MA45616A - Analogues d'oligonucléotide ciblant lmna humain - Google Patents

Analogues d'oligonucléotide ciblant lmna humain

Info

Publication number
MA45616A
MA45616A MA045616A MA45616A MA45616A MA 45616 A MA45616 A MA 45616A MA 045616 A MA045616 A MA 045616A MA 45616 A MA45616 A MA 45616A MA 45616 A MA45616 A MA 45616A
Authority
MA
Morocco
Prior art keywords
lmna
human
target oligonucleotide
oligonucleotide analogues
analogues
Prior art date
Application number
MA045616A
Other languages
English (en)
Inventor
Richard Keith Bestwick
Kan Cao
Francis S Collins
Michael R Erdos
Leslie B Gordon
Ryszard Kole
Original Assignee
Sarepta Therapeutics Inc
The Progeria Res Foundation
Us Health
Univ Maryland
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Sarepta Therapeutics Inc, The Progeria Res Foundation, Us Health, Univ Maryland filed Critical Sarepta Therapeutics Inc
Publication of MA45616A publication Critical patent/MA45616A/fr

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3183—Diol linkers, e.g. glycols or propanediols
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)

Abstract

La présente invention concerne des oligonucléotides antisens ciblés par lmna destinés à réduire l'expression d'un ou plusieurs isoformes d'arnm de lmna épissées de manière aberrante qui codent pour la progérine.
MA045616A 2016-04-29 2017-04-28 Analogues d'oligonucléotide ciblant lmna humain MA45616A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201662330027P 2016-04-29 2016-04-29

Publications (1)

Publication Number Publication Date
MA45616A true MA45616A (fr) 2019-03-06

Family

ID=59285320

Family Applications (1)

Application Number Title Priority Date Filing Date
MA045616A MA45616A (fr) 2016-04-29 2017-04-28 Analogues d'oligonucléotide ciblant lmna humain

Country Status (14)

Country Link
US (2) US10822608B2 (fr)
EP (1) EP3449000A1 (fr)
JP (2) JP7042255B2 (fr)
KR (1) KR102329187B1 (fr)
CN (1) CN109477109B (fr)
AU (2) AU2017258642B2 (fr)
CA (1) CA3022303A1 (fr)
CO (1) CO2018012873A2 (fr)
EA (1) EA201892467A1 (fr)
MA (1) MA45616A (fr)
MX (1) MX2018013157A (fr)
NZ (1) NZ747685A (fr)
SG (1) SG11201809468XA (fr)
WO (1) WO2017190041A1 (fr)

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
WO2017190041A1 (fr) * 2016-04-29 2017-11-02 Sarepta Therapeutics, Inc. Analogues d'oligonucléotide ciblant lmna humain
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2018039438A1 (fr) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation d'acides aminés non naturels dans des protéines au moyen de l'édition de bases
SG11201903089RA (en) 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
CN117298290A (zh) * 2016-12-19 2023-12-29 萨勒普塔医疗公司 用于肌肉萎缩症的外显子跳跃寡聚体缀合物
WO2018119359A1 (fr) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Édition du gène récepteur ccr5 pour protéger contre l'infection par le vih
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
EP3592381A1 (fr) 2017-03-09 2020-01-15 President and Fellows of Harvard College Vaccin contre le cancer
WO2018165629A1 (fr) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Éditeur de base cytosine à guanine
WO2018176009A1 (fr) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Éditeurs de nucléobase comprenant des protéines de liaison à l'adn programmable par acides nucléiques
WO2018209320A1 (fr) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Arn guides incorporés par aptazyme pour une utilisation avec crispr-cas9 dans l'édition du génome et l'activation transcriptionnelle
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3676376B1 (fr) 2017-08-30 2025-01-15 President and Fellows of Harvard College Éditeurs de bases à haut rendement comprenant une gam
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
WO2019118949A1 (fr) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systèmes et procédés de prédiction des résultats de la réparation en ingénierie génétique
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
EP3820495A4 (fr) 2018-07-09 2022-07-20 The Broad Institute Inc. Modificateurs d'arn épigénétiques programmables par arn et leurs utilisations
TW202020153A (zh) * 2018-07-27 2020-06-01 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
US20240173430A1 (en) * 2018-09-05 2024-05-30 The Broad Institute, Inc. Base editing for treating hutchinson-gilford progeria syndrome
WO2020092453A1 (fr) 2018-10-29 2020-05-07 The Broad Institute, Inc. Éditeurs de nucléobases comprenant geocas9 et utilisations associées
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3942043A2 (fr) 2019-03-19 2022-01-26 The Broad Institute, Inc. Procédés et compositions pour l'édition de séquences nucléotidiques
WO2020214842A1 (fr) 2019-04-17 2020-10-22 The Broad Institute, Inc. Éditeurs de base d'adénine présentant des effets hors cible réduits
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
BR112022022603A2 (pt) 2020-05-08 2023-01-17 Broad Inst Inc Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla
WO2022098014A1 (fr) * 2020-11-03 2022-05-12 한국생명공학연구원 Composition pour prévenir ou traiter la progéria et le vieillissement naturel par édition génique
EP4612292A1 (fr) * 2022-11-02 2025-09-10 Sarepta Therapeutics, Inc. Formulation d'un conjugué oligomère antisens
CN119101680A (zh) * 2023-06-09 2024-12-10 良渚实验室 多核苷酸、药物组合物及其用途
WO2025262186A2 (fr) * 2024-06-20 2025-12-26 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Oligonucléotides thérapeutiques pour le traitement de maladies associées ou causées par une mutation contre-sens, non-sens ou indel dans le cadre

