JP2019520079A5 - - Google Patents

Download PDF

Info

Publication number
JP2019520079A5
JP2019520079A5 JP2019500268A JP2019500268A JP2019520079A5 JP 2019520079 A5 JP2019520079 A5 JP 2019520079A5 JP 2019500268 A JP2019500268 A JP 2019500268A JP 2019500268 A JP2019500268 A JP 2019500268A JP 2019520079 A5 JP2019520079 A5 JP 2019520079A5
Authority
JP
Japan
Prior art keywords
composition
dna
scn9a
endonucleases
deoxyribonucleic 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
JP2019500268A
Other languages
English (en)
Japanese (ja)
Other versions
JP7548696B2 (ja
JP2019520079A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/IB2017/054086 external-priority patent/WO2018007980A1/en
Publication of JP2019520079A publication Critical patent/JP2019520079A/ja
Publication of JP2019520079A5 publication Critical patent/JP2019520079A5/ja
Priority to JP2022109956A priority Critical patent/JP7429264B2/ja
Priority to JP2024010029A priority patent/JP7719222B2/ja
Application granted granted Critical
Publication of JP7548696B2 publication Critical patent/JP7548696B2/ja
Priority to JP2025123989A priority patent/JP2025146901A/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2019500268A 2016-07-06 2017-07-06 疼痛関連障害を処置するための物質及び方法 Active JP7548696B2 (ja)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2022109956A JP7429264B2 (ja) 2016-07-06 2022-07-07 疼痛関連障害を処置するための物質及び方法
JP2024010029A JP7719222B2 (ja) 2016-07-06 2024-01-26 疼痛関連障害を処置するための物質及び方法
JP2025123989A JP2025146901A (ja) 2016-07-06 2025-07-24 疼痛関連障害を処置するための物質及び方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662358763P 2016-07-06 2016-07-06
US62/358,763 2016-07-06
US201762461874P 2017-02-22 2017-02-22
US62/461,874 2017-02-22
PCT/IB2017/054086 WO2018007980A1 (en) 2016-07-06 2017-07-06 Materials and methods for treatment of pain related disorders

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022109956A Division JP7429264B2 (ja) 2016-07-06 2022-07-07 疼痛関連障害を処置するための物質及び方法

Publications (3)

Publication Number Publication Date
JP2019520079A JP2019520079A (ja) 2019-07-18
JP2019520079A5 true JP2019520079A5 (https=) 2020-08-13
JP7548696B2 JP7548696B2 (ja) 2024-09-10

Family

ID=59649967

Family Applications (4)

Application Number Title Priority Date Filing Date
JP2019500268A Active JP7548696B2 (ja) 2016-07-06 2017-07-06 疼痛関連障害を処置するための物質及び方法
JP2022109956A Active JP7429264B2 (ja) 2016-07-06 2022-07-07 疼痛関連障害を処置するための物質及び方法
JP2024010029A Active JP7719222B2 (ja) 2016-07-06 2024-01-26 疼痛関連障害を処置するための物質及び方法
JP2025123989A Pending JP2025146901A (ja) 2016-07-06 2025-07-24 疼痛関連障害を処置するための物質及び方法

Family Applications After (3)

Application Number Title Priority Date Filing Date
JP2022109956A Active JP7429264B2 (ja) 2016-07-06 2022-07-07 疼痛関連障害を処置するための物質及び方法
JP2024010029A Active JP7719222B2 (ja) 2016-07-06 2024-01-26 疼痛関連障害を処置するための物質及び方法
JP2025123989A Pending JP2025146901A (ja) 2016-07-06 2025-07-24 疼痛関連障害を処置するための物質及び方法

Country Status (7)

