JP7108307B2 - 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法 - Google Patents

遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法 Download PDF

Info

Publication number
JP7108307B2
JP7108307B2 JP2018547872A JP2018547872A JP7108307B2 JP 7108307 B2 JP7108307 B2 JP 7108307B2 JP 2018547872 A JP2018547872 A JP 2018547872A JP 2018547872 A JP2018547872 A JP 2018547872A JP 7108307 B2 JP7108307 B2 JP 7108307B2
Authority
JP
Japan
Prior art keywords
seq
nucleotide sequence
set forth
sequence set
targeting domain
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
JP2018547872A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018534950A (ja
JP2018534950A5 (enExample
Inventor
チャールズ エイ ガーズバック
ジャクリーン エヌ ロビンソン－ハム
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.)
Duke University
Original Assignee
Duke University
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 Duke University filed Critical Duke University
Publication of JP2018534950A publication Critical patent/JP2018534950A/ja
Publication of JP2018534950A5 publication Critical patent/JP2018534950A5/ja
Priority to JP2022110179A priority Critical patent/JP7517724B2/ja
Application granted granted Critical
Publication of JP7108307B2 publication Critical patent/JP7108307B2/ja
Priority to JP2024104063A priority patent/JP2024153629A/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

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
    • 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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0016—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/80—Vectors or expression systems specially adapted for eukaryotic hosts for fungi
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • C12N2015/8527—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic for producing animal models, e.g. for tests or diseases
    • 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]
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Environmental Sciences (AREA)
  • Immunology (AREA)
  • Analytical Chemistry (AREA)
  • Virology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Mycology (AREA)
  • Biodiversity & Conservation Biology (AREA)
JP2018547872A 2015-11-30 2016-11-30 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法 Active JP7108307B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022110179A JP7517724B2 (ja) 2015-11-30 2022-07-08 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法
JP2024104063A JP2024153629A (ja) 2015-11-30 2024-06-27 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562260712P 2015-11-30 2015-11-30
US62/260,712 2015-11-30
US201662330336P 2016-05-02 2016-05-02
US62/330,336 2016-05-02
PCT/US2016/064285 WO2017095967A2 (en) 2015-11-30 2016-11-30 Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022110179A Division JP7517724B2 (ja) 2015-11-30 2022-07-08 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法

Publications (3)

Publication Number Publication Date
JP2018534950A JP2018534950A (ja) 2018-11-29
JP2018534950A5 JP2018534950A5 (enExample) 2020-02-27
JP7108307B2 true JP7108307B2 (ja) 2022-07-28

Family

ID=58797730

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2018547872A Active JP7108307B2 (ja) 2015-11-30 2016-11-30 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法
JP2022110179A Active JP7517724B2 (ja) 2015-11-30 2022-07-08 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法
JP2024104063A Pending JP2024153629A (ja) 2015-11-30 2024-06-27 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2022110179A Active JP7517724B2 (ja) 2015-11-30 2022-07-08 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法
JP2024104063A Pending JP2024153629A (ja) 2015-11-30 2024-06-27 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法

Country Status (11)

Country Link
US (1) US12214054B2 (enExample)
EP (2) EP4644567A2 (enExample)
JP (3) JP7108307B2 (enExample)
KR (2) KR20250044471A (enExample)
CN (2) CN108779466B (enExample)
AU (1) AU2016362282B2 (enExample)
CA (1) CA3001623A1 (enExample)
EA (1) EA201891317A3 (enExample)
IL (1) IL259100B2 (enExample)
MX (3) MX2018005377A (enExample)
WO (1) WO2017095967A2 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022160426A (ja) * 2015-11-30 2022-10-19 デューク ユニバーシティ 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
JP6905755B2 (ja) 2015-08-25 2021-07-21 デューク ユニバーシティ Ｒｎａガイド型エンドヌクレアーゼを使用してゲノム工学における特異性を改善する組成物および方法
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US11369692B2 (en) 2015-10-28 2022-06-28 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of Duchenne Muscular Dystrophy
WO2017180915A2 (en) 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
ES2957660T3 (es) * 2016-05-05 2024-01-23 Univ Duke Composiciones relacionadas con crispr/cas para tratar la distrofia muscular de duchenne
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
JOP20190166A1 (ar) * 2017-01-05 2019-07-02 Univ Texas استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي
US10687520B2 (en) 2017-03-07 2020-06-23 The Board Of Regents Of The University Of Texas System Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44
EP3668983A1 (en) * 2017-08-18 2020-06-24 The Board of Regents of The University of Texas System Exon deletion correction of duchenne muscular dystrophy mutations in the dystrophin actin binding domain 1 using crispr genome editing
EP3707155A2 (en) * 2017-11-09 2020-09-16 Vertex Pharmaceuticals Incorporated Crispr/cas systems for treatment of dmd
US11891635B2 (en) 2017-12-21 2024-02-06 Max-Delbrück-Centrum Für Molekulare Medizin In Der Helmholtz-Gemeinschaft Nucleic acid sequence replacement by NHEJ
BR112020019079A2 (pt) * 2018-03-23 2020-12-29 Massachusetts Eye And Ear Infirmary Abordagem de salto de éxon mediada por crispr/cas9 para síndrome de usher associada a ush2a
WO2019209777A1 (en) 2018-04-23 2019-10-31 The Curators Of The University Of Missouri Improved crispr therapy
JP7788855B2 (ja) * 2018-12-12 2025-12-19 ソリッド・バイオサイエンシーズ・インコーポレーテッド 筋ジストロフィーの治療のための併用療法
WO2020210776A1 (en) * 2019-04-12 2020-10-15 Duke University Crispr/cas-based base editing composition for restoring dystrophin function
BR112021020515A2 (pt) * 2019-04-14 2022-01-04 Univ Duke Deleção mediada por vetor de aav de hotspot mutacional grande para o tratamento de distrofia muscular de duchenne
EP3966327A1 (en) * 2019-05-08 2022-03-16 Vertex Pharmaceuticals Incorporated Crispr/cas all-in-two vector systems for treatment of dmd
CN110499333A (zh) * 2019-08-01 2019-11-26 广州德赫生物科技有限公司 用于修复dmd基因突变的核酸序列及系统
US20240091379A1 (en) * 2019-10-11 2024-03-21 Yale University Compositions and methods for upregulating isoforms of dystrophin as therapy for duchenne muscular dystrophy (dmd)
KR20220093324A (ko) * 2019-11-07 2022-07-05 칭다오 킹아그루트 케미컬 컴파운드 컴퍼니 리미티드 유기체에서 새로운 돌연변이를 발생시키는 방법 및 그 활용
EP4125349A4 (en) * 2020-04-27 2024-07-10 Duke University GENE EDITING OF SATELLITE CELLS IN VIVO USING AAV VECTORS ENCODING MUSCLE-SPECIFIC PROMOTERS
EP4126224A4 (en) * 2020-04-27 2024-07-03 Duke University HIGH-THROUGHPUT SCREENING METHODS TO DISCOVER OPTIMAL GRNA PAIRS FOR CRISPR-MEDIATED EXON DELETION
CN112522256B (zh) * 2020-08-19 2023-08-22 南京启真基因工程有限公司 CRISPR/Cas9系统及其在构建抗肌萎缩蛋白基因缺陷的猪源重组细胞中的应用
EP4225907A4 (en) * 2020-10-12 2025-01-22 Duke University CRISPR/CAS-BASED BASE EDITING COMPOSITION TO RESTORE DYSTROPHIN FUNCTION
US20230392132A1 (en) * 2020-10-21 2023-12-07 Duke University Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
WO2022104062A1 (en) * 2020-11-12 2022-05-19 Precision Biosciences, Inc. Engineered meganucleases having specificity for recognition sequences in the dystrophin gene
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression
WO2024193704A1 (en) * 2023-03-22 2024-09-26 Huidagene Therapeutics Co., Ltd. Guide nucleic acids targeting dmd and uses thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1991018114A1 (en) 1990-05-22 1991-11-28 Medical Research Council Polynucleotide amplification
JP2016521555A (ja) 2013-06-05 2016-07-25 デューク ユニバーシティ Ｒｎａガイド遺伝子編集及び遺伝子調節