Family Cites Families (74)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CH445129A (fr) 1964-04-29 1967-10-15 Nestle Sa Procédé pour la préparation de composés d'inclusion à poids moléculaire élevé
US3459731A (en) 1966-12-16 1969-08-05 Corn Products Co Cyclodextrin polyethers and their production
US3453257A (en) 1967-02-13 1969-07-01 Corn Products Co Cyclodextrin with cationic properties
US3426011A (en) 1967-02-13 1969-02-04 Corn Products Co Cyclodextrins with anionic properties
US3453259A (en) 1967-03-22 1969-07-01 Corn Products Co Cyclodextrin polyol ethers and their oxidation products
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
WO1986005519A1 (fr) 1985-03-15 1986-09-25 James Summerton Reactif et procede d'analyse de polynucleotides
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
KR0166088B1 (ko) 1990-01-23 1999-01-15 . 수용해도가 증가된 시클로덱스트린 유도체 및 이의 용도
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
AU659482B2 (en) 1991-06-28 1995-05-18 Massachusetts Institute Of Technology Localized oligonucleotide therapy
EP1251170A3 (fr) 1992-07-17 2002-10-30 Ribozyme Pharmaceuticals, Inc. Procédé et réactif pour le traitement de maladies animales dépendants de NF-kappaB
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
IL115849A0 (en) 1994-11-03 1996-01-31 Merz & Co Gmbh & Co Tangential filtration preparation of liposomal drugs and liposome product thereof
US6245747B1 (en) 1996-03-12 2001-06-12 The Board Of Regents Of The University Of Nebraska Targeted site specific antisense oligodeoxynucleotide delivery method
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
WO1999042091A2 (fr) 1998-02-19 1999-08-26 Massachusetts Institute Of Technology Compositions d'apport dans des cellules
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
NZ514348A (en) 1999-05-04 2004-05-28 Exiqon As L-ribo-LNA analogues
US7070807B2 (en) 1999-12-29 2006-07-04 Mixson A James Branched histidine copolymers and methods for using same
AU2281201A (en) 1999-12-29 2001-07-09 A. James Mixson Histidine copolymer and methods for using same
WO2003020739A2 (fr) 2001-09-04 2003-03-13 Exiqon A/S Compositions d'acides nucleiques verrouilles et utilisations
KR100464261B1 (ko) 2002-01-24 2005-01-03 주식회사 파나진 Ｐｎａ 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법
KR20030084444A (ko) 2002-04-26 2003-11-01 주식회사 파나진 Ｐｎａ 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
AU2003291682A1 (en) 2002-11-05 2004-06-03 Isis Pharmaceuticals, Inc. 2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations
EP1629105B1 (fr) 2003-04-29 2010-09-01 AVI BioPharma, Inc. Compositions pour ameliorer le transport et l'efficacite antisense d'analogues d'acides nucleiques dans des cellules
US7211668B2 (en) 2003-07-28 2007-05-01 Panagene, Inc. PNA monomer and precursor
WO2007047913A2 (fr) * 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions et procédés pour la modulation de l'expression du gène lmna
ES2645410T3 (es) 2006-05-10 2017-12-05 Sarepta Therapeutics, Inc. Análogos de oligonucleótidos que tienen enlaces intersubunitarios catiónicos
MX2009002859A (es) * 2006-09-15 2009-03-30 Enzon Pharmaceuticals Inc Enlazadores biodegradables a base de ester impedido para suministro de oligonucleotidos.
WO2009005793A2 (fr) 2007-06-29 2009-01-08 Avi Biopharma, Inc. Conjugués peptidiques spécifiques d'un tissu et procédés
