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DK3673080T3 - Antisense-oligomerer til behandling af lidelser og sygdomme - Google Patents

Antisense-oligomerer til behandling af lidelser og sygdomme Download PDF

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Publication number
DK3673080T3
DK3673080T3 DK18848036.2T DK18848036T DK3673080T3 DK 3673080 T3 DK3673080 T3 DK 3673080T3 DK 18848036 T DK18848036 T DK 18848036T DK 3673080 T3 DK3673080 T3 DK 3673080T3
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Denmark
Prior art keywords
disorders
diseases
treatment
antisense oligomers
antisense
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Application number
DK18848036.2T
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English (en)
Inventor
Isabel Aznarez
Zhou Han
Original Assignee
Stoke Therapeutics Inc
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Publication of DK3673080T3 publication Critical patent/DK3673080T3/da

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
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    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P25/06—Antimigraine agents
    • A—HUMAN NECESSITIES
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    • A61P25/08—Antiepileptics; Anticonvulsants
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    • A61P25/08—Antiepileptics; Anticonvulsants
    • A61P25/12—Antiepileptics; Anticonvulsants for grand-mal
    • A—HUMAN NECESSITIES
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    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
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DK18848036.2T 2017-08-25 2018-08-24 Antisense-oligomerer til behandling af lidelser og sygdomme DK3673080T3 (da)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762550462P 2017-08-25 2017-08-25
US201762575901P 2017-10-23 2017-10-23
US201862667356P 2018-05-04 2018-05-04
US201862671745P 2018-05-15 2018-05-15
PCT/US2018/048031 WO2019040923A1 (en) 2017-08-25 2018-08-24 ANTISENSE OLIGOMERS FOR THE TREATMENT OF DISEASE CONDITIONS AND OTHER DISEASES

Publications (1)

Publication Number Publication Date
DK3673080T3 true DK3673080T3 (da) 2023-11-27

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DK18848036.2T DK3673080T3 (da) 2017-08-25 2018-08-24 Antisense-oligomerer til behandling af lidelser og sygdomme

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US (4) US10913947B2 (da)
EP (2) EP3673080B1 (da)
JP (3) JP6827148B2 (da)
KR (3) KR20240035631A (da)
CN (2) CN114645048A (da)
AU (3) AU2018322319B2 (da)
CA (1) CA3073515A1 (da)
DK (1) DK3673080T3 (da)
ES (1) ES2965696T3 (da)
FI (1) FI3673080T3 (da)
GB (3) GB2582457B (da)
HR (1) HRP20231637T1 (da)
HU (1) HUE064635T2 (da)
IL (1) IL272761A (da)
LT (1) LT3673080T (da)
MX (2) MX380890B (da)
PL (1) PL3673080T3 (da)
PT (1) PT3673080T (da)
RS (1) RS65031B1 (da)
SG (2) SG10202108375XA (da)
SI (1) SI3673080T1 (da)
SM (1) SMT202400071T1 (da)
WO (1) WO2019040923A1 (da)

Families Citing this family (26)

* 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
EP3201339A4 (en) 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
EP3359685A1 (en) 2015-10-09 2018-08-15 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
SG11201804443UA (en) 2015-12-14 2018-06-28 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
FI3673080T3 (fi) 2017-08-25 2023-11-23 Stoke Therapeutics Inc Antisense-oligomeerejä häiriötilojen ja sairauksien hoitoon
US11713463B2 (en) 2018-01-17 2023-08-01 The Florey Institute Of Neuroscience And Mental Health Compositions and methods for increasing expression of SCN2A
MX2020011695A (es) 2018-05-04 2021-02-26 Stoke Therapeutics Inc Métodos y composiciones para el tratamiento de la enfermedad por almacenamiento de éster de colesterilo.