Family Cites Families (373)

* 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
US4554101A (en) 1981-01-09 1985-11-19 New York Blood Center, Inc. Identification and preparation of epitopes on antigens and allergens on the basis of hydrophilicity
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
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
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
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
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
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
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
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5405938A (en) 1989-12-20 1995-04-11 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
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
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
EP0260032B1 (en) 1986-09-08 1994-01-26 Ajinomoto Co., Inc. Compounds for the cleavage at a specific position of RNA, oligomers employed for the formation of said compounds, and starting materials for the synthesis of said oligomers
US5219740A (en) 1987-02-13 1993-06-15 Fred Hutchinson Cancer Research Center Retroviral gene transfer into diploid fibroblasts for gene therapy
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
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
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
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
WO1989005358A1 (en) 1987-11-30 1989-06-15 University Of Iowa Research Foundation Dna and rna molecules stabilized by modifications of the 3'-terminal phosphodiester linkage and their use as nucleic acid probes and as therapeutic agents to block the expression of specifically targeted genes
US5403711A (en) 1987-11-30 1995-04-04 University Of Iowa Research Foundation Nucleic acid hybridization and amplification method for detection of specific sequences in which a complementary labeled nucleic acid probe is cleaved
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
US5599923A (en) 1989-03-06 1997-02-04 Board Of Regents, University Of Tx Texaphyrin metal complexes having improved functionalization
US5457183A (en) 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
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
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences 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
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
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
US5623065A (en) 1990-08-13 1997-04-22 Isis Pharmaceuticals, Inc. Gapped 2' modified oligonucleotides
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US5220007A (en) 1990-02-15 1993-06-15 The Worcester Foundation For Experimental Biology Method of site-specific alteration of RNA and production of encoded polypeptides
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
DK0455905T3 (da) 1990-05-11 1998-12-07 Microprobe Corp Dipsticks til nukleinsyrehybridiseringsassays og fremgangsmåde til kovalent immobilisering af oligonukleotider
AU8284991A (en) 1990-06-29 1992-01-23 Regents Of The University Of Michigan, The Neurofibromatosis gene
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
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
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
WO1992002258A1 (en) 1990-07-27 1992-02-20 Isis Pharmaceuticals, Inc. Nuclease resistant, pyrimidine modified oligonucleotides that detect and modulate gene expression
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
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
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
EP0549686A4 (en) 1990-09-20 1995-01-18 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
DE69132510T2 (de) 1990-11-08 2001-05-03 Hybridon, Inc. Verbindung von mehrfachreportergruppen auf synthetischen oligonukleotiden
US5510473A (en) 1990-11-09 1996-04-23 The United States Of American As Represented By The Secretary Of Health And Human Services Cloning of the recA gene from thermus aquaticus YT-1
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
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
JP2823959B2 (ja) 1991-10-24 1998-11-11 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 改良された取り込みおよびその他の性質を持つ誘導化オリゴヌクレオチド
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US5700922A (en) 1991-12-24 1997-12-23 Isis Pharmaceuticals, Inc. PNA-DNA-PNA chimeric macromolecules
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
GB9206016D0 (en) 1992-03-19 1992-04-29 Sandoz Ltd Improvements in or relating to organic compounds
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
WO1993025673A1 (en) 1992-06-04 1993-12-23 The Regents Of The University Of California In vivo gene therapy with intron-free sequence of interest
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
US5652355A (en) 1992-07-23 1997-07-29 Worcester Foundation For Experimental Biology Hybrid oligonucleotide phosphorothioates
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
DK0681483T3 (da) 1993-01-26 2005-11-21 Univ Pennsylvania Præparater og fremgangsmåder til administration af genetisk materiale
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
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
WO1995015972A1 (en) 1993-12-09 1995-06-15 Thomas Jefferson University Compounds and methods for site-directed mutations in eukaryotic cells
