AU2015334469B2 - Permanent epigenetic gene silencing - Google Patents

Permanent epigenetic gene silencing Download PDF

Info

Publication number
AU2015334469B2
AU2015334469B2 AU2015334469A AU2015334469A AU2015334469B2 AU 2015334469 B2 AU2015334469 B2 AU 2015334469B2 AU 2015334469 A AU2015334469 A AU 2015334469A AU 2015334469 A AU2015334469 A AU 2015334469A AU 2015334469 B2 AU2015334469 B2 AU 2015334469B2
Authority
AU
Australia
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
AU2015334469A
Other languages
English (en)
Other versions
AU2015334469A1 (en
Inventor
Angelo AMABILE
Angelo Leone Lombardo
Alessandro MIGLIARA
Luigi Naldini
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.)
Fondazione Telethon
Ospedale San Raffaele SRL
Original Assignee
Fondazione Telethon
Ospedale San Raffaele SRL
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=AU2015334469(B2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Fondazione Telethon, Ospedale San Raffaele SRL filed Critical Fondazione Telethon
Publication of AU2015334469A1 publication Critical patent/AU2015334469A1/en
Application granted granted Critical
Publication of AU2015334469B2 publication Critical patent/AU2015334469B2/en
Priority to AU2022202407A priority Critical patent/AU2022202407B2/en
Priority to AU2025201007A priority patent/AU2025201007A1/en
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)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (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)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2015334469A 2014-10-24 2015-10-23 Permanent epigenetic gene silencing Active AU2015334469B2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2022202407A AU2022202407B2 (en) 2014-10-24 2022-04-12 Permanent epigenetic gene silencing
AU2025201007A AU2025201007A1 (en) 2014-10-24 2025-02-13 Permanent epigenetic gene silencing

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1418965.8A GB201418965D0 (OSRAM) 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
AU2022202407A Division AU2022202407B2 (en) 2014-10-24 2022-04-12 Permanent epigenetic gene silencing

Publications (2)

Publication Number Publication Date
AU2015334469A1 AU2015334469A1 (en) 2017-05-25
AU2015334469B2 true AU2015334469B2 (en) 2022-01-20

Family

ID=52103358

Family Applications (3)

Application Number Title Priority Date Filing Date
AU2015334469A Active AU2015334469B2 (en) 2014-10-24 2015-10-23 Permanent epigenetic gene silencing
AU2022202407A Active AU2022202407B2 (en) 2014-10-24 2022-04-12 Permanent epigenetic gene silencing
AU2025201007A Pending AU2025201007A1 (en) 2014-10-24 2025-02-13 Permanent epigenetic gene silencing

Family Applications After (2)

Application Number Title Priority Date Filing Date
AU2022202407A Active AU2022202407B2 (en) 2014-10-24 2022-04-12 Permanent epigenetic gene silencing
AU2025201007A Pending AU2025201007A1 (en) 2014-10-24 2025-02-13 Permanent epigenetic gene silencing

Country Status (13)

Country Link
US (1) US12152240B2 (OSRAM)
EP (2) EP3995584A1 (OSRAM)
JP (3) JP7002936B2 (OSRAM)
CN (2) CN116789846A (OSRAM)
AU (3) AU2015334469B2 (OSRAM)
CA (1) CA2965591A1 (OSRAM)
DK (1) DK3209783T3 (OSRAM)
ES (1) ES2906263T3 (OSRAM)
GB (1) GB201418965D0 (OSRAM)
HU (1) HUE057846T2 (OSRAM)
PL (1) PL3209783T3 (OSRAM)
PT (1) PT3209783T (OSRAM)
WO (1) WO2016063264A1 (OSRAM)

Families Citing this family (57)

