides targeting card9
US20220162599A1 (en) Methods and compositions for modulating splicing of alternative introns
WO2021231211A1 (en) Complement component c1s inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
JP2023526096A (ja) Card9のスプライス調節のためのオリゴヌクレオチド
TWI901675B (zh) 用於病症及疾病之治療的opa1反義寡聚物
US20250179497A1 (en) Use of dinucleotide repeat rnas to treat cancer
Gulam et al. Antisense therapy and its application in biopharmaceutical research: past, present and future
WO2025096087A1 (en) Treatment of myeloid cell dysfunction with membrane spanning 4-domains a6a (ms4a6a) inhibitors
WO2026003375A1 (en) Antisense oligonucleotides and their uses
WO2023196944A2 (en) Oligonucleotide interference treatments of prostate cancer