US11939582B2 (en) 2018-08-20 2024-03-26 Rogcon, Inc. Antisense oligonucleotides targeting SCN2A for the treatment of SCN1A encephalopathies
US10851376B2 (en) * 2018-12-28 2020-12-01 The Florida International University Board Of Trustees Long noncoding RNAs in pulmonary airway inflammation
WO2020154462A1 (en) * 2019-01-23 2020-07-30 Rogcon U.R., Inc. Antisense oligonucleotides targeting scn2a retained introns
SG11202109371VA (en) * 2019-02-27 2021-09-29 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
JP2022544702A (ja) * 2019-08-19 2022-10-20 ストーク セラピューティクス，インク． スプライシングおよびタンパク質発現を調節するための組成物および方法
EP4031242A4 (en) 2019-09-19 2023-12-20 Arnay Sciences, Llc COMPOUNDS AND METHODS USEFUL FOR MODULATING GENE SPLICING
WO2021113541A1 (en) * 2019-12-06 2021-06-10 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
EP4110919A4 (en) * 2020-02-28 2024-06-05 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSION
KR20230022409A (ko) 2020-05-11 2023-02-15 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머
CA3204779A1 (en) * 2021-01-26 2022-08-04 Brage Storstein Andresen Splice switching oligonucleotides targeting pseudoexons
US20240360449A1 (en) * 2021-08-27 2024-10-31 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating scn1a expression
MX2024003412A (es) * 2021-09-21 2024-04-08 Univ Johns Hopkins Terapia de regulon de arnm para el tratamiento de los trastornos por haploinsuficiencia.
US20250101419A1 (en) * 2022-01-26 2025-03-27 Syddansk Universitet Allele specific splice switching oligonucleotides targeting pseudoexons
IL318661A (en) * 2022-07-29 2025-03-01 Stoke Therapeutics Inc Compounds for the treatment of conditions and diseases
CN116024222B (zh) * 2022-11-30 2023-12-22 湖南家辉生物技术有限公司 一种导致婴儿严重肌阵挛性癫痫的nac1基因突变体及其应用
WO2024229421A1 (en) * 2023-05-03 2024-11-07 Stoke Therapeutics, Inc. Compounds and methods for treating human subjects
WO2025024568A1 (en) * 2023-07-24 2025-01-30 Stoke Therapeutics, Inc. Methods for treating conditions and diseases

Family Cites Families (275)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US7101993B1 (en) 1990-01-11 2006-09-05 Isis Pharmaceuticals, Inc. Oligonucleotides containing 2′-O-modified purines
US5914396A (en) 1990-01-11 1999-06-22 Isis Pharmaceuticals, Inc. 2'-O-modified nucleosides and phosphoramidites
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
JP3015464B2 (ja) 1992-07-23 2000-03-06 アイシス・ファーマシューティカルス・インコーポレーテッド 新規２’−０−アルキルヌクレオシドおよびホスホロアミダイトの製造法およびその使用
EP0669987B1 (en) 1992-09-25 2008-08-13 Aventis Pharma S.A. Adenovirus vectors for the transfer of foreign genes into cells of the central nervous system, particularly in brain
EP1897942A1 (en) 1993-05-11 2008-03-12 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
DE69729179T2 (de) 1996-02-14 2004-12-30 Isis Pharmaceuticals, Inc., Carlsbad Lückenhaft Zucker-modifizierte Oligonukleotide
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
USRE44779E1 (en) 1997-03-07 2014-02-25 Santaris Pharma A/S Bicyclonucleoside and oligonucleotide analogues
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
US7011973B1 (en) 1997-06-03 2006-03-14 Max-Planck-Gesellschaft Zur Foerderung Der Wissenschaften E.V. Regulatory sequences capable of conferring expression of a heterologous DNA sequence in endothelial cells in vivo and uses thereof
US6963589B1 (en) 1997-07-03 2005-11-08 Canon Kabushiki Kaisha Information processing apparatus for and method of transmitting and/or receiving broadcast signal
GB9716231D0 (en) 1997-07-31 1997-10-08 Amersham Int Ltd Base analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
