JP7305534B2 - 疼痛関連障害を処置するための物質及び方法 - Google Patents

疼痛関連障害を処置するための物質及び方法 Download PDF

Info

Publication number
JP7305534B2
JP7305534B2 JP2019500283A JP2019500283A JP7305534B2 JP 7305534 B2 JP7305534 B2 JP 7305534B2 JP 2019500283 A JP2019500283 A JP 2019500283A JP 2019500283 A JP2019500283 A JP 2019500283A JP 7305534 B2 JP7305534 B2 JP 7305534B2
Authority
JP
Japan
Prior art keywords
cells
sequence
cas9
gene
composition
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.)
Active
Application number
JP2019500283A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019520080A (ja
JP2019520080A5 (OSRAM
Inventor
スベン ランドバーグ アンテ
クルカーニー サマース
クレイン ローレンス
クマー パッドマナブハン ハリ
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Vertex Pharmaceuticals Inc
Original Assignee
Vertex Pharmaceuticals Inc
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 Vertex Pharmaceuticals Inc filed Critical Vertex Pharmaceuticals Inc
Publication of JP2019520080A publication Critical patent/JP2019520080A/ja
Publication of JP2019520080A5 publication Critical patent/JP2019520080A5/ja
Priority to JP2022167518A priority Critical patent/JP7544785B2/ja
Application granted granted Critical
Publication of JP7305534B2 publication Critical patent/JP7305534B2/ja
Priority to JP2024140986A priority patent/JP2024167272A/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/30—Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0619—Neurons
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0662—Stem cells
    • C12N5/0663—Bone marrow mesenchymal stem cells (BM-MSC)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Neurosurgery (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
JP2019500283A 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 JP7305534B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022167518A JP7544785B2 (ja) 2016-07-06 2022-10-19 疼痛関連障害を処置するための物質及び方法
JP2024140986A JP2024167272A (ja) 2016-07-06 2024-08-22 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
US201662358769P 2016-07-06 2016-07-06
US62/358,769 2016-07-06
US201762461876P 2017-02-22 2017-02-22
US62/461,876 2017-02-22
PCT/IB2017/054081 WO2018007976A1 (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
JP2022167518A Division JP7544785B2 (ja) 2016-07-06 2022-10-19 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
JP2019520080A JP2019520080A (ja) 2019-07-18
JP2019520080A5 JP2019520080A5 (OSRAM) 2020-08-13
JP7305534B2 true JP7305534B2 (ja) 2023-07-10

Family

ID=59649966

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2019500283A Active JP7305534B2 (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
JP2022167518A Active JP7544785B2 (ja) 2016-07-06 2022-10-19 疼痛関連障害を処置するための物質及び方法
JP2024140986A Pending JP2024167272A (ja) 2016-07-06 2024-08-22 疼痛関連障害を処置するための物質及び方法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2022167518A Active JP7544785B2 (ja) 2016-07-06 2022-10-19 疼痛関連障害を処置するための物質及び方法
JP2024140986A Pending JP2024167272A (ja) 2016-07-06 2024-08-22 疼痛関連障害を処置するための物質及び方法

Country Status (8)

