ES2906263T3 - Silenciamiento génico epigénico permanente - Google Patents

Silenciamiento génico epigénico permanente Download PDF

Info

Publication number
ES2906263T3
ES2906263T3 ES15794644T ES15794644T ES2906263T3 ES 2906263 T3 ES2906263 T3 ES 2906263T3 ES 15794644 T ES15794644 T ES 15794644T ES 15794644 T ES15794644 T ES 15794644T ES 2906263 T3 ES2906263 T3 ES 2906263T3
Authority
ES
Spain
Prior art keywords
domain
dna
binding
atr
atrs
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
ES15794644T
Other languages
English (en)
Spanish (es)
Inventor
Luigi Naldini
Angelo Leone Lombardo
Angelo Amabile
Alessandro Migliara
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.)
Telethon Foundation
IRCCS Ospedale San Raffaele
Original Assignee
Telethon Foundation
IRCCS Ospedale San Raffaele
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=ES2906263(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Telethon Foundation, IRCCS Ospedale San Raffaele filed Critical Telethon Foundation
Application granted granted Critical
Publication of ES2906263T3 publication Critical patent/ES2906263T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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)
    • 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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (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)
ES15794644T 2014-10-24 2015-10-23 Silenciamiento génico epigénico permanente Active ES2906263T3 (es)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
ES2906263T3 true ES2906263T3 (es) 2022-04-13

Family

ID=52103358

Family Applications (1)

Application Number Title Priority Date Filing Date
ES15794644T Active ES2906263T3 (es) 2014-10-24 2015-10-23 Silenciamiento génico epigénico permanente

Country Status (13)

Country Link
US (1) US12152240B2 (enExample)
EP (2) EP3209783B1 (enExample)
JP (3) JP7002936B2 (enExample)
CN (2) CN116789846A (enExample)
AU (4) 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)

Families Citing this family (63)