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
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
US5962428A (en) 1995-03-30 1999-10-05 Apollon, Inc. Compositions and methods for delivery of genetic material
US5652356A (en) 1995-08-17 1997-07-29 Hybridon, Inc. Inverted chimeric and hybrid oligonucleotides
DE19608753C1 (de) 1996-03-06 1997-06-26 Medigene Gmbh Transduktionssystem und seine Verwendung
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
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
CA2263784A1 (en) 1998-03-23 1999-09-23 Megabios Corporation Dual-tagged proteins and their uses
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US6734291B2 (en) 1999-03-24 2004-05-11 Exiqon A/S Synthesis of [2.2.1]bicyclo nucleosides
KR100782896B1 (ko) 1999-05-04 2007-12-06 엑시콘 에이/에스 Ｌ-리보-ｌｎａ 유사체
US20040192593A1 (en) 1999-07-26 2004-09-30 Baylor College Of Medicine Protease resistant ti-growth hormone releasing hormone
US6287860B1 (en) 2000-01-20 2001-09-11 Isis Pharmaceuticals, Inc. Antisense inhibition of MEKK2 expression
AU2001253812A1 (en) 2000-04-28 2001-11-12 Genzyme Corporation In vivo loading of mhc
ATE353361T1 (de) 2000-04-28 2007-02-15 Sangamo Biosciences Inc Gezielten modifikation der chromatinstruktur
JP4860886B2 (ja) 2000-06-01 2012-01-25 ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 二本鎖パルボウイルスベクター
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US7879540B1 (en) 2000-08-24 2011-02-01 Promega Corporation Synthetic nucleic acid molecule compositions and methods of preparation
US20040224385A1 (en) 2001-08-20 2004-11-11 Barbas Carlos F Zinc finger binding domains for cnn
EP1446412B1 (en) 2001-09-04 2012-03-07 Exiqon A/S Novel lna compositions and uses thereof
AU2003219847A1 (en) 2002-02-21 2003-09-09 The Wistar Institute Of Anatomy And Biology Methods and compositions for reversibly controlling expression of target genes in cells
US7449561B1 (en) 2002-02-26 2008-11-11 City Of Hope Alterations in the dystrophin gene associated with sporadic dilated cardiomyopathy
US7074596B2 (en) 2002-03-25 2006-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse mRNA cap analogues
US7901708B2 (en) 2002-06-28 2011-03-08 Protiva Biotherapeutics, Inc. Liposomal apparatus and manufacturing methods
US20040175727A1 (en) 2002-11-04 2004-09-09 Advisys, Inc. Synthetic muscle promoters with activities exceeding naturally occurring regulatory sequences in cardiac cells
US20070185042A1 (en) 2003-08-08 2007-08-09 The Brigham And Women's Hospital, Inc. President And Fellows Of Harvard College Sirna based methods for treating alzheimer's disease
CA2539439C (en) 2003-09-19 2012-10-23 Sangamo Biosciences, Inc. Engineered zinc finger proteins for regulation of gene expression
US20070192880A1 (en) 2003-10-03 2007-08-16 University Of Rochester Horming response element binding transregulators
US8507277B2 (en) 2003-10-24 2013-08-13 Gencia Corporation Nonviral vectors for delivering polynucleotides
US7799565B2 (en) 2004-06-07 2010-09-21 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
EP1781593B1 (en) 2004-06-07 2011-12-14 Protiva Biotherapeutics Inc. Cationic lipids and methods of use
US7728118B2 (en) 2004-09-17 2010-06-01 Promega Corporation Synthetic nucleic acid molecule compositions and methods of preparation
CA2616877C (en) 2005-07-27 2014-01-28 Protiva Biotherapeutics, Inc. Systems and methods for manufacturing liposomes
KR20080031504A (ko) 2005-08-05 2008-04-08 미시간 스테이트 유니버시티 악티노바실러스 숙시노제네스의 ｃ4-경로로부터 화학물질을생산하기 위해 악티노바실러스 숙시노제네스130ｚ(ａｔｃｃ 55618)로부터 얻은 유전자
US7943374B2 (en) 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
EP4174179B1 (en) 2005-08-23 2025-05-07 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
KR20090031938A (ko) 2006-07-05 2009-03-30 더 스크립스 리서치 인스티튜트 방향성 진화로 촉매 작용을 최적화시킨 키메라 징크 핑거 리컴비나제
WO2008073303A2 (en) 2006-12-07 2008-06-19 Switchgear Genomics Transcriptional regulatory elements of biological pathways, tools, and methods
WO2008070859A2 (en) 2006-12-07 2008-06-12 The Government Of The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Treatment of skin conditions by dickkopf1 (dkk1)
EP2118118B1 (en) 2007-01-19 2017-09-27 Exiqon A/S Mediated cellular delivery of lna oligonucleotides
US20100261175A1 (en) 2007-06-15 2010-10-14 Exiqon A/S Use of short oligonucleotides for reagent redundancy experiments in rna functional analysis
EP2614827B1 (en) 2007-10-26 2017-06-28 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
CA2740000C (en) 2008-10-09 2017-12-12 Tekmira Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
CN103725717A (zh) 2008-10-17 2014-04-16 焦耳无限科技公司 微生物的乙醇生产
EP4174178A1 (en) 2008-10-24 2023-05-03 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for dmd
WO2010075424A2 (en) 2008-12-22 2010-07-01 The Regents Of University Of California Compositions and methods for downregulating prokaryotic genes
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
WO2010108126A2 (en) 2009-03-19 2010-09-23 Fate Therapeutics, Inc. Reprogramming compositions and methods of using the same
NZ622843A (en) 2009-06-10 2015-10-30 Tekmira Pharmaceuticals Corp Improved lipid formulation
EP2281579A1 (en) 2009-08-05 2011-02-09 BioNTech AG Vaccine composition comprising 5'-Cap modified RNA
EP2480659A2 (en) 2009-09-24 2012-08-01 Cellectis Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations
SG10201702120VA (en) 2009-10-31 2017-05-30 Genesis Technologies Ltd Methods for Reprogramming Cells and Uses Thereof
DK2510096T4 (en) 2009-12-10 2018-05-14 Univ Iowa State Res Found Inc NUMBER EFFECTOR-MEDIATED DNA MODIFICATION
EP2533629B1 (en) 2010-02-11 2018-11-28 Recombinetics, Inc. Methods and materials for producing transgenic artiodactyls
KR101953237B1 (ko) 2010-05-17 2019-02-28 상가모 테라퓨틱스, 인코포레이티드 신규 dna 결합 단백질 및 이의 용도
IT1400425B1 (it) 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
EP2517731A1 (en) 2011-04-07 2012-10-31 Ludwig-Maximilians-Universität München Method of activating a target gene in a cell
US9540623B2 (en) 2011-07-08 2017-01-10 Cellectis Method for increasing the efficiency of double-strand-break induced mutagenesis
WO2013074999A1 (en) 2011-11-16 2013-05-23 Sangamo Biosciences, Inc. Modified dna-binding proteins and uses thereof
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
EP4043039A1 (en) 2012-01-27 2022-08-17 BioMarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
US20130274129A1 (en) 2012-04-04 2013-10-17 Geneart Ag Tal-effector assembly platform, customized services, kits and assays
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
US9738879B2 (en) 2012-04-27 2017-08-22 Duke University Genetic correction of mutated genes
US10266850B2 (en) 2012-05-25 2019-04-23 The Regents Of The University Of California Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
US9890364B2 (en) 2012-05-29 2018-02-13 The General Hospital Corporation TAL-Tet1 fusion proteins and methods of use thereof
KR20230065381A (ko) 2012-07-25 2023-05-11 더 브로드 인스티튜트, 인코퍼레이티드 유도 ｄｎａ 결합 단백질 및 게놈 교란 도구 및 이의 적용
US9567581B2 (en) 2012-08-07 2017-02-14 The General Hospital Corporation Selective reactivation of genes on the inactive X chromosome
EP2906602B1 (en) 2012-10-12 2019-01-16 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
CN110669758A (zh) 2012-10-23 2020-01-10 基因工具股份有限公司 用于切割靶dna 的组合物及其用途
BR122019025678B1 (pt) 2012-11-01 2023-04-18 Factor Bioscience Inc Composições que compreendem ácido nucleico que codifica uma proteína de edição gênica
US20140140969A1 (en) 2012-11-20 2014-05-22 Sangamo Biosciences, Inc. Methods and compositions for muscular dystrophies
CA3034794A1 (en) 2012-12-06 2014-06-12 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
EP3434776A1 (en) 2012-12-12 2019-01-30 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
CN105121648B (zh) 2012-12-12 2021-05-07 布罗德研究所有限公司 用于序列操纵的系统、方法和优化的指导组合物的工程化
EP4234696A3 (en) 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
RU2721275C2 (ru) 2012-12-12 2020-05-18 Те Брод Инститьют, Инк. Доставка, конструирование и оптимизация систем, способов и композиций для манипуляции с последовательностями и применения в терапии
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
EP2931891B1 (en) 2012-12-17 2019-05-15 President and Fellows of Harvard College Rna-guided human genome engineering
KR102271291B1 (ko) 2013-03-15 2021-07-02 더 제너럴 하스피탈 코포레이션 특정 게놈 좌위에 대한 유전적 및 후성적 조절 단백질의 rna-안내 표적화
JP2016516066A (ja) * 2013-03-15 2016-06-02 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィを処置するための改善された組成物
CA3159715A1 (en) 2013-03-15 2014-09-18 Edward J. Britt Energy conversion device and method for making and using same
JP2016521975A (ja) 2013-05-15 2016-07-28 サンガモ バイオサイエンシーズ， インコーポレイテッド 遺伝的状態の処置のための方法および組成物
US9267135B2 (en) 2013-06-04 2016-02-23 President And Fellows Of Harvard College RNA-guided transcriptional regulation
AU2014274939B2 (en) 2013-06-04 2020-03-19 President And Fellows Of Harvard College RNA-guideded transcriptional regulation
EP3011035B1 (en) 2013-06-17 2020-05-13 The Broad Institute, Inc. Assay for quantitative evaluation of target site cleavage by one or more crispr-cas guide sequences
BR112015031608A2 (pt) 2013-06-17 2017-08-22 Massachusetts Inst Technology Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos
AU2014281030B2 (en) 2013-06-17 2020-07-09 Massachusetts Institute Of Technology Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
SI3019619T1 (sl) 2013-07-11 2021-12-31 Modernatx, Inc. Sestave, ki zajemajo sintetične polinukleotide, ki kodirajo proteine, pozvezane s crispr, in sintetične sgrna, ter metode uporabe
US10421957B2 (en) 2013-07-29 2019-09-24 Agilent Technologies, Inc. DNA assembly using an RNA-programmable nickase
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
WO2015021426A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. A crispr/cas system-based novel fusion protein and its application in genome editing
EP3040414B1 (en) 2013-08-29 2018-12-12 Norimasa Miura Biomolecular group related to cell anti-aging
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US10822606B2 (en) 2013-09-27 2020-11-03 The Regents Of The University Of California Optimized small guide RNAs and methods of use
EP3066201B1 (en) 2013-11-07 2018-03-07 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
MX374529B (es) 2013-12-12 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para edicion del genoma.
EP3080257A1 (en) 2013-12-12 2016-10-19 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
AU2014361834B2 (en) 2013-12-12 2020-10-22 Massachusetts Institute Of Technology CRISPR-Cas systems and methods for altering expression of gene products, structural information and inducible modular Cas enzymes
WO2015114059A1 (en) 2014-01-29 2015-08-06 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Trans-differentiation of differentiated cells