* 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 (OSRAM) * 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
EP3215623A4 (en) * 2014-11-06 2018-09-26 President and Fellows of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
BR112017020750A2 (pt) * 2015-03-27 2018-06-26 Harvard College células t modificadas e métodos de produção e utilização das mesmas
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
AU2016362282B2 (en) 2015-11-30 2023-03-16 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
AU2016362129A1 (en) 2015-12-04 2018-06-14 Intellia Therapeutics, Inc. 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
WO2017205837A1 (en) * 2016-05-27 2017-11-30 The Regents Of The Univeristy 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
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
EP3500675A4 (en) 2016-08-19 2020-01-29 Whitehead Institute for Biomedical Research Methods of editing dna methylation
CA3035910A1 (en) 2016-09-07 2018-03-15 Flagship Pioneering, Inc. Methods and compositions for modulating gene expression
US20200149039A1 (en) * 2016-12-12 2020-05-14 Whitehead Institute For Biomedical Research Regulation of transcription through ctcf loop anchors
ES2949801T3 (es) 2017-01-09 2023-10-03 Whitehead Inst Biomedical Res Métodos para alterar la expresión génica mediante la perturbación de multímeros de factores de transcripción que estructuran bucles reguladores
US20210322577A1 (en) * 2017-03-03 2021-10-21 Flagship Pioneering Innovations V, 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
JP7356354B2 (ja) 2017-05-12 2023-10-04 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 細胞の操作のための材料及び方法並びに免疫腫瘍学におけるその使用
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
WO2019079462A1 (en) 2017-10-17 2019-04-25 President And Fellows Of Harvard College TRANSCRIPTION MODULATION SYSTEMS BASED ON CAS9
CN117534769A (zh) * 2018-04-19 2024-02-09 加利福尼亚大学董事会 用于基因编辑的组合物和方法
EP3790629A1 (en) 2018-05-11 2021-03-17 CRISPR Therapeutics AG Methods and compositions for treating cancer
CN112805371B (zh) * 2018-09-21 2025-10-31 克莱格医学有限公司 基于CRISPR/Cas系统对细胞进行基因编辑的方法
GB201817821D0 (en) * 2018-10-31 2018-12-19 Ospedale San Raffaele Srl TCR and peptides
WO2020178831A1 (en) * 2019-03-05 2020-09-10 Ramot At Tel-Aviv University Ltd. Plant dna methyltransferases and uses thereof
MX2021012836A (es) 2019-04-23 2021-12-10 Sangamo Therapeutics Inc Moduladores de la expresion del gen del marco de lectura abierto 72 del cromosoma 9 y usos de los mismos.
CN113939319A (zh) 2019-04-30 2022-01-14 克里斯珀医疗股份公司 靶向cd19的基因工程化t细胞针对b细胞恶性肿瘤的同种异体细胞疗法
JP2022548399A (ja) 2019-09-23 2022-11-18 オメガ セラピューティクス， インコーポレイテッド 肝細胞核因子４－アルファ（ＨＮＦ４α）遺伝子発現をモジュレートするための組成物および方法
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
AU2020382219A1 (en) * 2019-11-13 2022-05-12 Crispr Therapeutics Ag Manufacturing process for making T cells expressing chimeric antigen receptors
JP2023543218A (ja) * 2020-09-24 2023-10-13 フラッグシップ パイオニアリング イノベーションズ ブイ， インコーポレイテッド 遺伝子発現を阻害するための組成物及び方法
AU2021409729A1 (en) * 2020-12-22 2023-07-13 Nchroma Bio, Inc. Compositions and methods for epigenetic editing
AU2022213505A1 (en) * 2021-02-01 2023-09-07 Epsilen Bio S.R.L. Gene silencing
US20240101623A1 (en) * 2021-02-15 2024-03-28 Ospedale San Raffaele S.R.L. Epigenetic silencing for treatment of cancer
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
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
IL317913A (en) * 2022-06-23 2025-02-01 Chroma Medicine Inc Compositions and methods for epigenetic regulation of B2M expression
WO2023250490A1 (en) * 2022-06-23 2023-12-28 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of trac expression
EP4544058A1 (en) * 2022-06-23 2025-04-30 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of ciita expression
CN119731321A (zh) 2022-06-24 2025-03-28 图恩疗法股份有限公司 通过靶向基因阻遏减少低密度脂蛋白的组合物、系统和方法
JP2025527567A (ja) 2022-08-19 2025-08-22 チューン セラピューティクス インコーポレイテッド ターゲティングされた遺伝子抑制によるｂ型肝炎ウイルスの調節のための組成物、システム、および方法
CN120283052A (zh) * 2022-09-23 2025-07-08 恩科洛玛生物公司 用于表观遗传调控hbv基因表达的组合物和方法
WO2024081879A1 (en) * 2022-10-14 2024-04-18 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of cd247 expression
AU2024220624A1 (en) 2023-02-17 2025-09-11 Whitehead Institute For Biomedical Research Compositions and methods for making epigenetic modifications
WO2024220857A1 (en) * 2023-04-20 2024-10-24 Chroma Medicine, Inc. Fusion proteins for epigenetic regulation
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024238700A1 (en) * 2023-05-15 2024-11-21 Chroma Medicine, 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
CN119464241A (zh) * 2023-08-09 2025-02-18 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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US20120207744A1 (en) * 2009-03-19 2012-08-16 Mendlein John D Reprogramming compositions and methods of using the same

Family Cites Families (54)