Country Link
US (3) US11459587B2 (https=)
EP (1) EP3481857B1 (https=)
JP (4) JP7548696B2 (https=)
CN (2) CN109843914B (https=)
AU (1) AU2017292173B2 (https=)
CA (1) CA3029141A1 (https=)
WO (1) WO2018007980A1 (https=)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017106377A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
CN109843914B (zh) 2016-07-06 2024-03-15 沃泰克斯药物股份有限公司 用于治疗疼痛相关病症的材料和方法
JP7305534B2 (ja) 2016-07-06 2023-07-10 バーテックス ファーマシューティカルズ インコーポレイテッド 疼痛関連障害を処置するための物質及び方法
IL315358A (en) * 2016-08-18 2024-11-01 Univ California CRISPR-CAS genome engineering using a modular AAV delivery system
KR102318434B1 (ko) 2017-08-25 2021-11-01 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
MX2020008581A (es) * 2018-02-21 2020-09-21 Bristol Myers Squibb Co Oligonucleotidos antisentido de la proteina cinasa del tipo ii delta dependiente del calcio/calmodulina (camk2d) y sus usos.
EP3810776A1 (en) 2018-06-22 2021-04-28 F. Hoffmann-La Roche AG Oligonucleotides for modulating scn9a expression
US12281305B2 (en) 2018-11-21 2025-04-22 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing prion expression
AU2020227825B2 (en) 2019-02-27 2026-03-26 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
US20220202957A1 (en) * 2019-04-09 2022-06-30 The Regents Of The University Of California Long-lasting analgesia via targeted in vivo epigenetic repression
MX2021012493A (es) * 2019-04-12 2022-01-06 Vertex Pharma Sistemas de edición génica para modificar un gen scn9a o scn10a y métodos para su uso.
WO2021030344A1 (en) * 2019-08-12 2021-02-18 Lifeedit, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
JP7528212B2 (ja) 2019-11-26 2024-08-05 ディスアーム セラピューティクス, インコーポレイテッド 神経保護のための方法および組成物
EP4077672A1 (en) * 2019-12-20 2022-10-26 F. Hoffmann-La Roche AG Enhanced oligonucleotides for inhibiting scn9a expression
TW202208626A (zh) 2020-04-24 2022-03-01 美商生命編輯公司 Rna引導核酸酶及其活性片段與變體,以及使用方法
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
WO2021247995A2 (en) * 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
KR20230095056A (ko) * 2020-07-22 2023-06-28 더 플로레이 인스티튜트 오브 뉴로사이언스 앤드 멘탈 헬스 Scn2a에서 기능-상실 돌연변이와 연관된 장애를 치료하기 위한 조성물 및 방법
JP2023542889A (ja) 2020-09-17 2023-10-12 キュー-ステート バイオサイエンシーズ, インコーポレイテッド 複数の標的を介して疼痛を処置するための治療用組成物
WO2022147541A1 (en) * 2021-01-04 2022-07-07 Exicure Operating Company Compounds for modulating scn9a expression
CA3227103A1 (en) * 2021-07-30 2023-02-02 Matthew P. GEMBERLING Compositions and methods for modulating expression of frataxin (fxn)
WO2023022974A2 (en) * 2021-08-16 2023-02-23 Editas Medicine, Inc. Treatment of pain
GB202114871D0 (en) * 2021-10-18 2021-12-01 Ucl Business Ltd Guide RNA and uses thereof
KR20240126870A (ko) 2021-12-22 2024-08-21 캠프4 테라퓨틱스 코포레이션 조절 rna들을 표적으로 하는 안티센스 올리고뉴클레오티드를 사용한 유전자 전사의 조절
CN115844918B (zh) * 2022-09-27 2024-11-22 首都医科大学附属北京天坛医院 一种miR-3131抑制剂在制备药物中的应用
WO2025106999A1 (en) * 2023-11-16 2025-05-22 The Johns Hopkins University Deprivation of human pluripotent stem cell-derived trpv1+, mrgprx1+ and scn9a+ sensory neurons and their functional characterization
WO2025128967A1 (en) * 2023-12-15 2025-06-19 Iowa State University Research Foundation, Inc. Compositions and methods for restoring susceptibility to pyrethroid insecticides in resistant populations