DK3116997T3 (da) 2014-03-10 2019-08-19 Editas Medicine Inc Crispr/cas-relaterede fremgangsmåder og sammensætninger til behandling af lebers kongenitale amaurose 10 (lca10)
JP2017512481A (ja) 2014-04-08 2017-05-25 ノースカロライナ ステート ユニバーシティーＮｏｒｔｈ Ｃａｒｏｌｉｎａ Ｓｔａｔｅ Ｕｎｉｖｅｒｓｉｔｙ Ｃｒｉｓｐｒ関連遺伝子を用いた、ｒｎａ依存性の転写抑制のための方法および組成物
KR102827558B1 (ko) 2014-04-18 2025-07-11 에디타스 메디신, 인코포레이티드 암 면역요법을 위한 crispr-cas-관련 방법, 조성물 및 구성성분
CN106852157B (zh) 2014-06-16 2022-04-12 约翰斯·霍普金斯大学 用于使用h1启动子表达crispr向导rna的组合物和方法
CA2954791C (en) 2014-07-14 2025-11-18 The Regents Of The University Of California CRISPR/CAS TRANSCRIPTIONAL MODULATION
WO2016011070A2 (en) 2014-07-14 2016-01-21 The Regents Of The University Of California A protein tagging system for in vivo single molecule imaging and control of gene transcription
WO2016022866A1 (en) 2014-08-07 2016-02-11 Agilent Technologies, Inc. Cis-blocked guide rna
WO2016025469A1 (en) 2014-08-11 2016-02-18 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by crispr/cas9-mediated gene editing
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
GB201418965D0 (enExample) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
MA40880A (fr) 2014-10-30 2017-09-05 Temple Univ Of The Commonwealth Éradication guidée par l'arn du virus jc humain et d'autres polyomavirus
US11319555B2 (en) 2014-11-20 2022-05-03 Duke University Compositions, systems and methods for cell therapy
CA3176380A1 (en) 2014-11-21 2016-05-26 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide rnas
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
US10190106B2 (en) 2014-12-22 2019-01-29 Univesity Of Massachusetts Cas9-DNA targeting unit chimeras
US11208638B2 (en) 2015-01-12 2021-12-28 The Regents Of The University Of California Heterodimeric Cas9 and methods of use thereof
KR102605464B1 (ko) 2015-01-30 2023-11-22 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 일차 조혈 세포에서의 단백질 전달
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
US20160281166A1 (en) 2015-03-23 2016-09-29 Parabase Genomics, Inc. Methods and systems for screening diseases in subjects
EP3277816B1 (en) * 2015-04-01 2020-06-17 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy
JP2018512876A (ja) 2015-04-22 2018-05-24 ミナ セラピューティクス リミテッド ｓａＲＮＡ組成物および使用方法
US11180793B2 (en) 2015-04-24 2021-11-23 Editas Medicine, Inc. Evaluation of Cas9 molecule/guide RNA molecule complexes
WO2016187717A1 (en) 2015-05-26 2016-12-01 Exerkine Corporation Exosomes useful for genome editing
WO2016205613A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzyme mutations reducing off-target effects
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2017016915A1 (en) 2015-07-24 2017-02-02 Basf Se Pyridine compounds useful for combating phytopathogenic fungi
WO2017069829A2 (en) 2015-07-31 2017-04-27 The Trustees Of Columbia University In The City Of New York High-throughput strategy for dissecting mammalian genetic interactions
JP6905755B2 (ja) 2015-08-25 2021-07-21 デューク ユニバーシティ Ｒｎａガイド型エンドヌクレアーゼを使用してゲノム工学における特異性を改善する組成物および方法
US11091780B2 (en) 2015-09-18 2021-08-17 The Regents Of The University Of California Methods for autocatalytic genome editing and neutralizing autocatalytic genome editing and compositions thereof
US20180265859A1 (en) 2015-09-23 2018-09-20 UNIVERSITé LAVAL Modification of the dystrophin gene and uses thereof
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof
US11369692B2 (en) 2015-10-28 2022-06-28 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of Duchenne Muscular Dystrophy
EP3368689B1 (en) 2015-10-28 2020-06-17 The Broad Institute, Inc. Composition for modulating immune responses by use of immune cell gene signature
WO2017081288A1 (en) 2015-11-11 2017-05-18 Lonza Ltd Crispr-associated (cas) proteins with reduced immunogenicity
WO2017083766A1 (en) 2015-11-13 2017-05-18 Massachusetts Institute Of Technology High-throughput crispr-based library screening
CN108779466B (zh) 2015-11-30 2024-03-29 杜克大学 用于通过基因编辑修正人肌营养不良蛋白基因的治疗靶标和使用方法
NZ743983A (en) 2016-01-11 2025-08-29 Univ Leland Stanford Junior Chimeric proteins and methods of regulating gene expression
EP3199632A1 (en) 2016-01-26 2017-08-02 ACIB GmbH Temperature-inducible crispr/cas system
WO2017139505A2 (en) 2016-02-11 2017-08-17 The Regents Of The University Of California Methods and compositions for modifying a mutant dystrophin gene in a cell's genome
WO2017165859A1 (en) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
WO2017180915A2 (en) 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
EP3443108A4 (en) 2016-04-15 2019-11-20 The Trustees of The University of Pennsylvania NOVEL AAV8 MUTANT CAPSIDES AND COMPOSITIONS THEREOF
ES2979174T3 (es) 2016-04-15 2024-09-24 Res Institute At Nationwidechildrens Hospital Suministro de vectores de virus adenoasociados de â-sarcoglicano y microrna-29 y tratamiento de la distrofia muscular
ES2957660T3 (es) 2016-05-05 2024-01-23 Univ Duke Composiciones relacionadas con crispr/cas para tratar la distrofia muscular de duchenne
WO2017201199A1 (en) 2016-05-17 2017-11-23 Duke University Compositions and methods for the treatment of prader-willi syndrome
US20170362635A1 (en) 2016-06-20 2017-12-21 University Of Washington Muscle-specific crispr/cas9 editing of genes
US11427838B2 (en) 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
TW201811807A (zh) 2016-06-30 2018-04-01 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
