JP7002936B2 - 持続的エピジェネティック遺伝子サイレンシング - Google Patents

持続的エピジェネティック遺伝子サイレンシング Download PDF

Info

Publication number
JP7002936B2
JP7002936B2 JP2017522083A JP2017522083A JP7002936B2 JP 7002936 B2 JP7002936 B2 JP 7002936B2 JP 2017522083 A JP2017522083 A JP 2017522083A JP 2017522083 A JP2017522083 A JP 2017522083A JP 7002936 B2 JP7002936 B2 JP 7002936B2
Authority
JP
Japan
Prior art keywords
domain
atr
dna
cells
binding 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
JP2017522083A
Other languages
English (en)
Japanese (ja)
Other versions
JP2017537611A5 (enExample
JP2017537611A (ja
Inventor
ナルディーニ，ルイージ
レオーネ ロンバールド，アンジェロ
アマービレ，アンジェロ
ミリャーラ，アレッサンドロ
Original Assignee
オスペダーレ サン ラファエレ エス．アール．エル
フォンダツィオーネ テレトン
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=52103358&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=JP7002936(B2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by オスペダーレ サン ラファエレ エス．アール．エル, フォンダツィオーネ テレトン filed Critical オスペダーレ サン ラファエレ エス．アール．エル
Publication of JP2017537611A publication Critical patent/JP2017537611A/ja
Publication of JP2017537611A5 publication Critical patent/JP2017537611A5/ja
Priority to JP2021213691A priority Critical patent/JP7623272B2/ja
Application granted granted Critical
Publication of JP7002936B2 publication Critical patent/JP7002936B2/ja
Priority to JP2025005873A priority patent/JP2025063201A/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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4703—Inhibitors; Suppressors
    • 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
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1007—Methyltransferases (general) (2.1.1.)
    • 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
    • C12Y—ENZYMES
    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/01—Methyltransferases (2.1.1)
    • C12Y201/01037—DNA (cytosine-5-)-methyltransferase (2.1.1.37)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/01—Methyltransferases (2.1.1)
    • C12Y201/01043—Histone-lysine N-methyltransferase (2.1.1.43)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Cell Biology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2017522083A 2014-10-24 2015-10-23 持続的エピジェネティック遺伝子サイレンシング Active JP7002936B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2021213691A JP7623272B2 (ja) 2014-10-24 2021-12-28 持続的エピジェネティック遺伝子サイレンシング
JP2025005873A JP2025063201A (ja) 2014-10-24 2025-01-16 持続的エピジェネティック遺伝子サイレンシング

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1418965.8A GB201418965D0 (enExample) 2014-10-24 2014-10-24
GB1418965.8 2014-10-24
PCT/IB2015/058202 WO2016063264A1 (en) 2014-10-24 2015-10-23 Permanent epigenetic gene silencing

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2021213691A Division JP7623272B2 (ja) 2014-10-24 2021-12-28 持続的エピジェネティック遺伝子サイレンシング

Publications (3)

Publication Number Publication Date
JP2017537611A JP2017537611A (ja) 2017-12-21
JP2017537611A5 JP2017537611A5 (enExample) 2018-12-06
JP7002936B2 true JP7002936B2 (ja) 2022-02-04

Family

ID=52103358

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2017522083A Active JP7002936B2 (ja) 2014-10-24 2015-10-23 持続的エピジェネティック遺伝子サイレンシング
JP2021213691A Active JP7623272B2 (ja) 2014-10-24 2021-12-28 持続的エピジェネティック遺伝子サイレンシング
JP2025005873A Pending JP2025063201A (ja) 2014-10-24 2025-01-16 持続的エピジェネティック遺伝子サイレンシング

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2021213691A Active JP7623272B2 (ja) 2014-10-24 2021-12-28 持続的エピジェネティック遺伝子サイレンシング
JP2025005873A Pending JP2025063201A (ja) 2014-10-24 2025-01-16 持続的エピジェネティック遺伝子サイレンシング