Family Cites Families (193)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4476301A (en) 1982-04-29 1984-10-09 Centre National De La Recherche Scientifique Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon
JPS5927900A (ja) 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
FR2540122B1 (fr) 1983-01-27 1985-11-29 Centre Nat Rech Scient Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application
US4605735A (en) 1983-02-14 1986-08-12 Wakunaga Seiyaku Kabushiki Kaisha Oligonucleotide derivatives
US4948882A (en) 1983-02-22 1990-08-14 Syngene, Inc. Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis
US4824941A (en) 1983-03-10 1989-04-25 Julian Gordon Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems
US4587044A (en) 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5118802A (en) 1983-12-20 1992-06-02 California Institute Of Technology DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside
US5550111A (en) 1984-07-11 1996-08-27 Temple University-Of The Commonwealth System Of Higher Education Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
US4828979A (en) 1984-11-08 1989-05-09 Life Technologies, Inc. Nucleotide analogs for nucleic acid labeling and detection
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US4762779A (en) 1985-06-13 1988-08-09 Amgen Inc. Compositions and methods for functionalizing nucleic acids
US5317098A (en) 1986-03-17 1994-05-31 Hiroaki Shizuya Non-radioisotope tagging of fragments
JPS638396A (ja) 1986-06-30 1988-01-14 Wakunaga Pharmaceut Co Ltd ポリ標識化オリゴヌクレオチド誘導体
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
EP0366685B1 (en) 1987-06-24 1994-10-19 Howard Florey Institute Of Experimental Physiology And Medicine Nucleoside derivatives
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US4924624A (en) 1987-10-22 1990-05-15 Temple University-Of The Commonwealth System Of Higher Education 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
US5525465A (en) 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
DE3738460A1 (de) 1987-11-12 1989-05-24 Max Planck Gesellschaft Modifizierte oligonukleotide
US5082830A (en) 1988-02-26 1992-01-21 Enzo Biochem, Inc. End labeled nucleotide probe
JPH03503894A (ja) 1988-03-25 1991-08-29 ユニバーシィティ オブ バージニア アランミ パテンツ ファウンデイション オリゴヌクレオチド n‐アルキルホスホラミデート
US5278302A (en) 1988-05-26 1994-01-11 University Patents, Inc. Polynucleotide phosphorodithioates
US5109124A (en) 1988-06-01 1992-04-28 Biogen, Inc. Nucleic acid probe linked to a label having a terminal cysteine
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5262536A (en) 1988-09-15 1993-11-16 E. I. Du Pont De Nemours And Company Reagents for the preparation of 5'-tagged oligonucleotides
US5512439A (en) 1988-11-21 1996-04-30 Dynal As Oligonucleotide-linked magnetic particles and uses thereof
US5457183A (en) 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
US5599923A (en) 1989-03-06 1997-02-04 Board Of Regents, University Of Tx Texaphyrin metal complexes having improved functionalization
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5451463A (en) 1989-08-28 1995-09-19 Clontech Laboratories, Inc. Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5254469A (en) 1989-09-12 1993-10-19 Eastman Kodak Company Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5292873A (en) 1989-11-29 1994-03-08 The Research Foundation Of State University Of New York Nucleic acids labeled with naphthoquinone probe
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5486603A (en) 1990-01-08 1996-01-23 Gilead Sciences, Inc. Oligonucleotide having enhanced binding affinity
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5214136A (en) 1990-02-20 1993-05-25 Gilead Sciences, Inc. Anthraquinone-derivatives oligonucleotides
AU7579991A (en) 1990-02-20 1991-09-18 Gilead Sciences, Inc. Pseudonucleosides and pseudonucleotides and their polymers
US5321131A (en) 1990-03-08 1994-06-14 Hybridon, Inc. Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
ES2116977T3 (es) 1990-05-11 1998-08-01 Microprobe Corp Soportes solidos para ensayos de hibridacion de acidos nucleicos y metodos para inmovilizar oligonucleotidos de modo covalente.
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
JPH0874B2 (ja) 1990-07-27 1996-01-10 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 遺伝子発現を検出および変調するヌクレアーゼ耐性、ピリミジン修飾オリゴヌクレオチド
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
MY107332A (en) 1990-08-03 1995-11-30 Sterling Drug Inc Compounds and methods for inhibiting gene expression.
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
US5177196A (en) 1990-08-16 1993-01-05 Microprobe Corporation Oligo (α-arabinofuranosyl nucleotides) and α-arabinofuranosyl precursors thereof