CA2693742A1 (fr) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules pour cibler des composes vers divers organes, tissus ou cellules tumorales selectionnes
DK2207779T3 (da) 2007-11-15 2014-07-14 Sarepta Therapeutics Inc Fremgangsmåde til syntese af morpholinooligomerer
SG171914A1 (en) 2008-12-02 2011-07-28 Chiralgen Ltd Method for the synthesis of phosphorus atom modified nucleic acids
JP2012523225A (ja) 2009-04-10 2012-10-04 アソシアシオン・アンスティテュ・ドゥ・ミオロジー 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
US20110269665A1 (en) * 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
EP2451461A4 (fr) 2009-07-06 2013-05-29 Ontorii Inc Nouveaux précurseurs d'acide nucléique et leurs méthodes d'utilisation
CN102574888A (zh) 2009-09-16 2012-07-11 株式会社启拉坚 用于rna及其衍生物的合成的新型保护基
ES2816898T3 (es) * 2010-05-13 2021-04-06 Sarepta Therapeutics Inc Compuestos que modulan la actividad de señalización de las interleucinas 17 y 23
KR102182663B1 (ko) * 2010-05-28 2020-11-25 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
DK2623507T3 (da) 2010-09-30 2017-01-02 Nippon Shinyaku Co Ltd Morpholinonukleinsyrederivat
JP5653160B2 (ja) 2010-10-15 2015-01-14 ダンロップスポーツ株式会社 ゴルフボール
IL273838B (en) 2011-05-05 2022-09-01 Sarepta Therapeutics Inc Peptide and oligonucleotide conjugates, preparations containing them and their uses
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
AU2012300633B2 (en) 2011-08-30 2017-04-27 United Kingdom Research And Innovation Cell- penetrating peptides having a central hydrophobic domain
EP2581448B1 (fr) 2011-10-13 2015-01-28 Association Institut de Myologie ADN tricyclo-phosphorothioate
JP2015500204A (ja) 2011-11-18 2015-01-05 サレプタ セラピューティクス， インコーポレイテッド 機能的に修飾されたオリゴヌクレオチドおよびそのサブユニット
WO2013086441A2 (fr) 2011-12-08 2013-06-13 Sarepta Therapeutics, Inc. Analogues d'oligonucléotide ciblant lmna humain
CN118581086A (zh) 2012-01-27 2024-09-03 比奥马林技术公司 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
DE102012101676A1 (de) 2012-02-29 2013-08-29 Klaus-Dieter Rösler Verfahren und Vorrichtung zum Bearbeiten von Formularen mit einer Datenverarbeitungsanlage
DK2872485T3 (da) 2012-07-13 2021-03-08 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
CN112007045B (zh) 2012-07-13 2026-01-06 波涛生命科学有限公司 手性控制
EP2986710A4 (fr) * 2013-04-16 2016-11-09 Sloan Kettering Inst Cancer Cellules à âge modifié et procédés de préparation de cellules à âge modifié
EP3095461A4 (fr) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Adjuvant d'acide nucléique chiral possédant une activité d'induction d'immunité, et activateur d'induction d'immunité
WO2015108048A1 (fr) 2014-01-15 2015-07-23 株式会社新日本科学 Adjuvant d'acide nucléique chiral ayant un effet antitumoral et agent antitumoral
US10322173B2 (en) 2014-01-15 2019-06-18 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
KR102423317B1 (ko) 2014-01-16 2022-07-22 웨이브 라이프 사이언시스 리미티드 키랄 디자인
CA2948373A1 (fr) * 2014-05-16 2015-11-19 Oregon State University Composes antisens antibacteriens et procedes associes
EP3569252B1 (fr) * 2014-05-19 2021-12-15 Oregon State University Composés antibactériens et méthodes antisens
WO2015179743A1 (fr) * 2014-05-23 2015-11-26 Genzyme Corporation Banque et approche de criblage permettant d'obtenir des peptides de pénétration cellulaire améliorés
WO2017190041A1 (fr) * 2016-04-29 2017-11-02 Sarepta Therapeutics, Inc. Analogues d'oligonucléotide ciblant lmna humain