Country Status (13)

Country Link
US (1) US12152240B2 (enExample)
EP (2) EP3209783B1 (enExample)
JP (3) JP7002936B2 (enExample)
CN (2) CN116789846A (enExample)
AU (3) AU2015334469B2 (enExample)
CA (1) CA2965591A1 (enExample)
DK (1) DK3209783T3 (enExample)
ES (1) ES2906263T3 (enExample)
GB (1) GB201418965D0 (enExample)
HU (1) HUE057846T2 (enExample)
PL (1) PL3209783T3 (enExample)
PT (1) PT3209783T (enExample)
WO (1) WO2016063264A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022068875A (ja) * 2014-10-24 2022-05-10 オスペダーレ サン ラファエレ エス．アール．エル 持続的エピジェネティック遺伝子サイレンシング

Families Citing this family (58)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013163628A2 (en) 2012-04-27 2013-10-31 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
WO2016073955A2 (en) * 2014-11-06 2016-05-12 President And Fellows Of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
EP3256487A4 (en) 2015-02-09 2018-07-18 Duke University Compositions and methods for epigenome editing
EP3273976B1 (en) * 2015-03-27 2023-07-26 President and Fellows of Harvard College Modified t cells and methods of making and using the same
US11674144B2 (en) * 2015-04-16 2023-06-13 California Institute Of Technology Fractional regulation of transcription
EP3294342A4 (en) 2015-05-08 2018-11-07 President and Fellows of Harvard College Universal donor stem cells and related methods
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
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
CA3006432A1 (en) 2015-12-04 2017-06-08 Novartis Ag Compositions and methods for immunooncology
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
EP3463484B1 (en) * 2016-05-27 2026-01-07 The Regents of the University of California Methods and compositions for targeting rna polymerases and non-coding rna biogenesis to specific loci
WO2017208247A1 (en) * 2016-06-02 2017-12-07 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Assay for the removal of methyl-cytosine residues from dna
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
CA3034369A1 (en) 2016-08-19 2018-02-22 Whitehead Institute For Biomedical Research Methods of editing dna methylation
WO2018049079A1 (en) 2016-09-07 2018-03-15 Flagship Pioneering, Inc. Methods and compositions for modulating gene expression
EP3551218A4 (en) * 2016-12-12 2020-12-09 Whitehead Institute for Biomedical Research REGULATION OF TRANSCRIPTION THANKS TO CTCF LOOP ANCHORS
FI3565891T3 (fi) 2017-01-09 2023-07-04 Whitehead Inst Biomedical Res Menetelmiä geeniekspression muuttamiseksi häiritsemällä säätelysilmukoita muodostavia transkriptiotekijämultimeerejä
WO2018160908A1 (en) * 2017-03-03 2018-09-07 Flagship Pioneering, Inc. Methods and systems for modifying dna
SG11201907434RA (en) 2017-03-22 2019-10-30 Novartis Ag Compositions and methods for immunooncology
US11166985B2 (en) 2017-05-12 2021-11-09 Crispr Therapeutics Ag Materials and methods for engineering cells and uses thereof in immuno-oncology
AU2018367896B2 (en) 2017-05-12 2023-06-01 Crispr Therapeutics Ag Materials and methods for engineering cells and uses thereof in immuno-oncology
US11072782B2 (en) 2017-07-24 2021-07-27 Albert-Ludwigs-Universitaet Freiburg Construct for epigenetic modification and its use in the silencing of genes
CN107759673B (zh) * 2017-09-27 2021-06-04 复旦大学 可对hbv的dna进行表观甲基化修饰的蛋白分子及其应用
US11629342B2 (en) 2017-10-17 2023-04-18 President And Fellows Of Harvard College Cas9-based transcription modulation systems