Country Link
US (2) US11801313B2 (OSRAM)
EP (2) EP4650364A2 (OSRAM)
JP (3) JP7305534B2 (OSRAM)
CN (2) CN110214149B (OSRAM)
AU (2) AU2017292169B2 (OSRAM)
CA (1) CA3029132A1 (OSRAM)
DK (1) DK3481856T3 (OSRAM)
WO (1) WO2018007976A1 (OSRAM)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3029132A1 (en) 2016-07-06 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of pain related disorders
US11459587B2 (en) 2016-07-06 2022-10-04 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
JP7109547B2 (ja) * 2018-02-15 2022-07-29 シグマ－アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニー 真核ゲノム修飾のための操作されたＣａｓ９システム
RU2706298C1 (ru) * 2018-09-14 2019-11-15 Закрытое Акционерное Общество "Биокад" НУКЛЕАЗА PaCas9
AU2020272031A1 (en) * 2019-04-12 2021-11-18 Vertex Pharmaceuticals Incorporated Gene-editing systems for modifying a SCN9A or SCN10A gene and methods of use thereof
EP4214319A4 (en) 2020-09-17 2025-12-03 Quiver Holdings Inc THERAPEUTIC COMPOSITIONS TO TREAT PAIN THROUGH MULTIPLE TARGETS
US20240318172A1 (en) * 2020-12-01 2024-09-26 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of peripheral myelin protein-22
WO2023022974A2 (en) * 2021-08-16 2023-02-23 Editas Medicine, Inc. Treatment of pain
CN116949179A (zh) * 2023-07-04 2023-10-27 中国医学科学院北京协和医院 结直肠肿瘤多基因遗传风险评分系统、存储介质和电子设备
US20250205352A1 (en) * 2023-12-13 2025-06-26 Eli Lilly And Company COMPOSITIONS AND METHODS FOR MODULATING NaV1.8

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015175642A2 (en) 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease
WO2016010148A1 (ja) 2014-07-18 2016-01-21 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

Family Cites Families (194)