DE04020014T1 (de) 1997-09-12 2006-01-26 Exiqon A/S Bi-zyklische - Nukleosid,Nnukleotid und Oligonukleotid-Analoga
US7045610B2 (en) 1998-04-03 2006-05-16 Epoch Biosciences, Inc. Modified oligonucleotides for mismatch discrimination
KR100608276B1 (ko) * 1998-08-24 2006-08-02 무라마쯔 다카시 동맥경화 및 ｐｔｃａ술후의 혈관 재협착에 대한예방·치료제
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
EP1152009B2 (en) 1999-02-12 2017-09-06 Daiichi Sankyo Company, Limited Novel nucleosides and oligonucleotide analogues
ATE465168T1 (de) 1999-03-18 2010-05-15 Exiqon As Xylo-lna analoge
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
DK1163250T3 (da) 1999-03-24 2006-11-13 Exiqon As Forbedret syntese af [2.2.1]bicyclonukleosider
US6734291B2 (en) 1999-03-24 2004-05-11 Exiqon A/S Synthesis of [2.2.1]bicyclo nucleosides
US6383752B1 (en) 1999-03-31 2002-05-07 Hybridon, Inc. Pseudo-cyclic oligonucleobases
PT1178999E (pt) 1999-05-04 2007-06-26 Santaris Pharma As Análogos de l-ribo-lna
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
WO2001083740A2 (en) 2000-05-04 2001-11-08 Avi Biopharma, Inc. Splice-region antisense composition and method
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
EP1314734A1 (en) 2000-08-29 2003-05-28 Takeshi Imanishi Novel nucleoside analogs and oligonucleotide derivatives containing these analogs
ATE385505T1 (de) 2000-09-02 2008-02-15 Gruenenthal Gmbh Antisense oligonukleotide gegen vr 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
EP1409497B1 (en) 2001-07-12 2005-01-19 Santaris Pharma A/S Method for preparation of lna phosphoramidites
WO2003016492A2 (en) 2001-08-16 2003-02-27 The Regents Of The University Of Michigan Adamts13 genes and proteins and variants, and uses thereof
CA2459347C (en) 2001-09-04 2012-10-09 Exiqon A/S Locked nucleic acid (lna) compositions and uses thereof
US6936589B2 (en) 2001-09-28 2005-08-30 Albert T. Naito Parenteral delivery systems
AU2002347981A1 (en) 2001-11-07 2003-05-19 Applera Corporation Universal nucleotides for nucleic acid analysis
US7615619B2 (en) 2002-02-13 2009-11-10 Takeshi Imanishi Nucleoside analogues and oligonucleotide derivative comprising nucleotide analogue thereof
ATE369375T1 (de) 2002-05-08 2007-08-15 Santaris Pharma As Synthese von locked nucleic acid-derivaten
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
CA2495085A1 (en) 2002-08-12 2004-02-19 The Regents Of The University Of Michigan Diagnosis and treatment of tuberous sclerosis
JP2005535318A (ja) 2002-08-12 2005-11-24 ユニヴェルシテ ドゥ シェルブルック プレメッセンジャーｒｎａのスプライス部位選択の再プログラミング方法
CA2504929C (en) 2002-11-05 2014-07-22 Charles Allerson Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
AU2003291753B2 (en) 2002-11-05 2010-07-08 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
DK2284269T3 (da) 2002-11-18 2017-10-23 Roche Innovation Ct Copenhagen As Antisense-design
US7217807B2 (en) 2002-11-26 2007-05-15 Rosetta Genomics Ltd Bioinformatically detectable group of novel HIV regulatory genes and uses thereof
US8110674B2 (en) 2003-03-07 2012-02-07 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
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
NZ542687A (en) 2003-04-13 2010-02-26 Enzon Pharmaceuticals Inc Polymeric oligonucleotide prodrugs
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
EP1755669A4 (en) * 2004-04-27 2008-06-25 Illumigen Biosciences Inc METHODS AND COMPOSITIONS FOR SPECIFIC TARGETING OF HEPATOCELLULAR CARCINOMA CELLS IN MAN
JP5697297B2 (ja) 2004-05-14 2015-04-08 ロゼッタ ジノミクス リミテッド マイクロｎａｓおよびその使用