US11674158B2 (en) 2016-07-15 2023-06-13 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells
EP3484526A4 (en) 2016-07-18 2020-04-15 Jaan Biotherapeutics, LLC COMPOSITIONS AND METHODS FOR THE TREATMENT OF HEART DISEASE
US20190235624A1 (en) 2016-07-19 2019-08-01 Supereye, Inc. Systems and methods for predictive visual rendering
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
AU2017306676B2 (en) 2016-08-03 2024-02-22 President And Fellows Of Harvard College Adenosine nucleobase editors and uses thereof
EP4485466A3 (en) 2016-08-17 2025-04-02 The Broad Institute Inc. Novel crispr enzymes and systems
AU2017313912B2 (en) 2016-08-19 2024-01-04 Whitehead Institute For Biomedical Research Methods of editing DNA methylation
BR112019003124A2 (pt) 2016-08-19 2019-10-01 Toolgen Incorporated sistema regulador de angiogênse artificialmente engendrado
EP3500677A4 (en) 2016-08-20 2020-04-01 Avellino Lab USA, Inc. Single guide rna, crispr/cas9 systems, and methods of use thereof
WO2018071868A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
EP3532616A1 (en) 2016-10-28 2019-09-04 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus
CN110382695A (zh) 2016-11-28 2019-10-25 得克萨斯州大学系统董事会 通过crispr/cpf1介导的基因编辑来预防肌营养不良
CA3045335A1 (en) 2016-12-01 2018-06-07 Universite Laval Crispr-based treatment of friedreich ataxia
JP2020500541A (ja) 2016-12-08 2020-01-16 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム ヒト化デュシェンヌ型筋ジストロフィー変異を有するｄｍｄレポーターモデル
JOP20190166A1 (ar) 2017-01-05 2019-07-02 Univ Texas استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي
US11136555B2 (en) 2017-03-01 2021-10-05 Elixirgen Scientific, Inc. Compositions and methods for differentiation of human pluripotent stem cells into desired cell types
WO2018160908A1 (en) 2017-03-03 2018-09-07 Flagship Pioneering, Inc. Methods and systems for modifying dna
US10687520B2 (en) 2017-03-07 2020-06-23 The Board Of Regents Of The University Of Texas System Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44
US20200010519A1 (en) 2017-03-10 2020-01-09 Institut National De La Sante Et De La Recherche Medicale(Inserm) Nuclease fusions for enhancing genome editing by homology-directed transgene integration
CA3057320A1 (en) 2017-03-23 2018-09-27 DNARx Systems and methods for nucleic acid expression in vivo
AU2017407272B2 (en) 2017-03-30 2024-06-13 Kyoto University Method for inducing exon skipping by genome editing
KR20240155360A (ko) 2017-04-12 2024-10-28 더 브로드 인스티튜트, 인코퍼레이티드 신규 타입 vi crispr 오르소로그 및 시스템
US20180305719A1 (en) 2017-04-19 2018-10-25 The Board Of Trustees Of The University Of Illinois Vectors For Integration Of DNA Into Genomes And Methods For Altering Gene Expression And Interrogating Gene Function
WO2018208998A1 (en) 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9
EP3420811A1 (en) 2017-06-29 2019-01-02 Paris Sciences et Lettres - Quartier Latin Non-human model for neurofibromatosis type 1
JP7562931B2 (ja) 2017-07-07 2024-10-08 ツールゲン インコーポレイテッド 標的特異的なｃｒｉｓｐｒバリアント
WO2019014122A1 (en) 2017-07-08 2019-01-17 The Brigham And Women's Hospital, Inc. METHODS FOR ENHANCING ANTIANGIOGENIC THERAPY AND IMMUNOTHERAPY
CN109295053B (zh) 2017-07-25 2023-12-22 中国科学院上海营养与健康研究所 通过诱导剪接位点碱基突变或多聚嘧啶区碱基置换调控rna剪接的方法
WO2019023291A2 (en) 2017-07-25 2019-01-31 Dana-Farber Cancer Institute, Inc. COMPOSITIONS AND METHODS FOR PRODUCTION AND DECODING OF GUIDE RNA LIBRARIES AND USES THEREOF
EP3441461A1 (en) 2017-08-11 2019-02-13 Baylor College of Medicine Cd1d-restricted nkt cells as a platform for off-the-shelf cancer immunotherapy
EP3668983A1 (en) 2017-08-18 2020-06-24 The Board of Regents of The University of Texas System Exon deletion correction of duchenne muscular dystrophy mutations in the dystrophin actin binding domain 1 using crispr genome editing
EP3675836A4 (en) 2017-08-31 2021-05-26 Sarepta Therapeutics, Inc. METHODS FOR TREATING MUSCLE DYSTROPHY
EP3687548A4 (en) 2017-09-29 2021-05-26 The Regents of the University of California MULTI-ARM POLYROTAXANE PLATFORM FOR PROTECTED NUCLEIC ACID ADMINISTRATION
WO2019079514A1 (en) 2017-10-17 2019-04-25 Massachusetts Institute Of Technology METHODS OF FUNCTIONAL DISSECTION AT THE GENOME SCALE AT HIGH RESOLUTION OF TRANSCRIPTIONAL REGULATORY REGIONS
WO2019077001A1 (en) 2017-10-19 2019-04-25 Curevac Ag NEW ARTIFICIAL NUCLEIC ACID MOLECULES
CN120290560A (zh) 2017-10-23 2025-07-11 斯托克制药公司 用于治疗基于无义介导的rna衰变的病况和疾病的反义寡聚体
EP3707155A2 (en) 2017-11-09 2020-09-16 Vertex Pharmaceuticals Incorporated Crispr/cas systems for treatment of dmd
CA3084810A1 (en) 2017-12-08 2019-06-13 University Of Connecticut Compositions and methods for treating disorders of genomic imprinting
BR112020011893A2 (pt) 2017-12-15 2020-11-24 The Board Of Trustees Of The Leland Stanford Junior University composições e métodos para inibir a exaustão de células t
US11293019B2 (en) 2017-12-22 2022-04-05 Gflas Life Sciences, Inc. Chimeric genome engineering molecules and methods
US12031130B2 (en) 2017-12-28 2024-07-09 Takeda Pharmaceutical Company Limited Composition for modifying target gene
EP3735462A1 (en) 2018-01-05 2020-11-11 The Board of Regents of The University of Texas System Therapeutic crispr/cas9 compositions and methods of use
US20220010293A1 (en) 2018-02-23 2022-01-13 Pioneer Hi-Bred International, Inc. Novel cas9 orthologs