US5512667A (en) 1990-08-28 1996-04-30 Reed; Michael W. Trifunctional intermediates for preparing 3'-tailed oligonucleotides
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
JPH06505704A (ja) 1990-09-20 1994-06-30 ギリアド サイエンシズ,インコーポレイテッド 改変ヌクレオシド間結合
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
KR930702373A (ko) 1990-11-08 1993-09-08 안토니 제이. 페이네 합성 올리고누클레오티드에 대한 다중 리포터(Reporter)그룹의 첨합
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
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5371241A (en) 1991-07-19 1994-12-06 Pharmacia P-L Biochemicals Inc. Fluorescein labelled phosphoramidites
US5571799A (en) 1991-08-12 1996-11-05 Basco, Ltd. (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response
DE69233046T2 (de) 1991-10-24 2004-03-04 Isis Pharmaceuticals, Inc., Carlsfeld Derivatisierte oligonukleotide mit verbessertem aufnahmevermögen
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
HU9501974D0 (en) 1993-03-31 1995-09-28 Sterling Winthrop Inc Oligonucleotides with amide linkages replacing phosphodiester linkages
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5597696A (en) 1994-07-18 1997-01-28 Becton Dickinson And Company Covalent cyanine dye oligonucleotide conjugates
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
US6632670B1 (en) 1995-09-08 2003-10-14 Genzyme Corporation AAV vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6287860B1 (en) 2000-01-20 2001-09-11 Isis Pharmaceuticals, Inc. Antisense inhibition of MEKK2 expression
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
US20030158403A1 (en) 2001-07-03 2003-08-21 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
EP2447370B1 (en) 2001-09-28 2018-07-18 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. MicroRNA molecules
US8927269B2 (en) 2003-05-19 2015-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
US8137960B2 (en) 2003-12-04 2012-03-20 The United States Of America As Represented By The Department Of Health And Human Services Bovine adeno-associated viral (BAAV) vector and uses thereof
US7427396B2 (en) 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
US7670840B2 (en) 2006-01-05 2010-03-02 The Ohio State University Research Foundation Micro-RNA expression abnormalities of pancreatic, endocrine and acinar tumors
EP1969147B1 (en) 2006-01-05 2014-07-30 The Ohio State University Research Foundation microRNA-based methods for the diagnosis of stomach cancer
EP2522746B1 (en) 2006-03-02 2014-11-12 The Ohio State University Research Foundation MicroRNA expression profile associated with pancreatic cancer
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US20090131348A1 (en) 2006-09-19 2009-05-21 Emmanuel Labourier Micrornas differentially expressed in pancreatic diseases and uses thereof
US8252538B2 (en) 2006-11-01 2012-08-28 The Ohio State University MicroRNA expression signature for predicting survival and metastases in hepatocellular carcinoma
WO2008073915A2 (en) 2006-12-08 2008-06-19 Asuragen, Inc. Micrornas differentially expressed in leukemia and uses thereof
WO2008147974A1 (en) 2007-05-23 2008-12-04 University Of South Florida Micro-rnas modulating immunity and inflammation
US20090099034A1 (en) 2007-06-07 2009-04-16 Wisconsin Alumni Research Foundation Reagents and Methods for miRNA Expression Analysis and Identification of Cancer Biomarkers
CN101918594A (zh) 2007-11-30 2010-12-15 俄亥俄州立大学研究基金会 肺癌中外周血中的微rna表达特征谱和靶向
EP2260110B1 (en) 2008-02-08 2014-11-12 Asuragen, INC. miRNAs DIFFERENTIALLY EXPRESSED IN LYMPH NODES FROM CANCER PATIENTS
CA2717026A1 (en) 2008-02-28 2009-09-03 The Ohio State University Research Foundation Microrna signatures associated with human chronic lymphocytic leukemia (ccl) and uses thereof
JP2009215191A (ja) * 2008-03-07 2009-09-24 Keio Gijuku 神経損傷治療剤及び神経損傷治療方法
EP2112235A1 (en) 2008-04-24 2009-10-28 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Compositions and methods for microRNA expression profiling of nasopharyngeal carcinoma
WO2010018563A2 (en) 2008-08-12 2010-02-18 Rosetta Genomics Ltd. Compositions and methods for the prognosis of lymphoma
US9074206B2 (en) 2008-11-13 2015-07-07 Fudan University Compositions and methods for micro-RNA expression profiling of colorectal cancer
US20110016541A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of sensory-related genes in animals
US20110023148A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of addiction-related genes in animals
EP2358902A1 (en) 2008-12-10 2011-08-24 Universität Regensburg Compositions and methods for micro-rna expression profiling of cancer stem cells
EP2427574A2 (en) 2009-05-08 2012-03-14 The Ohio State University Research Foundation Microrna expression profiling and targeting in chronic obstructive pulmonary disease (copd) lung tissue and methods of use thereof