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
US7718625B2 (en) 2005-01-27 2010-05-18 University Of South Florida Polynucleotides targeted against the extended 5′-UTR region of argininosuccinate synthase and uses thereof
WO2007094755A2 (en) 2005-02-04 2007-08-23 Massachusetts Institute Of Technology Compositions and methods for modulating cognitive function
US7645584B2 (en) 2005-04-01 2010-01-12 University Of Florida Research Foundation, Inc. Biomarkers of liver injury
JP2008546376A (ja) 2005-06-16 2008-12-25 バイオノミックス リミテッド Ｓｃｎ１ａ遺伝子における変異を検出することによっててんかんを診断および処置するための方法
CA2606351C (en) 2005-06-17 2016-12-13 Baxter International Inc. Adamts13-comprising compositions having thrombolytic activity
EP1910395B1 (en) 2005-06-23 2012-10-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of SMN2 splicing
EP4332227A1 (en) 2005-08-23 2024-03-06 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
WO2007047913A2 (en) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
WO2007048628A2 (en) 2005-10-28 2007-05-03 Max Planck Gesellschaft 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
US20090291437A1 (en) * 2005-11-02 2009-11-26 O'brien Sean Methods for targeting quadruplex sequences
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
EP2161038B1 (en) 2006-01-26 2013-12-25 Isis Pharmaceuticals, Inc. Compositions and their uses directed to Huntingtin
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
AU2007211082B2 (en) 2006-01-27 2012-09-27 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of microRNAs
DE602007009487D1 (de) 2006-01-27 2010-11-11 Isis Pharmaceutical Inc 6-modifizierte bicyclische nukleinsäureanaloga
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
WO2007131238A2 (en) 2006-05-05 2007-11-15 Isis Pharmaceuticals , Inc. Compounds and methods for modulating expression apob
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
AU2007255732B2 (en) 2006-06-08 2014-04-03 Amino Up Chemical Co., Ltd. Sense oligonucleotide capable of controlling the expression of iNOS and composition comprising the same
WO2008001778A1 (fr) 2006-06-27 2008-01-03 Takeda Pharmaceutical Company Limited Animal génétiquement modifié et son utilisation
US8771946B2 (en) 2006-07-24 2014-07-08 Athena Diagnostics, Inc. PKD mutations and evaluation of same
WO2008049085A1 (en) 2006-10-18 2008-04-24 Isis Pharmaceuticals, Inc. Antisense compounds
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
WO2007054100A2 (en) 2006-11-13 2007-05-18 Santaris Pharma A/S Lna nucleoside phosphoramidates
US8293684B2 (en) 2006-11-29 2012-10-23 Exiqon Locked nucleic acid reagents for labelling nucleic acids
WO2008086807A2 (en) 2007-01-19 2008-07-24 Exiqon A/S Mediated cellular delivery of lna oligonucleotides
EP2641971A1 (en) 2007-01-29 2013-09-25 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
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
KR101551985B1 (ko) 2007-03-09 2015-09-09 리가가쿠 겐큐쇼 모노뉴클레오시드 또는 모노뉴클레오티드로부터 유도되는 구조를 갖는 화합물, 핵산, 표지 물질, 핵산 검출 방법 및 키트
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
US8278425B2 (en) 2007-05-30 2012-10-02 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
ES2386492T3 (es) 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos carbocíclicos
WO2009006478A2 (en) 2007-07-05 2009-01-08 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
ES2439591T3 (es) 2007-08-15 2014-01-23 Isis Pharmaceuticals, Inc. Análogos de ácido nucleico de tetrahidropirano
CN101896186A (zh) 2007-10-26 2010-11-24 莱顿教学医院 对抗肌肉病症的方式和方法