US20210254049A1 (en) 2018-04-20 2021-08-19 Cellino Biotech, Inc. Directed cell fate specification and targeted maturation
WO2019213626A1 (en) 2018-05-03 2019-11-07 President And Fellows Of Harvard College In vivo homology directed repair in heart, skeletal muscle, and muscle stem cells
EP3802802A4 (en) 2018-05-30 2023-04-19 Tune Therapeutics, Inc. CELLULAR THERAPY
WO2020018918A1 (en) 2018-07-19 2020-01-23 The Board Of Trustees Of The University Of Illinois Methods for exon skipping and gene knockout using base editors
WO2020132226A1 (en) 2018-12-20 2020-06-25 Ohio State Innovation Foundation Compositions and methods for reprogramming diseased musculoskeletal cells
BR102019009665A2 (pt) 2018-12-21 2022-02-08 Jacques P. Tremblay Modificação da proteína precursora beta amiloide (app) através da edição de base usando o sistema crispr/cas9
US11946040B2 (en) 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
AU2020223060B2 (en) 2019-02-13 2023-04-13 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
WO2020210776A1 (en) 2019-04-12 2020-10-15 Duke University Crispr/cas-based base editing composition for restoring dystrophin function
BR112021020515A2 (pt) 2019-04-14 2022-01-04 Univ Duke Deleção mediada por vetor de aav de hotspot mutacional grande para o tratamento de distrofia muscular de duchenne
EP3930766A4 (en) 2019-04-14 2023-02-22 Duke University CRISPR/CAS-BASED GENE EDITING COMPOSITION TO RESTORE DYSTROPHIN FUNCTION
US20220249528A1 (en) 2019-06-20 2022-08-11 Celgene Corporation Azacitidine in combination with venetoclax, gilteritinib, midostaurin or other compounds for treating leukemia or myelodysplastic syndrome
WO2021026516A1 (en) 2019-08-08 2021-02-11 Duke University High-throughput screening platform for engineering next-generation gene therapy vectors
EP4017971A4 (en) 2019-08-19 2023-09-13 Duke University COMPOSITIONS AND METHODS FOR IDENTIFYING REGULATORS FOR DETERMINING THE FATE OF CELL SPECIES
WO2021034984A2 (en) 2019-08-19 2021-02-25 Duke University Skeletal myoblast progenitor cell lineage specification by crispr/cas9-based transcriptional activators
WO2021055956A1 (en) 2019-09-20 2021-03-25 The Uab Research Foundation Methods of treatment of neurofibromatosis type 1 (nf1) and nf1-mediated conditions and compositions for use in such methods
WO2021067878A1 (en) 2019-10-02 2021-04-08 Duke University Epigenetic modulation of genomic targets to control expression of pws-associated genes
EP4069314A4 (en) 2019-12-03 2024-06-26 Duke University SYSTEMS AND METHODS FOR LIPID NANOPARTICLE DELIVERY OF GENE EDITING MACHINERY
EP4126224A4 (en) 2020-04-27 2024-07-03 Duke University HIGH-THROUGHPUT SCREENING METHODS TO DISCOVER OPTIMAL GRNA PAIRS FOR CRISPR-MEDIATED EXON DELETION
EP4125349A4 (en) 2020-04-27 2024-07-10 Duke University GENE EDITING OF SATELLITE CELLS IN VIVO USING AAV VECTORS ENCODING MUSCLE-SPECIFIC PROMOTERS
US20230201375A1 (en) 2020-04-27 2023-06-29 Duke University Targeted genomic integration to restore neurofibromin coding sequence in neurofibromatosis type 1 (nf1)
EP4200406A1 (en) 2020-08-21 2023-06-28 Julius-Maximilians-Universität Würzburg Modified lymphocytes
US20230383297A1 (en) 2020-10-09 2023-11-30 Duke University Novel targets for reactivation of prader-willi syndrome-associated genes
EP4225907A4 (en) 2020-10-12 2025-01-22 Duke University CRISPR/CAS-BASED BASE EDITING COMPOSITION TO RESTORE DYSTROPHIN FUNCTION
US20230392132A1 (en) 2020-10-21 2023-12-07 Duke University Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
EP4244344A4 (en) 2020-11-11 2024-10-16 The Trustees Of Columbia University In The City Of New York MULTIPLEX EPIGENOME EDITION
CA3201631A1 (en) 2020-11-13 2022-05-19 Duke University Targeted gene regulation of human immune cells with crispr-cas systems
WO2022133062A1 (en) 2020-12-16 2022-06-23 Epicrispr Biotechnologies, Inc. Systems and methods for engineering characteristics of a cell
WO2022187288A2 (en) 2021-03-01 2022-09-09 Duke University Systems and methods for genome-wide annotation of gene regulatory elements linked to cell fitness
CA3227105A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
US20240254483A1 (en) 2021-07-30 2024-08-01 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
US20250154503A1 (en) 2022-01-14 2025-05-15 Tune Therapeutics, Inc. Compositions, systems, and methods for programming t cell phenotypes through targeted gene repression
US20250134999A1 (en) 2022-01-14 2025-05-01 Tune Therapeutics, Inc. Compositions, systems, and methods for programming t cell phenotypes through targeted gene activation
US20240058425A1 (en) 2022-03-08 2024-02-22 Duke University Systems and methods for genome-wide annotation of gene regulatory elements linked to cell fitness
EP4508096A2 (en) 2022-04-13 2025-02-19 Duke University Effector domains for crispr-cas systems
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression
CA3261865A1 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS
WO2024040253A1 (en) 2022-08-18 2024-02-22 Duke University Epigenetic modulation of genomic targets to control expression of pws-associated genes
WO2024040254A2 (en) 2022-08-19 2024-02-22 Tune Therapeutics, Inc. Compositions, systems, and methods for regulation of hepatitis b virus through targeted gene repression
CN120239746A (zh) 2022-09-19 2025-07-01 图恩疗法股份有限公司 用于调节t细胞功能的组合物、系统和方法
WO2024081937A2 (en) 2022-10-13 2024-04-18 Duke University Cas12a fusion proteins and methods of using same