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
JP7555822B2 (ja) * 2018-04-19 2024-09-25 ザ・リージエンツ・オブ・ザ・ユニバーシテイー・オブ・カリフオルニア ゲノム編集のための組成物および方法
MX2020012028A (es) 2018-05-11 2021-03-29 Crispr Therapeutics Ag Metodos y composiciones para tratar el cancer.
US20220017926A1 (en) * 2018-09-21 2022-01-20 Cafa Therapeutics Limited Method for gene editing of cell on the basis of crispr/cas system
GB201817821D0 (en) * 2018-10-31 2018-12-19 Ospedale San Raffaele Srl TCR and peptides
US12428639B2 (en) 2019-03-05 2025-09-30 Ramot At Tel-Aviv University Ltd. Plant DNA methyltransferases and uses thereof
JP7618581B2 (ja) 2019-04-23 2025-01-21 サンガモ セラピューティクス， インコーポレイテッド ９番染色体オープンリーディングフレーム７２遺伝子発現のモジュレーターおよびその使用
EP3962535A1 (en) 2019-04-30 2022-03-09 CRISPR Therapeutics AG Allogeneic cell therapy of b cell malignancies using genetically engineered t cells targeting cd19
AU2020352552A1 (en) 2019-09-23 2022-03-17 Omega Therapeutics, Inc. Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (HNF4α) gene expression
JP2023501617A (ja) * 2019-11-13 2023-01-18 クリスパー セラピューティクス アクチェンゲゼルシャフト Ｃａｒ－ｔ細胞を製造する方法
US20210139850A1 (en) * 2019-11-13 2021-05-13 Crispr Therapeutics Ag Manufacturing process for making t cells expressing chimeric antigen receptors
EP4217477A4 (en) * 2020-09-24 2024-10-16 Flagship Pioneering Innovations V, Inc. COMPOSITIONS AND METHODS FOR INHIBITING GENE EXPRESSION
AU2021409729A1 (en) * 2020-12-22 2023-07-13 Nchroma Bio, Inc. Compositions and methods for epigenetic editing
EP4284932A1 (en) * 2021-02-01 2023-12-06 Epsilen Bio S.r.l. Gene silencing
JP2024506208A (ja) * 2021-02-15 2024-02-09 オスペダーレ サン ラファエレ エス．アール．エル 癌の治療のためのエピジェネティックサイレンシング
CN115247175B (zh) * 2021-11-25 2025-10-17 南京启真基因工程有限公司 构建setdb1基因突变的表观遗传失调模型猪核移植供体细胞的基因编辑系统及其应用
WO2023165597A1 (en) * 2022-03-04 2023-09-07 Epigenic Therapeutics , Inc. Compositions and methods of genome editing
EP4544057A1 (en) * 2022-06-23 2025-04-30 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of trac expression
JP2025525389A (ja) * 2022-06-23 2025-08-05 エヌクロマ・バイオ，インコーポレーテッド Ｃｉｉｔａ発現のエピジェネティック調節のための組成物及び方法
JP2025521581A (ja) * 2022-06-23 2025-07-10 エヌクロマ・バイオ，インコーポレーテッド エピジェネティック編集用の組成物および方法
CA3260474A1 (en) * 2022-06-23 2023-12-28 Chroma Medicine, Inc. COMPOSITIONS AND METHODS OF EPIGENETIC REGULATION OF B2M EXPRESSION
AU2023289696A1 (en) 2022-06-24 2025-01-16 Tune Therapeutics, Inc. Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression
IL318767A (en) 2022-08-19 2025-04-01 Tune Therapeutics Inc Compounds, systems and methods for regulating hepatitis B virus through targeted gene repression
JP2025532177A (ja) * 2022-09-23 2025-09-29 エヌクロマ・バイオ，インコーポレーテッド Ｈｂｖ遺伝子発現のエピジェネティック調節のための組成物および方法
WO2024081879A1 (en) * 2022-10-14 2024-04-18 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of cd247 expression
KR20250165443A (ko) 2023-02-17 2025-11-25 화이트헤드 인스티튜트 포 바이오메디칼 리서치 후성유전학적 변형 제조를 위한 조성물 및 방법
WO2024220857A1 (en) * 2023-04-20 2024-10-24 Chroma Medicine, Inc. Fusion proteins for epigenetic regulation
WO2024238700A1 (en) * 2023-05-15 2024-11-21 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
IT202300011790A1 (it) 2023-06-08 2024-12-08 Fond Telethon Ets Protocolli di manipolazione genica in cellule immunitarie
WO2025031446A1 (zh) * 2023-08-09 2025-02-13 厦门大学 瓜氨酸化setdb1多肽及其在诊断类风湿性关节炎中的应用
WO2025160155A1 (en) 2024-01-22 2025-07-31 The Broad Institute, Inc. Epigenetic targeting of prion diseases
WO2025261473A1 (zh) * 2024-06-21 2025-12-26 益杰立科(上海)生物科技有限公司 工程化基因转录阻抑工具及其用途