US8637478B2 (en) 2007-11-13 2014-01-28 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
US8546556B2 (en) 2007-11-21 2013-10-01 Isis Pharmaceuticals, Inc Carbocyclic alpha-L-bicyclic nucleic acid analogs
EP2231195B1 (en) 2007-12-04 2017-03-29 Arbutus Biopharma Corporation 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
US8530640B2 (en) 2008-02-07 2013-09-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
WO2009151691A2 (en) 2008-03-13 2009-12-17 Celera Corporation Genetic polymorphisms associated wiith venous thrombosis, methods of detection and uses thereof
WO2009124238A1 (en) 2008-04-04 2009-10-08 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising neutrally linked terminal bicyclic nucleosides
US8846639B2 (en) 2008-04-04 2014-09-30 Isis Pharmaceutical, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
JP5559159B2 (ja) 2008-06-11 2014-07-23 バイオニュークレオン ソチエタ・レスポンサビリタ・リミタータ 修飾オリゴヌクレオチドを使用するｈｒｐ−３の阻害
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
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
CA2735129C (en) 2008-11-07 2012-06-26 Centre Hospitalier Universitaire Sainte-Justine Syngap1 dysfunctions and uses thereof in diagnostic and therapeutic applications for mental retardation
CA2746003C (en) 2008-12-04 2020-03-31 Opko Curna, Llc Treatment of vascular endothelial growth factor (vegf) related diseases by inhibition of natural antisense transcript to vegf
WO2010080509A1 (en) 2008-12-19 2010-07-15 Philadelphia Health And Education Corporation 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
AU2010204639B2 (en) 2009-01-14 2014-11-27 Drexel University Modulation of pre-mRNA using splice modulating oligonucleotides as therapeutic agents in the treatment of disease
JP5931720B2 (ja) 2009-05-08 2016-06-08 クルナ・インコーポレーテッド Ｄｍｄファミリーに対する天然アンチセンス転写物の抑制によるジストロフィンファミリー関連疾患の治療
MX361732B (es) 2009-06-17 2018-12-14 Cold Spring Harbor Laboratory Composiciones y metodos para la modulacion de division de smn2 en un sujeto.
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
US20120252877A1 (en) 2009-08-14 2012-10-04 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex
SG179038A1 (en) 2009-09-08 2012-04-27 Lab Corp America Holdings Compositions and methods 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
CN102596983B (zh) 2009-10-29 2016-06-15 国立大学法人大阪大学 交联型人工核苷和核苷酸
BR112012011195B1 (pt) 2009-11-12 2024-02-15 The University Of Western Australia Oligonucleotídeo antissentido, composição farmacêutica e uso de uma composição farmacêutica
JP2013511984A (ja) 2009-11-25 2013-04-11 エリテック・ホールディング・ベスローテン・フェンノートシャップ 副溝結合剤（ＭＧＢ）−オリゴヌクレオチドｍｉＲＮＡアンタゴニスト
WO2011085102A1 (en) 2010-01-11 2011-07-14 Isis Pharmaceuticals, Inc. Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom
ES2733708T3 (es) 2010-02-08 2019-12-02 Ionis Pharmaceuticals Inc Reducción selectiva de variantes alélicas
US9193752B2 (en) 2010-03-17 2015-11-24 Isis Pharmaceuticals, Inc. 5′-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
JP2013529181A (ja) 2010-03-25 2013-07-18 ザ ジェイ． デヴィッド グラッドストーン インスティテューツ 神経障害を治療するための組成物および方法
US9006415B2 (en) 2010-04-06 2015-04-14 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
US9193969B2 (en) 2010-04-19 2015-11-24 Nlife Therapeutics, S.L. Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types
WO2011139695A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. Modified 5' diphosphate nucleosides and oligomeric compounds prepared therefrom
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
WO2011146879A2 (en) 2010-05-20 2011-11-24 University Of Rochester Methods and compositions related to modulating autophagy