* 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
GB201418965D0 (enExample) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
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
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
BR112017020750A2 (en) * 2015-03-27 2018-06-26 President And Fellows Of Harvard College modified t-cells and methods of producing and using them
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
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US12214054B2 (en) 2015-11-30 2025-02-04 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
WO2017093969A1 (en) 2015-12-04 2017-06-08 Novartis Ag Compositions and methods for immunooncology
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
US11286493B2 (en) 2016-05-27 2022-03-29 The Regents Of The University Of California Methods and compositions for targeting RNA polymerases and non-coding RNA biogenesis to specific loci
US20190100732A1 (en) * 2016-06-02 2019-04-04 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Assay for the removal of methyl-cytosine residues from dna
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
MX2019002004A (es) 2016-08-19 2019-10-09 Whitehead Inst Biomedical Res Metodos para editar la metilacion del adn.
WO2018049075A1 (en) 2016-09-07 2018-03-15 Flagship Pioneering, Inc. Methods and compositions for modulating gene expression
WO2018111944A1 (en) 2016-12-12 2018-06-21 Whitehead Institute For Biomedical Research Regulation of transcription through ctcf loop anchors
US11873496B2 (en) 2017-01-09 2024-01-16 Whitehead Institute For Biomedical Research Methods of altering gene expression by perturbing transcription factor multimers that structure regulatory loops
WO2018160908A1 (en) * 2017-03-03 2018-09-07 Flagship Pioneering, Inc. Methods and systems for modifying dna
KR20190130608A (ko) 2017-03-22 2019-11-22 노파르티스 아게 면역종양학을 위한 조성물 및 방법
BR112019023608A2 (pt) 2017-05-12 2020-05-26 Crispr Therapeutics Ag Materiais e métodos para células manipuladas e seus usos em imuno-oncologia
US11166985B2 (en) 2017-05-12 2021-11-09 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
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
AU2019255789B2 (en) * 2018-04-19 2025-11-20 The Regents Of The University Of California Compositions and methods for gene editing
US20200063100A1 (en) 2018-05-11 2020-02-27 Crispr Therapeutics Ag Methods and compositions for treating cancer
AU2019341365A1 (en) * 2018-09-21 2021-05-27 Crage Medical Co., 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
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 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
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
CA3138633A1 (en) 2019-04-30 2020-11-05 Crispr Therapeutics Ag Allogeneic cell therapy of b cell malignancies using genetically engineered t cells targeting cd19
CN114729376A (zh) 2019-09-23 2022-07-08 欧米茄治疗公司 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法
AU2020382219A1 (en) * 2019-11-13 2022-05-12 Crispr Therapeutics Ag Manufacturing process for making T cells expressing chimeric antigen receptors
WO2021095012A1 (en) * 2019-11-13 2021-05-20 Crispr Therapeutics Ag Methods of manufacturing car-t cells
WO2022067033A1 (en) * 2020-09-24 2022-03-31 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
MX2023009036A (es) * 2021-02-01 2023-10-30 Epsilen Bio S R L Silenciamiento de genes.
CA3207426A1 (en) * 2021-02-15 2022-08-18 Vania Broccoli Epigenetic silencing for treatment of cancer
IL311610A (en) 2021-09-21 2024-05-01 Scribe Therapeutics Inc Engineered CASX suppressor systems
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
CA3254504A1 (en) * 2022-03-04 2023-09-07 Epigenic Therapeutics Pte. Ltd. GENOME COMPOSITION AND EDITING METHODS
CN119998446A (zh) 2022-06-07 2025-05-13 斯克里贝治疗公司 用于靶向pcsk9的组合物和方法
WO2023250148A1 (en) * 2022-06-23 2023-12-28 Chroma Medicine, Inc. Compositions and methods for epigenetic editing
EP4544058A1 (en) * 2022-06-23 2025-04-30 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of ciita expression
WO2023250490A1 (en) * 2022-06-23 2023-12-28 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of trac expression
CA3260474A1 (en) * 2022-06-23 2023-12-28 Chroma Medicine, Inc. COMPOSITIONS AND METHODS OF EPIGENETIC REGULATION OF B2M EXPRESSION
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression
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
KR20250110209A (ko) * 2022-09-23 2025-07-18 엔크로마 바이오, 인크. Hbv 유전자 발현의 에피제네틱 조절을 위한 조성물 및 방법
WO2024081879A1 (en) * 2022-10-14 2024-04-18 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of cd247 expression
JP2026500040A (ja) * 2022-12-23 2026-01-05 エピジェニック セラピューティクス プライベート リミテッド 複合体及びその使用
JP2026506083A (ja) 2023-02-17 2026-02-20 ホワイトヘッド・インスティテュート・フォー・バイオメディカル・リサーチ エピジェネティック修飾を行うための組成物および方法
WO2024220857A1 (en) * 2023-04-20 2024-10-24 Chroma Medicine, Inc. Fusion proteins for epigenetic regulation
JP2026523341A (ja) 2023-05-15 2026-07-14 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
IT202300011790A1 (it) 2023-06-08 2024-12-08 Fond Telethon Ets Protocolli di manipolazione genica in cellule immunitarie
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
EP4696705A1 (en) * 2024-08-13 2026-02-18 Fondazione Telethon ETS Engineered transcription repressors and uses thereof
WO2026064753A1 (en) 2024-09-23 2026-03-26 Tune Therapeutics, Inc. Dna methyltransferase-like protein (dnmt3l) or dna methyltransferase 3a (dnmt3a) repressor systems for epigenetic editing

Family Cites Families (59)