JP5700301B2 (ja) 2009-06-03 2015-04-15 国立大学法人大阪大学 多能性幹細胞からの神経堤細胞群の分化誘導方法
EP2456877A4 (en) 2009-07-24 2012-05-30 Sigma Aldrich Co Llc METHOD OF GENOMIC PROCESSING
CN102858985A (zh) 2009-07-24 2013-01-02 西格马-奥尔德里奇有限责任公司 基因组编辑方法
WO2011076142A1 (en) 2009-12-24 2011-06-30 Fudan University Compositions and methods for microrna expession profiling in plasma of colorectal cancer
WO2011076143A1 (en) 2009-12-24 2011-06-30 Fudan University Compositions and methods for microrna expression profiling of lung cancer
EP2341145A1 (en) 2009-12-30 2011-07-06 febit holding GmbH miRNA fingerprint in the diagnosis of diseases
WO2011094683A2 (en) 2010-01-29 2011-08-04 H. Lee Moffitt Cancer Center And Research Institute, Inc. Method of identifying myelodysplastic syndromes
EP2354246A1 (en) 2010-02-05 2011-08-10 febit holding GmbH miRNA in the diagnosis of ovarian cancer
US20130059015A1 (en) 2010-03-11 2013-03-07 H. Lee Moffitt Cancer Center & Research Institute Human Cancer micro-RNA Expression Profiles Predictive of Chemo-Response
WO2011157294A1 (en) 2010-06-16 2011-12-22 Universita' Degli Studi Di Padova Compositions for use in treating or preventing cancer, breast cancer, lung cancer, ovarian cancer, metastasis, heart failure, cardiac remodelling, dilated cardiomyopathy, autoimmune diseases, or diseases or disorders related thereto
WO2012151212A1 (en) 2011-05-01 2012-11-08 University Of Rochester Multifocal hepatocellular carcinoma microrna expression patterns and uses thereof
EP2705370A2 (en) 2011-05-06 2014-03-12 Xentech Markers for cancer prognosis and therapy and methods of use
DK2732052T3 (en) 2011-07-15 2017-02-20 Leo Pharma As Diagnostic microRNA profiling in cutaneous T cell lymphoma (CTCL)
EP2751292A4 (en) 2011-09-01 2015-05-20 Allegro Diagnostics Corp METHOD AND COMPOSITIONS FOR DETECTING CANCER BASED ON MIRNA EXPRESSION PROFILES
CA2850925C (en) 2011-10-26 2017-01-10 Pfizer Limited (4-phenylimidazol-2-yl) ethylamine derivatives useful as sodium channel modulators
US20140243240A1 (en) 2011-10-26 2014-08-28 Georgetown University microRNA EXPRESSION PROFILING OF THYROID CANCER
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
AU2013293270B2 (en) 2012-07-25 2018-08-16 Massachusetts Institute Of Technology Inducible DNA binding proteins and genome perturbation tools and applications thereof
MX2015010749A (es) 2013-03-14 2015-11-30 Regeneron Pharma Anticuerpos humanos para nav1.7.
ES2926774T3 (es) 2013-03-15 2022-10-28 Univ North Carolina Chapel Hill Métodos y composiciones para vectores AAV de doble unión de glicano
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
MX2016004282A (es) 2013-10-03 2016-10-12 Janssen Biotech Inc Variantes de protoxina-ii y metodos de uso.
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2015141521A1 (ja) 2014-03-21 2015-09-24 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及び記録媒体
WO2015175642A2 (en) 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease
JP6715482B2 (ja) 2014-07-18 2020-07-01 国立大学法人京都大学 多能性幹細胞から免疫細胞療法用t細胞を誘導する方法
JP6723230B2 (ja) 2014-10-09 2020-07-15 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 神経障害性疼痛を治療するためのcsf1−dap12経路のメンバー遺伝子の標的破壊
WO2016191684A1 (en) 2015-05-28 2016-12-01 Finer Mitchell H Genome editing vectors
WO2017024318A1 (en) 2015-08-06 2017-02-09 Dana-Farber Cancer Institute, Inc. Targeted protein degradation to attenuate adoptive t-cell therapy associated adverse inflammatory responses
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
US11221329B2 (en) 2015-10-30 2022-01-11 Lieber Institute, Inc. Treatment of neurological and neurodevelopmental diseases and disorders associated with aberrant ion channel expression and activity
WO2017083722A1 (en) 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy
JP2019500268A (ja) * 2015-12-25 2019-01-10 コンパニー ゼネラール デ エタブリッスマン ミシュラン ラジアルカーカスまたはバイアスカーカスを有するタイヤ
JP7305534B2 (ja) 2016-07-06 2023-07-10 バーテックス ファーマシューティカルズ インコーポレイテッド 疼痛関連障害を処置するための物質及び方法
CN109843914B (zh) 2016-07-06 2024-03-15 沃泰克斯药物股份有限公司 用于治疗疼痛相关病症的材料和方法
EP3490542A4 (en) 2016-07-26 2020-07-08 Flagship Pioneering Innovations V, Inc. NEUROMODULATION COMPOSITIONS AND RELATED THERAPEUTIC METHODS FOR THE TREATMENT OF CANCER BY MODULATION OF AN ANTI-CANCER RESPONSE
IL315358A (en) * 2016-08-18 2024-11-01 Univ California CRISPR-CAS genome engineering using a modular AAV delivery system
US20190255106A1 (en) 2016-09-07 2019-08-22 Flagship Pioneering Inc. Methods and compositions for modulating gene expression
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
WO2018165503A1 (en) 2017-03-10 2018-09-13 Vanderbilt University Nasopharyngeal catheters and applications of same