WO2011156278A1 (en) 2010-06-07 2011-12-15 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
WO2011159836A2 (en) 2010-06-15 2011-12-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating interaction between proteins and target nucleic acids
JP6023705B2 (ja) 2010-06-23 2016-11-09 カッパーアールエヌエー，インコーポレイテッド ナトリウムチャネル、電位依存性、αサブユニット（ＳＣＮＡ）に対する天然アンチセンス転写物の阻害によるＳＣＮＡ関連疾患の治療
RU2597972C2 (ru) 2010-10-22 2016-09-20 Курна Инк. Лечение заболеваний, связанных с геном альфа-l-идуронидазы (idua), путем ингибирования природного антисмыслового транскрипта гена 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
US20140128449A1 (en) 2011-04-07 2014-05-08 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
EP3072977B1 (en) 2011-04-28 2018-09-19 Life Technologies Corporation Methods and compositions for multiplex pcr
WO2012168435A1 (en) 2011-06-10 2012-12-13 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of leber congenital amaurosis
US20140357558A1 (en) 2011-06-24 2014-12-04 Cold Spring Harbor Laboratory Compositions and methods for treatment of spinal muscular atrophy
ES2626488T3 (es) 2011-07-19 2017-07-25 Wave Life Sciences Pte. Ltd. Procedimientos para la síntesis de ácidos nucleicos funcionalizados
US8592156B2 (en) 2011-08-08 2013-11-26 Roche Molecular Systems, Inc. Predicting response to anti-CD20 therapy in DLBCL patients
DK2751270T3 (da) 2011-08-29 2018-10-29 Ionis Pharmaceuticals Inc Oligomer-konjugatkomplekser og deres anvendelse
AU2012305053B2 (en) 2011-09-05 2017-12-21 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for the treatment of Leber congenital amaurosis
CA2847811C (en) 2011-09-06 2019-10-22 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
CN103946380A (zh) 2011-11-11 2014-07-23 桑塔瑞斯制药公司 用于调控smn2剪接的化合物
US9534222B2 (en) 2011-11-15 2017-01-03 University Of Utah Research Foundation Morpholinos, morpholino upregulating, and associated methods
EP2966180B1 (en) 2011-11-29 2017-08-16 Life Technologies Corporation Methods and compositions for multiplex pcr
EP2802674B1 (en) 2012-01-11 2020-12-16 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of ikbkap splicing
EP3330278A1 (en) 2012-02-08 2018-06-06 Ionis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
UA116981C2 (uk) 2012-02-10 2018-06-11 ПіТіСі ТЕРАП'ЮТІКС, ІНК. Сполуки для лікування спінальної м'язової атрофії
US8846885B2 (en) 2012-02-17 2014-09-30 Ajinomoto Co., Inc. Oligonucleotide with protected base
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
EP2839006B1 (en) 2012-04-20 2018-01-03 Ionis 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
EP2852605B1 (en) 2012-05-22 2018-01-31 Idenix Pharmaceuticals LLC 3',5'-cyclic phosphate prodrugs for hcv infection
US9109001B2 (en) 2012-05-22 2015-08-18 Idenix Pharmaceuticals, Inc. 3′,5′-cyclic phosphoramidate prodrugs for HCV infection
US20150267192A1 (en) 2012-06-08 2015-09-24 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
EP2885313A4 (en) 2012-08-20 2016-03-09 Univ California POLYNUCLEOTIDES WITH BIOREVERSIBLE GROUPS
WO2014045283A1 (en) 2012-09-24 2014-03-27 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
EP2935304A1 (en) 2012-12-19 2015-10-28 IDENIX Pharmaceuticals, Inc. 4'-fluoro nucleosides for the treatment of hcv
CN103667438B (zh) 2013-01-07 2015-04-01 赵晨 一种筛查HRDs致病突变的方法及涉及的基因芯片杂交探针设计方法
CA2897522A1 (en) 2013-01-18 2014-07-24 Anna Mary Rose Therapeutics and diagnostics based on minisatellite repeat element 1 (msr1)
WO2014121287A2 (en) 2013-02-04 2014-08-07 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