Also Published As

Publication number Publication date
SG11201809468XA (en) 2018-11-29
NZ747685A (en) 2023-05-26
US20210010001A1 (en) 2021-01-14
JP2022046725A (ja) 2022-03-23
KR20190025821A (ko) 2019-03-12
EP3449000A1 (fr) 2019-03-06
MX2018013157A (es) 2019-05-23
JP7042255B2 (ja) 2022-03-25
CO2018012873A2 (es) 2020-06-19
AU2023274161A1 (en) 2023-12-21
CA3022303A1 (fr) 2017-11-02
AU2017258642A1 (en) 2018-11-15
AU2017258642B2 (en) 2023-08-31
US20190127735A1 (en) 2019-05-02
WO2017190041A1 (fr) 2017-11-02
CN109477109A (zh) 2019-03-15
CN109477109B (zh) 2022-09-23
JP2019514426A (ja) 2019-06-06
EA201892467A1 (ru) 2019-05-31
US11802283B2 (en) 2023-10-31
KR102329187B1 (ko) 2021-11-22
AU2017258642A8 (en) 2018-11-29
US10822608B2 (en) 2020-11-03
BR112018072279A2 (pt) 2019-02-12

Similar Documents

Publication Publication Date Title
EA201892467A1 (ru) Олигонуклеотидные аналоги, нацеленные на lmna человека
MX2022004101A (es) Composiciones de oligonucleotidos y metodos de uso de las mismas.
ZA202306507B (en) Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
BR112016026950A2 (pt) composições de rnai para angiotensinogênio (agt) e métodos de uso das mesmas
IL278086A (en) PSCK9 targeting oligonucleotides for the treatment of hypercholesterolemia and related diseases
CY1123119T1 (el) Αντινοηματικο νουκλεϊκο οξυ
JOP20200115A1 (ar) تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
PH12019550076A1 (en) SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP3700570A4 (fr) Oligomères antisens pour le traitement d'états et de maladies basés sur le déclin d'arnm à médiation non-sens
MX2023012080A (es) Composiciones de acido ribonucleico interferente (arni) de transtiretina (ttr) y sus usos en el tratamiento o prevencion de enfermedades asociadas con transtiretina (ttr).
NZ733882A (en) Compositions for modulating c9orf72 expression
IL284447A (en) Dux4 rna silencing using rna targeting crispr-cas13b
EP3942049A4 (fr) Thérapie d'augmentation de la progranuline à base d'oligonucléotides anti-sens dans les maladies neurodégénératives
CO2021018034A2 (es) Oligonucleótidos gapmer modificados y métodos de uso
WO2016130806A3 (fr) Compositions d'arni du gène codant pour la protéine 3 contenant un domaine phospholipase de type patatine (pnpla3) et leurs procédés d'utilisation
MX2019003376A (es) Tratamiento de aav de la enfermedad de huntington.
IL275950A (en) Antisense alpha-synuclein oligonucleotides and their use
IL275903A (en) Antisense oligonucleotides directed against alpha-synculin and their uses
IL269735A (en) Antisense oligonucleotides for the treatment of stargardt disease
MX2020006297A (es) Variantes de cd19.
EP4175654A4 (fr) Oligonucléotides antisens ciblant le sars-cov-2
IL275902A (en) Antisense oligonucleotides directed against alpha-synculin and their uses
EP3999523A4 (fr) Oligonucléotides antisens de mélanophiline
MX2017012610A (es) Composiciones y metodos para inhibir la expresion del gen lect2.
EP3794124A4 (fr) Oligonucléotides antisens de métalloprotéinase-1 matricielle