* 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
CZ297278B6 (cs) 1996-08-07 2006-10-11 Darwin Discovery Limited Deriváty hydroxamové a karboxylové kyseliny a jejich pouzití
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
EP1458872A1 (en) * 2001-10-05 2004-09-22 Vlaams Interuniversitair Instituut voor Biotechnologie vzw. An efficient system for rna silencing
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
CA2506798C (en) * 2002-11-22 2013-01-22 Institut Clayton De La Recherche Compositions and systems for the regulation of genes
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
US7253273B2 (en) 2004-04-08 2007-08-07 Sangamo Biosciences, 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
GB0700374D0 (en) 2007-01-09 2007-02-14 Oncomethylome Sciences S A NDRG family methylation markers
US20110172107A1 (en) * 2008-04-30 2011-07-14 Fox Chase Cancer Center Assay for identifying agents that modulate epigenetic silencing, and agents identified thereby
WO2010108126A2 (en) * 2009-03-19 2010-09-23 Fate Therapeutics, Inc. 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
EP2857499A1 (en) 2010-10-01 2015-04-08 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
WO2013066438A2 (en) 2011-07-22 2013-05-10 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
GB201121924D0 (en) 2011-12-20 2012-02-01 Fahy Gurteen Labs Ltd Detection of breast cancer
CN105658792B (zh) 2012-02-28 2020-11-10 西格马-奥尔德里奇有限责任公司 靶向组蛋白乙酰化
CA2865011C (en) 2012-02-29 2021-06-15 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
CA2865642C (en) 2012-03-14 2021-10-26 Salk Institute For Biological Studies Adenoviral tumor diagnostics
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
EP4397759A3 (en) 2012-10-23 2024-10-09 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
DK3363902T3 (da) 2012-12-06 2020-01-02 Sigma Aldrich Co Llc Crispr-baseret genommodifikation og -regulering
PT2931898E (pt) 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais
CN105209621B (zh) 2012-12-12 2021-05-25 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
MX380562B (es) 2012-12-12 2025-03-12 Broad Inst Inc Modificaciones de sistemas, métodos y composiciones guía optimizadas para la manipulación de secuencias.
KR102955556B1 (ko) 2012-12-12 2026-04-22 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물
IL308158A (en) 2012-12-17 2023-12-01 Harvard College Rna-guided human genome engineering
CN105408483A (zh) 2013-03-15 2016-03-16 通用医疗公司 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向
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
EP3603679B1 (en) 2013-06-04 2022-08-10 President and Fellows of Harvard College Rna-guided transcriptional regulation
WO2014197748A2 (en) 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
CN105517579B (zh) 2013-07-10 2019-11-15 哈佛大学校长及研究员协会 用于RNA向导的基因调节和编辑的正交Cas9蛋白
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
EP3126498A4 (en) 2014-03-20 2017-08-23 Université Laval Crispr-based methods and products for increasing frataxin levels and uses thereof
EP3169776B1 (en) 2014-07-14 2025-09-03 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
AU2016211517B2 (en) 2015-01-28 2021-05-13 Caribou Biosciences, Inc. CRISPR hybrid DNA/RNA polynucleotides and methods of use
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
ES3009026T3 (en) 2015-07-22 2025-03-25 Univ Duke High-throughput screening of regulatory element function with epigenome editing technologies
SG10201913151TA (en) 2015-09-09 2020-03-30 Seattle Childrens Hospital Dba Seattle Childrens Res Inst Genetic engineering of macrophages for immunotherapy
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
EP3382018B1 (en) 2015-11-25 2022-03-30 National University Corporation Gunma University Dna methylation editing kit and dna methylation editing method

Also Published As

Publication number Publication date
JP2022068875A (ja) 2022-05-10
US20190032049A1 (en) 2019-01-31
EP3209783B1 (en) 2021-11-24
CA2965591A1 (en) 2016-04-28
GB201418965D0 (enExample) 2014-12-10
DK3209783T3 (da) 2022-02-14
CN107109433A (zh) 2017-08-29
EP3209783A1 (en) 2017-08-30
HUE057846T2 (hu) 2022-06-28
CN107109433B (zh) 2023-01-10
AU2015334469A1 (en) 2017-05-25
US12152240B2 (en) 2024-11-26
JP2025063201A (ja) 2025-04-15
JP7623272B2 (ja) 2025-01-28
AU2025201007A1 (en) 2025-03-06
AU2022202407B2 (en) 2024-11-14
PL3209783T3 (pl) 2022-04-19
JP2017537611A (ja) 2017-12-21
PT3209783T (pt) 2022-02-10
AU2022202407A1 (en) 2022-05-05
WO2016063264A1 (en) 2016-04-28
CN116789846A (zh) 2023-09-22
AU2026206081A1 (en) 2026-08-06
JP7002936B2 (ja) 2022-02-04
EP3995584A1 (en) 2022-05-11
AU2015334469B2 (en) 2022-01-20

Similar Documents

Publication Publication Date Title
AU2022202407B2 (en) Permanent epigenetic gene silencing
US20240139319A1 (en) Gene silencing
KR102553518B1 (ko) Hiv 감염의 rna-가이드된 치료를 위한 방법 및 조성물
US20230340471A1 (en) Permanent Epigenetic Gene Silencing
CN107532182A (zh) 治疗血红蛋白病的材料和方法
AU2017219605A1 (en) Excision of retroviral nucleic acid sequences
EP3938510A1 (en) Improved process for integration of dna constructs using rna-guided endonucleases
CA3104948A1 (en) Gene therapy
US20250236870A1 (en) Permanent epigenetic gene silencing
US9816077B2 (en) Use of integrase for targeted gene expression
WO2026090544A1 (en) Gene silencing
US20240318154A1 (en) Gene therapy for the treatment of severe combined immunodeficiency (scid) related to rag1
Gutiérrez-Guerrero Gene editing as an alternative to retroviral vectors for Wiskott-Aldrich syndrome gene therapy