EP2957567B1 (en) 2013-02-18 2019-06-05 Shionogi & Co., Ltd. Nucleoside and nucleotide, having nitrogen-containing hetercycle structure
US9339541B2 (en) 2013-03-04 2016-05-17 Merck Sharp & Dohme Corp. Thiophosphate nucleosides for the treatment of HCV
EP2970358B1 (en) 2013-03-04 2021-06-30 Idenix Pharmaceuticals LLC 3'-deoxy nucleosides for the treatment of hcv
US9187515B2 (en) 2013-04-01 2015-11-17 Idenix Pharmaceuticals Llc 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
NZ728517A (en) 2013-05-01 2021-12-24 Ionis Pharmaceuticals Inc Compositions and methods for modulating ttr expression
US9549909B2 (en) 2013-05-03 2017-01-24 The Katholieke Universiteit Leuven Method for the treatment of dravet syndrome
EP3008184A1 (en) 2013-06-13 2016-04-20 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
EP3033422A4 (en) 2013-08-16 2017-08-02 Rana Therapeutics Inc. Oligonucleotides targeting euchromatin regions of genes
US20160201064A1 (en) 2013-08-16 2016-07-14 Rana Therapeutics, Inc. Compositions and methods for modulating expression of frataxin
EP3033423A4 (en) 2013-08-16 2017-04-26 Rana Therapeutics Inc. Epigenetic regulators of frataxin
JP2016528897A (ja) 2013-08-16 2016-09-23 ラナ セラピューティクス インコーポレイテッド Ｒｎａを調節するための組成物および方法
KR20160045063A (ko) 2013-08-19 2016-04-26 에프. 호프만-라 로슈 아게 스크리닝 방법
DK3041958T3 (da) * 2013-09-04 2020-03-09 Cold Spring Harbor Laboratory Reducering af nonsense-medieret mrna-degradering
US11236338B2 (en) 2013-09-05 2022-02-01 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
WO2015036451A1 (en) 2013-09-11 2015-03-19 Synthena Ag Nucleic acids and methods for the treatment of pompe disease
KR20230008892A (ko) 2013-11-21 2023-01-16 메모리얼 슬로안 케터링 캔서 센터 인간 다능성 줄기 세포로부터 기능적 두개 기원판 유도체의 전문화
US10119168B2 (en) 2014-03-12 2018-11-06 The Brigham And Women's Hospital, Inc. Methods for the treatment of kidney fibrosis
US20170044540A1 (en) 2014-04-22 2017-02-16 Mina Therapeutics Limited Sarna compositions and methods of use
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)
EP3201339A4 (en) 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
WO2016061509A1 (en) 2014-10-17 2016-04-21 The Broad Institute, Inc. Compositions and methods of treatng muscular dystrophy
CA2964979A1 (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
JP2018509387A (ja) 2015-01-21 2018-04-05 フェーズアールエックス インコーポレイテッド 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム
EP3256126B1 (en) 2015-02-09 2024-03-27 F. Hoffmann-La Roche AG Compounds for the treatment of cancer
US9840709B2 (en) 2015-02-20 2017-12-12 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting genes associated with cystic fibrosis
BR112017018383B1 (pt) 2015-02-27 2023-04-25 Murdoch University Compostos anti-sentido que induzem a inclusão de éxon2, composições farmacêuticas que compreendem ditos compostos e usos dos mesmos para tratar doença de armazenamento de glicogênio tipo ii
RU2017137410A (ru) 2015-04-03 2019-05-06 Ионис Фармасьютикалз, Инк. Соединения и способы модулирования экспрессии tmprss6
US10668171B2 (en) 2015-05-30 2020-06-02 Ptc Therapeutics, Inc. Methods for modulating RNA splicing
WO2017053982A1 (en) 2015-09-24 2017-03-30 The Trustees Of The University Of Pennsylvania Triptycene derivatives for nucleic acid junction stabilization
EP3359685A1 (en) 2015-10-09 2018-08-15 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
GB2591194B (en) 2015-10-09 2021-10-13 Univ Southampton Modulation of gene expression and screening for deregulated protein expression