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1991018114A1 (en) 1990-05-22 1991-11-28 Medical Research Council Polynucleotide amplification
JP2016521555A (ja) 2013-06-05 2016-07-25 デューク ユニバーシティ Ｒｎａガイド遺伝子編集及び遺伝子調節

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
OUSTEROUT, D.G., et al.，"Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy."，NATURE COMMUNICATIONS，2015年02月18日，Vol.6，6244(pp.1-13)，[online], doi: 10.1038/ncomms7244
WOOD, H.，"Neuromuscular disease: CRISPR/Cas9 gene-editing platform corrects mutations associated with Duchenne muscular dystrophy."，NATURE REVIEW NEUROLOGY，2015年03月10日，Vol.11, No.4，184，[online], doi: 10.1038/nrneurol.2015.37

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022160426A (ja) * 2015-11-30 2022-10-19 デューク ユニバーシティ 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法
JP7517724B2 (ja) 2015-11-30 2024-07-17 デューク ユニバーシティ 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法

Also Published As

Publication number Publication date
EP3384055B1 (en) 2025-07-16
US20180353615A1 (en) 2018-12-13
MX2024004386A (es) 2024-04-29
CA3001623A1 (en) 2017-06-08
AU2016362282B2 (en) 2023-03-16
JP7517724B2 (ja) 2024-07-17
JP2024153629A (ja) 2024-10-29
CN118147141A (zh) 2024-06-07
KR20250044471A (ko) 2025-03-31
BR112018011133A2 (en) 2018-11-21
CN108779466A (zh) 2018-11-09
MX2018005377A (es) 2018-11-09
NZ741354A (en) 2025-03-28
IL259100A (en) 2018-06-28
EP4644567A2 (en) 2025-11-05
AU2016362282A1 (en) 2018-05-10
EP3384055A4 (en) 2019-04-17
EA201891317A2 (ru) 2018-12-28
IL259100B2 (en) 2023-09-01
NZ782841A (en) 2025-03-28
KR102787119B1 (ko) 2025-03-27
IL259100B1 (en) 2023-05-01
JP2022160426A (ja) 2022-10-19
JP2018534950A (ja) 2018-11-29
KR20180081618A (ko) 2018-07-16
US12214054B2 (en) 2025-02-04
WO2017095967A2 (en) 2017-06-08
EA201891317A3 (ru) 2019-04-30
EP3384055A2 (en) 2018-10-10
MX2024013018A (es) 2024-11-08
CN108779466B (zh) 2024-03-29

Similar Documents

Publication Publication Date Title
JP7517724B2 (ja) 遺伝子編集によるヒトジストロフィン遺伝子の修正用の治療標的および使用方法
JP7633699B2 (ja) Ｃｐｆ１に基づくゲノム編集の治療適用
TWI887234B (zh) 治療杜興氏（ｄｕｃｈｅｎｎｅ）肌肉失養症之大型突變熱點之ａａｖ載體介導之刪除
EP3452498B1 (en) Crispr/cas-related compositions for treating duchenne muscular dystrophy
WO2019209777A1 (en) Improved crispr therapy
US20230392132A1 (en) Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
HK40112232A (zh) 用於通过基因编辑修正人肌营养不良蛋白基因的治疗靶标和使用方法
EA049120B1 (ru) Опосредуемая aav-вектором делеция крупной мутационной "горячей точки" для лечения мышечной дистрофии дюшенна
BR112018011133B1 (pt) Composições, polinucleotídeo isolado, vetores e kit
BR122024013043A2 (pt) Rna-guia, polinucleotídeo isolado, vetores, células, composição, kit e usos do rna-guia para corrigir um gene mutante de distrofina, editar o genoma de um gene mutante de distrofina e tratar um gene mutante de distrofina
EA042798B1 (ru) Терапевтические мишени для коррекции гена дистрофина человека с помощью редактирования генов и способы их применения
HK40004669B (en) Therapeutic applications of cpf1-based genome editing
HK40004669A (en) Therapeutic applications of cpf1-based genome editing

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20191202

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20191202

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210104

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20210402

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20210603

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210702

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20211101

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220131

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: 20220509

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220608

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20220708

R150 Certificate of patent or registration of utility model

Ref document number: 7108307

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250