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2010515448A (ja) 2007-01-09 2010-05-13 オンコメチローム サイエンシズ エスエー 選択された遺伝子のエピジェネティックな変化及び癌
WO2013093489A2 (en) 2011-12-20 2013-06-27 The European Molecular Biology Laboratory Detection and treatment of breast cancer
WO2013138650A1 (en) 2012-03-14 2013-09-19 Salk Institute For Biological Studies Adenoviral tumor diagnostics
JP2013543381A (ja) 2010-10-01 2013-12-05 モデルナ セラピューティクス インコーポレイテッド 工学操作された核酸およびその使用方法

Family Cites Families (52)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5723315A (en) 1996-08-23 1998-03-03 Genetics Institute, Inc. Secreted proteins and polynucleotides encoding them
KR20000029858A (ko) 1996-08-07 2000-05-25 마르크 젠너 엠엠피와티엔에프억제활성을갖는히드록삼산및카르복실산유도체
US6126939A (en) 1996-09-03 2000-10-03 Yeda Research And Development Co. Ltd. Anti-inflammatory dipeptide and pharmaceutical composition thereof
GB9803351D0 (en) 1998-02-17 1998-04-15 Oxford Biomedica Ltd Anti-viral vectors
US20020188103A1 (en) 1998-10-09 2002-12-12 Timothy H. Bestor Chimeric dna-binding/dna methyltransferase nucleic acid and polypeptide and uses thereof
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
GB0009760D0 (en) 2000-04-19 2000-06-07 Oxford Biomedica Ltd Method
EP1276859B1 (en) * 2000-04-28 2007-02-07 Sangamo Biosciences Inc. Targeted modification of chromatin structure
US6919204B2 (en) 2000-09-29 2005-07-19 Sangamo Biosciences, Inc. Modulation of gene expression using localization domains
WO2003072788A1 (en) 2002-02-21 2003-09-04 The Wistar Institute Of Anatomy And Biology Methods and compositions for reversibly controlling expression of target genes in cells
WO2005028630A2 (en) 2003-09-19 2005-03-31 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
AU2005233550B2 (en) 2004-04-08 2010-11-18 Sangamo Therapeutics, Inc. Treatment of neuropathic pain with zinc finger proteins
GB0526449D0 (en) 2005-12-23 2006-02-08 Medical Res Council Polypeptide targeting
KR100756055B1 (ko) 2006-01-27 2007-09-07 연세대학교 산학협력단 신생혈관 생성을 조절하는 재조합 아데노바이러스
GB0607063D0 (en) 2006-04-07 2006-05-17 Cellcentric Ltd Compositions and methods for epigenetic modification of nucleic acid sequences in vivo
US20120207744A1 (en) 2009-03-19 2012-08-16 Mendlein John D Reprogramming compositions and methods of using the same
CA2769262C (en) 2009-07-28 2019-04-30 Sangamo Biosciences, Inc. Methods and compositions for treating trinucleotide repeat disorders
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
EP2820127B1 (en) 2012-02-28 2018-09-26 Sigma Aldrich Co. LLC Targeted histone acetylation
MX374399B (es) 2012-02-29 2025-03-06 Sangamo Biosciences Inc Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
EP4570908A3 (en) 2012-05-25 2025-10-22 The Regents of the University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
CN116064532A (zh) 2012-10-23 2023-05-05 基因工具股份有限公司 用于切割靶dna的组合物及其用途
SG11201503824SA (en) 2012-12-06 2015-06-29 Sigma Aldrich Co Llc Crispr-based genome modification and regulation