EP3183347A4 (en) 2015-10-17 2018-04-18 Lifesplice Pharma LLC Splice modulating oligonucleotides and methods of use thereof
TW201729807A (zh) 2015-10-30 2017-09-01 Ｐｔｃ治療公司 用於治療癲癇的方法
EP4104867A3 (en) 2015-12-14 2023-03-01 Cold Spring Harbor Laboratory Compositions and methods for treatment of central nervous system diseases
CA3005128A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of eye diseases
SG11201804443UA (en) 2015-12-14 2018-06-28 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
EP3390666A4 (en) 2015-12-14 2019-08-07 Cold Spring Harbor Laboratory COMPOSITIONS AND METHODS OF TREATING NURSE DISEASES
EP3389672A4 (en) 2015-12-14 2019-08-14 Cold Spring Harbor Laboratory COMPOSITIONS AND METHOD FOR THE TREATMENT OF LIVER DISEASES
ES2903394T3 (es) * 2015-12-14 2022-04-01 Cold Spring Harbor Laboratory Composiciones para el tratamiento de la retinitis pigmentosa 13
CA3005247A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of polycystic kidney disease
EP3390635A4 (en) 2015-12-14 2019-05-01 Cold Spring Harbor Laboratory Antisense oligomers for the treatment of a tuberous sclerosis complex
CA3005245A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of alagille syndrome
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
US11446398B2 (en) 2016-04-11 2022-09-20 Obsidian Therapeutics, Inc. Regulated biocircuit systems
WO2018007980A1 (en) 2016-07-06 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of pain related disorders
US20190218255A1 (en) 2016-09-16 2019-07-18 Olipass Corporation Scn9a antisense oligonucleotides
WO2018187363A1 (en) 2017-04-03 2018-10-11 Encoded Therapeutics, Inc. Tissue selective transgene expression
EP3609481A4 (en) 2017-04-13 2021-05-26 Ovid Therapeutics Inc. DEVELOPMENTAL ENCEPHALOPATHY TREATMENT METHODS
KR20190142364A (ko) 2017-05-09 2019-12-26 조게닉스 인터내셔널 리미티드 펜플루라민을 사용하여 두즈 증후군을 치료하는 방법
US20200085838A1 (en) 2017-05-16 2020-03-19 Praxis Precision Medicines, Inc. Methods of treating epilepsy and neurodevelopmental disorders
FI3673080T3 (fi) * 2017-08-25 2023-11-23 Stoke Therapeutics Inc Antisense-oligomeerejä häiriötilojen ja sairauksien hoitoon
SG11202003692YA (en) 2017-10-23 2020-05-28 Stoke Therapeutics Inc Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
MX2020005561A (es) 2017-12-01 2020-10-12 Encoded Therapeutics Inc Proteinas modificadas de union a adn.
WO2019191341A1 (en) 2018-03-27 2019-10-03 Factor Bioscience Inc. Nucleic acid-based therapeutics
AU2019253700A1 (en) 2018-04-09 2020-11-26 Allen Institute Rescuing voltage-gated sodium channel function in inhibitory neurons
WO2019224864A1 (ja) 2018-05-21 2019-11-28 国立研究開発法人理化学研究所 Ｓｃｎ１ａ遺伝子の発現増強法とそれによるドラベ症候群の治療法
WO2019227096A1 (en) 2018-05-25 2019-11-28 Rogcon U.R., Inc. Dynamic clamps and methods of use thereof
WO2019236750A2 (en) 2018-06-05 2019-12-12 Tufts Medical Center, Inc. Targeted treatment of autism spectrum disorder and other neurological or psychiatric disorders
EP3810776A1 (en) 2018-06-22 2021-04-28 F. Hoffmann-La Roche AG Oligonucleotides for modulating scn9a expression
US11939582B2 (en) 2018-08-20 2024-03-26 Rogcon, Inc. Antisense oligonucleotides targeting SCN2A for the treatment of SCN1A encephalopathies
US10905778B2 (en) 2018-09-26 2021-02-02 Case Western Reserve University Methods and compositions for treating a premature stop codon-mediated disorder
SG11202109371VA (en) 2019-02-27 2021-09-29 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
CN116334086A (zh) * 2023-03-29 2023-06-27 广州医科大学附属第二医院 一种用于修复或抑制SCN1A基因异常剪接的U1-snRNA及其用途

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