* 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
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
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
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
CA1340032C (en) 1987-06-24 1998-09-08 Jim Haralambidis Lucleoside 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
WO1989009221A1 (en) 1988-03-25 1989-10-05 University Of Virginia Alumni Patents Foundation Oligonucleotide n-alkylphosphoramidates
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
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
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
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
DE69032425T2 (de) 1990-05-11 1998-11-26 Microprobe Corp., Bothell, Wash. Teststreifen zum Eintauchen für Nukleinsäure-Hybridisierungsassays und Verfahren zur kovalenten Immobilisierung von Oligonucleotiden
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5614617A (en) 1990-07-27 1997-03-25 Isis Pharmaceuticals, Inc. Nuclease resistant, pyrimidine modified oligonucleotides that detect and modulate gene expression
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
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
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
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
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
PT98562B (pt) 1990-08-03 1999-01-29 Sanofi Sa Processo para a preparacao de composicoes que compreendem sequencias de nucleo-sidos com cerca de 6 a cerca de 200 bases resistentes a nucleases
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
AU662298B2 (en) 1990-09-20 1995-08-31 Gilead Sciences, Inc. Modified internucleoside linkages
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
EP0556301B1 (en) 1990-11-08 2001-01-10 Hybridon, Inc. Incorporation of multiple reporter groups on synthetic oligonucleotides
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
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
CA2122030C (en) 1991-10-24 1997-03-04 Muthiah Manoharan Derivatized oligonucleotides having improved uptake and other properties
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl 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
ATE160572T1 (de) 1993-03-31 1997-12-15 Sanofi Sa Oligonucleotide mit amidverkettungen die phosphoesterverkettungen einsetzen
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
DE69433922T2 (de) 1993-11-09 2005-07-28 Medical College Of Ohio, Toledo Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
DK0733103T3 (da) 1993-11-09 2004-07-12 Univ Johns Hopkins Fremstilling af höje titre af rekombinante AAV vektorer
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
CA2230758A1 (en) 1995-09-08 1997-03-13 Genzyme Corporation Improved 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
EP1944362B1 (en) 1997-09-05 2015-10-21 Genzyme Corporation Methods for generating high titer helper-free preparations of recombinant AAV vectors
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
JP2004514407A (ja) 2000-04-28 2004-05-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
US20030158403A1 (en) 2001-07-03 2003-08-21 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
WO2003029459A2 (en) 2001-09-28 2003-04-10 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Micro-rna 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
EP1648914A4 (en) 2003-07-31 2009-12-16 Regulus Therapeutics Inc OLIGOMERIC COMPOUNDS AND COMPOSITIONS USEFUL FOR MODULATING SMALL NON-CODING RNA
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
ES2536423T3 (es) 2006-01-05 2015-05-25 The Ohio State University Research Foundation Métodos basados en microARN para el diagnóstico de cáncer de próstata
WO2007081680A2 (en) 2006-01-05 2007-07-19 The Ohio State University Research Foundation Microrna expression abnormalities in pancreatic endocrine and acinar tumors
EP1991706B1 (en) 2006-03-02 2014-01-15 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
EP2115138A2 (en) 2006-09-19 2009-11-11 Asuragen, Inc. Micrornas differentially expressed in pancreatic diseases and uses thereof
EP2087135B8 (en) 2006-11-01 2013-07-24 The Ohio State University Research Foundation 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
WO2008154098A2 (en) 2007-06-07 2008-12-18 Wisconsin Alumni Research Foundation Reagents and methods for mirna expression analysis and identification of cancer biomarkers
WO2009070653A1 (en) 2007-11-30 2009-06-04 The Ohio State University Research Foundation Microrna expression profiling and targeting in peripheral blood in lung cancer
EP2260110B1 (en) 2008-02-08 2014-11-12 Asuragen, INC. miRNAs DIFFERENTIALLY EXPRESSED IN LYMPH NODES FROM CANCER PATIENTS
JP2011517932A (ja) 2008-02-28 2011-06-23 ジ・オハイオ・ステイト・ユニバーシティ・リサーチ・ファウンデイション ヒト慢性リンパ球性白血病（ｃｃｌ）と関連するマイクロｒｎａシグネチャーおよびその使用
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
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
AU2010275432A1 (en) 2009-07-24 2012-02-02 Sigma-Aldrich Co. Llc. Method for genome editing
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
WO2011113030A2 (en) 2010-03-11 2011-09-15 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
CA2835179A1 (en) 2011-05-06 2012-11-15 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)
WO2013033640A1 (en) 2011-09-01 2013-03-07 Allegro Diagnostics Corp. Methods and compositions for detecting cancer based on mirna expression profiles
US20140243240A1 (en) 2011-10-26 2014-08-28 Georgetown University microRNA EXPRESSION PROFILING OF THYROID CANCER
CN103906746B (zh) * 2011-10-26 2015-12-09 辉瑞有限公司 用作钠通道调节剂的(4-苯基咪唑-2-基)乙胺衍生物
US9209196B2 (en) 2011-11-30 2015-12-08 Sharp Kabushiki Kaisha Memory circuit, method of driving the same, nonvolatile storage device using the same, and liquid crystal display device
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
MY189533A (en) 2012-05-25 2022-02-16 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
KR20230065381A (ko) 2012-07-25 2023-05-11 더 브로드 인스티튜트, 인코퍼레이티드 유도 ｄｎａ 결합 단백질 및 게놈 교란 도구 및 이의 적용
HK1220468A1 (zh) 2013-03-14 2017-05-05 Regeneron Pharmaceuticals, Inc. Nav1．7的人抗体
CN110885854A (zh) 2013-03-15 2020-03-17 北卡罗来纳-查佩尔山大学 双重聚糖结合aav载体的方法和组合物
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
AU2014329454B2 (en) 2013-10-03 2019-01-17 Janssen Biotech, Inc. Protoxin-II variants and methods of use
PT3066201T (pt) 2013-11-07 2018-06-04 Massachusetts Inst Technology Métodos e composições relacionadas com crispr com arng reguladores
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
JP6202701B2 (ja) 2014-03-21 2017-09-27 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プログラム
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
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
WO2017024319A1 (en) 2015-08-06 2017-02-09 Dana-Farber Cancer Institute, Inc. Tunable endogenous protein degradation
EP3365356B1 (en) 2015-10-23 2023-06-28 President and Fellows of Harvard College Nucleobase editors and uses thereof
WO2017075222A1 (en) 2015-10-30 2017-05-04 Lieber Institute For Brain Development 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
US11459587B2 (en) 2016-07-06 2022-10-04 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
CA3029132A1 (en) * 2016-07-06 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of pain related disorders
US20190240293A1 (en) 2016-07-26 2019-08-08 Flagship Pioneering Innovations V, Inc. Neuromodulating compositions and related therapeutic methods for the treatment of cancer by modulating an anti-cancer immune response
US20200340012A1 (en) 2016-08-18 2020-10-29 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system
CA3035910A1 (en) 2016-09-07 2018-03-15 Flagship Pioneering, Inc. Methods and compositions for modulating gene expression
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
US20200384226A1 (en) 2017-03-10 2020-12-10 Vanderbilt University Nasopharyngeal catheters and applications of same