WO2014093595A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
DK2931898T3 (en) 2012-12-12 2016-06-20 Massachusetts Inst Technology CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS
PT2784162E (pt) 2012-12-12 2015-08-27 Broad Inst Inc Engenharia de sistemas, métodos e composições guia otimizadas para a manipulação de sequências
DK3064585T3 (da) 2012-12-12 2020-04-27 Broad Inst Inc Konstruering og optimering af forbedrede systemer, fremgangsmåder og enzymsammensætninger til sekvensmanipulation
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
IL308158A (en) 2012-12-17 2023-12-01 Harvard College Rna-guided human genome engineering
KR102405549B1 (ko) 2013-03-15 2022-06-08 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
JP7085716B2 (ja) 2013-06-05 2022-06-17 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
WO2015006294A2 (en) 2013-07-10 2015-01-15 President And Fellows Of Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150191744A1 (en) 2013-12-17 2015-07-09 University Of Massachusetts Cas9 effector-mediated regulation of transcription, differentiation and gene editing/labeling
CA2942915A1 (en) 2014-03-20 2015-09-24 Universite Laval Crispr-based methods and products for increasing frataxin levels and uses thereof
WO2016011080A2 (en) 2014-07-14 2016-01-21 The Regents Of The University Of California Crispr/cas transcriptional modulation
CA2954920A1 (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
US20170233762A1 (en) 2014-09-29 2017-08-17 The Regents Of The University Of California Scaffold rnas
CN107530399B (zh) 2014-10-10 2022-03-18 马萨诸塞眼科耳科诊所 治疗分子在体外和体内的有效递送
GB201418965D0 (enExample) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
LT3250691T (lt) 2015-01-28 2023-09-11 Caribou Biosciences, Inc. Crispr hibridiniai dnr/rnr polinukleotidai ir naudojimo būdai
EP3256487A4 (en) 2015-02-09 2018-07-18 Duke University Compositions and methods for epigenome editing
EP3325018B1 (en) 2015-07-22 2025-01-15 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
HK1257460A1 (zh) 2015-09-09 2019-10-18 Seattle Children's Hospital (dba Seattle Children's Research Institute) 用於免疫疗法的巨噬细胞的遗传工程
EP3147363B1 (en) 2015-09-26 2019-10-16 B.R.A.I.N. Ag Activation of taste receptor genes in mammalian cells using crispr-cas-9
US10612044B2 (en) 2015-11-25 2020-04-07 National University Corporation Gunma University DNA methylation editing kit and DNA methylation editing method

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2010515448A (ja) 2007-01-09 2010-05-13 オンコメチローム サイエンシズ エスエー 選択された遺伝子のエピジェネティックな変化及び癌
JP2013543381A (ja) 2010-10-01 2013-12-05 モデルナ セラピューティクス インコーポレイテッド 工学操作された核酸およびその使用方法
WO2013093489A2 (en) 2011-12-20 2013-06-27 The European Molecular Biology Laboratory Detection and treatment of breast cancer
WO2013138650A1 (en) 2012-03-14 2013-09-19 Salk Institute For Biological Studies Adenoviral tumor diagnostics