Similar Documents

Publication Publication Date Title
JP2019520079A5 (https=)
JP2019520080A5 (https=)
JP2020508659A5 (https=)
JP2024028643A5 (https=)
JP2019513407A5 (https=)
JP2018535691A5 (https=)
FI3445388T3 (fi) Materiaaleja ja menetelmiä hemoglobinopatioiden hoitamiseksi
CN116836962B (zh) 工程化的腺苷脱氨酶及碱基编辑器
RU2020104969A (ru) Способы и продукты для получения и доставки нуклеиновых кислот
KR102882704B1 (ko) CRISPR/Cas9 시스템을 이용한 유전체 단일염기 편집 방법 및 이의 용도
Choi et al. Base editing strategies to convert CAG to CAA diminish the disease-causing mutation in Huntington’s disease
CN107746859A (zh) 靶向血红蛋白hbb突变基因的造血干细胞基因修饰方法
CN109536494A (zh) 一种用于修复HBB1基因点突变的gRNA、基因编辑系统、表达载体和基因编辑试剂盒
WO2023019164A2 (en) High-throughput precision genome editing in human cells
Bhushan et al. Advancements in CRISPR-Based therapies for genetic modulation in neurodegenerative disorders
CA3233534A1 (en) Direct transdifferentiation for treatment of neurological disease
JP7644722B2 (ja) 真核細胞への遺伝子編集システムの送達のための細菌プラットフォーム
KR20250056942A (ko) B2m 세이프 하버 부위에서의 녹인을 위한 전략
CN115873850A (zh) 腺嘌呤碱基编辑系统及其应用
Singh et al. Harnessing CRISPR for the future: Innovations, applications, and challenges in genome editing
US20240335561A1 (en) A method for in vivo gene therapy to cure scd without myeloablative toxicity
WO2025190256A1 (en) Type ii cas protein and uses thereof
WO2019194320A1 (ja) エンジニアリングされたBlCas9ヌクレアーゼ
Morales et al. CRISPR/CAS9 gene editing in diabetes mellitus: a future cure
WO2024026478A1 (en) Compositions and methods for treating a congenital eye disease