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015175642A2 (en) 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease
WO2016010148A1 (ja) 2014-07-18 2016-01-21 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

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
FEBS Lett.,2014,588(21),p.3954-3958
J.Neurosci.,2013,33(35),p.14087-14097
Matthew D. Rannals, et al.，Psychiatric Risk Gene Transcription Factor 4 Regulates Intrinsic Excitability of Prefrontal Neurons via Repression of SCN10a and KCNQ1，Neuron，2016年04月06日，90(1)，p.43-55

Also Published As

Publication number Publication date
CN110214149A (zh) 2019-09-06
EP3481856B1 (en) 2025-09-03
JP2022189889A (ja) 2022-12-22
EP3481856A1 (en) 2019-05-15
DK3481856T3 (da) 2025-11-10
AU2017292169A1 (en) 2019-01-24
JP7544785B2 (ja) 2024-09-03
CN110214149B (zh) 2024-05-14
EP4650364A2 (en) 2025-11-19
WO2018007976A1 (en) 2018-01-11
AU2022202000B2 (en) 2025-01-23
CN119074961A (zh) 2024-12-06
JP2024167272A (ja) 2024-12-03
US20240066150A1 (en) 2024-02-29
AU2022202000A1 (en) 2022-04-14
US20190224340A1 (en) 2019-07-25
JP2019520080A (ja) 2019-07-18
AU2017292169B2 (en) 2021-12-23
US11801313B2 (en) 2023-10-31
CA3029132A1 (en) 2018-01-11

Similar Documents

Publication Publication Date Title
JP7719222B2 (ja) 疼痛関連障害を処置するための物質及び方法
JP7556092B2 (ja) 遺伝子編集のための組成物および方法
JP7277052B2 (ja) プロタンパク質転換酵素サブチリシン／ケキシン９型（ｐｃｓｋ９）関連障害の処置のための組成物および方法
JP7544785B2 (ja) 疼痛関連障害を処置するための物質及び方法
WO2018154459A1 (en) Materials and methods for treatment of primary hyperoxaluria type 1 (ph1) and other alanine-glyoxylate aminotransferase (agxt) gene related conditions or disorders
WO2018154413A1 (en) Materials and methods for treatment of dystrophic epidermolysis bullosa (deb) and other collagen type vii alpha 1 chain (col7a1) gene related conditions or disorders
AU2017290614A1 (en) Materials and methods for treatment of friedreich ataxia and other related disorders
EP3478841A1 (en) Materials and methods for treatment of apolipoprotein c3 (apociii)-related disorders

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200703

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20200703

A711 Notification of change in applicant

Free format text: JAPANESE INTERMEDIATE CODE: A711

Effective date: 20200901

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20200901

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210810

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20211109

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220107

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220419

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220719

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20221019

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20221220

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230420

C60 Trial request (containing other claim documents, opposition documents)

Free format text: JAPANESE INTERMEDIATE CODE: C60

Effective date: 20230420

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20230421

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20230515

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20230530

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20230628

R150 Certificate of patent or registration of utility model

Ref document number: 7305534

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150