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
Development，2014年01月，Vol. 141，p. 219-223
J. Biol. Chem.，2006年，Vol. 281，p. 19489-19500
KOCAK, Daniel Dewran，Synthetic Transcription Factors and their Effects on Endogenous DNA Methylation in Human Cells，Thesis submitted in partial fulfillment of the requirements for the degree of Master of Science in the Department of Biomedical Engineering in the Graduate School of Duke University，2013年，p. 1-29
Mol. Cell. Biol.，1995年，Vol. 15, No. 4，p. 1907-1914
Stem Cell Reports，2013年，Vol. 1，p. 218-225

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022068875A (ja) * 2014-10-24 2022-05-10 オスペダーレ サン ラファエレ エス．アール．エル 持続的エピジェネティック遺伝子サイレンシング
JP7623272B2 (ja) 2014-10-24 2025-01-28 オスペダーレ サン ラファエレ エス．アール．エル 持続的エピジェネティック遺伝子サイレンシング
JP2025063201A (ja) * 2014-10-24 2025-04-15 オスペダーレ サン ラファエレ エス．アール．エル 持続的エピジェネティック遺伝子サイレンシング

Also Published As

Publication number Publication date
CN107109433B (zh) 2023-01-10
EP3209783B1 (en) 2021-11-24
GB201418965D0 (enExample) 2014-12-10
CA2965591A1 (en) 2016-04-28
JP7623272B2 (ja) 2025-01-28
CN107109433A (zh) 2017-08-29
WO2016063264A1 (en) 2016-04-28
AU2025201007A1 (en) 2025-03-06
JP2025063201A (ja) 2025-04-15
CN116789846A (zh) 2023-09-22
AU2022202407A1 (en) 2022-05-05
JP2022068875A (ja) 2022-05-10
ES2906263T3 (es) 2022-04-13
PL3209783T3 (pl) 2022-04-19
AU2022202407B2 (en) 2024-11-14
AU2015334469B2 (en) 2022-01-20
DK3209783T3 (da) 2022-02-14
EP3209783A1 (en) 2017-08-30
PT3209783T (pt) 2022-02-10
HUE057846T2 (hu) 2022-06-28
AU2015334469A1 (en) 2017-05-25
US20190032049A1 (en) 2019-01-31
EP3995584A1 (en) 2022-05-11
JP2017537611A (ja) 2017-12-21
US12152240B2 (en) 2024-11-26

Similar Documents

Publication Publication Date Title
JP7623272B2 (ja) 持続的エピジェネティック遺伝子サイレンシング
JP2024506751A (ja) 遺伝子サイレンシング
US20230272429A1 (en) Modification of blood type antigens
US20220033856A1 (en) Methods for increasing fetal hemoglobin content in eukaryotic cells and uses thereof for the treatment of hemoglobinopathies
AU2018253958B2 (en) Gene therapy
JP7642548B2 (ja) 免疫療法の改善のための遺伝子調節組成物及び遺伝子調節方法
EP3810638A1 (en) Gene therapy
US20230340471A1 (en) Permanent Epigenetic Gene Silencing
JP2023527622A (ja) 新規形質導入エンハンサーおよびその使用
US20250288617A1 (en) Gene Therapy
US20250236870A1 (en) Permanent epigenetic gene silencing
JP2025506976A (ja) 低免疫原性幹細胞、幹細胞から分化または由来した低免疫原性細胞およびこの製造方法
US20240318154A1 (en) Gene therapy for the treatment of severe combined immunodeficiency (scid) related to rag1

Legal Events

Date Code Title Description
RD01 Notification of change of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7426

Effective date: 20180129

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20180129

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20181023

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20181023

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20191126

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200226

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20200818

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20201113

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210511

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20210805

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211111

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: 20211130

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20211228

R150 Certificate of patent or registration of utility model

Ref document number: 7002936

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250