* 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
EP0968182B1 (en) 1996-08-07 2004-05-06 Darwin Discovery Limited Hydroxamic and carboxylic acid derivatives having mmp and tnf inhibitory activity
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
ATE353361T1 (de) * 2000-04-28 2007-02-15 Sangamo Biosciences Inc Gezielten modifikation der chromatinstruktur
US6919204B2 (en) 2000-09-29 2005-07-19 Sangamo Biosciences, Inc. Modulation of gene expression using localization domains
AU2004274957B2 (en) 2003-09-19 2009-02-05 Sangamo Therapeutics, 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
GB0700374D0 (en) 2007-01-09 2007-02-14 Oncomethylome Sciences S A NDRG family methylation markers
WO2011016840A2 (en) 2009-07-28 2011-02-10 Sangamo Biosciences, Inc. Methods and compositions for treating trinucleotide repeat disorders
CA2821992A1 (en) 2010-10-01 2012-04-05 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
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
GB201121924D0 (en) 2011-12-20 2012-02-01 Fahy Gurteen Labs Ltd Detection of breast cancer
US10570378B2 (en) 2012-02-28 2020-02-25 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.
IN2014DN06898A (OSRAM) 2012-03-14 2015-05-15 Salk Inst For Biological Studi
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
SI3401400T1 (sl) 2012-05-25 2019-10-30 Univ California Postopki in sestavki za RNA usmerjeno modifikacijo tarčne DNA in za RNA usmerjeno modulacijo prepisovanja
SG11201503059XA (en) 2012-10-23 2015-06-29 Toolgen Inc 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
EP3138911B1 (en) 2012-12-06 2018-12-05 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
CN113355357B (zh) 2012-12-12 2024-12-03 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
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
CN105658796B (zh) 2012-12-12 2021-10-26 布罗德研究所有限公司 用于序列操纵的crispr-cas组分系统、方法以及组合物
ES2536353T3 (es) 2012-12-12 2015-05-22 The Broad Institute, Inc. Ingeniería de sistemas, métodos y composiciones de guía optimizadas para manipulación de secuencias
DK3553174T3 (da) 2012-12-17 2025-08-04 Harvard College Rna-guided modificering af humant genom
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
US9885033B2 (en) 2013-03-15 2018-02-06 The General Hospital Corporation Increasing specificity for RNA-guided genome editing
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
KR20230136697A (ko) 2013-06-05 2023-09-26 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
SG11201600060VA (en) 2013-07-10 2016-02-26 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
US10323073B2 (en) 2014-03-20 2019-06-18 UNIVERSITé LAVAL CRISPR-based methods and products for increasing frataxin levels and uses thereof
US20170219596A1 (en) 2014-07-14 2017-08-03 The Regents Of The University Of California A protein tagging system for in vivo single molecule imaging and control of gene transcription
EP3169776B1 (en) 2014-07-14 2025-09-03 The Regents of The University of California Crispr/cas transcriptional modulation
US20170233762A1 (en) 2014-09-29 2017-08-17 The Regents Of The University Of California Scaffold rnas
CA2964234A1 (en) 2014-10-10 2016-04-14 Massachusetts Eye And Ear Infirmary Efficient delivery of therapeutic molecules in vitro and in vivo
GB201418965D0 (OSRAM) * 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
SG10201804715WA (en) 2015-01-28 2018-07-30 Pioneer Hi Bred Int Crispr hybrid dna/rna polynucleotides and methods of use
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
WO2017015637A1 (en) 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
CA2997912A1 (en) 2015-09-09 2017-03-16 Seattle Children's Hospital (dba Seattle Children's Research Institute) 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
WO2017090724A1 (ja) 2015-11-25 2017-06-01 国立大学法人 群馬大学 Ｄｎａメチル化編集用キットおよびｄｎａメチル化編集方法

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US20120207744A1 (en) * 2009-03-19 2012-08-16 Mendlein John D Reprogramming compositions and methods of using the same

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Ll HONGWEI ET AL, "The histone methyltransferase SETDB1 and the DNA methyltransferase DNMT3A interact directly and localize to promoters silenced in cancer cells", JOURNAL OF BIOLOGICAL CHEMISTRY, 2006, vol. 281, no. 28, pages 19489 - 19500 *

Also Published As

Publication number Publication date
AU2025201007A1 (en) 2025-03-06
US20190032049A1 (en) 2019-01-31
ES2906263T3 (es) 2022-04-13
JP7623272B2 (ja) 2025-01-28
GB201418965D0 (OSRAM) 2014-12-10
JP7002936B2 (ja) 2022-02-04
JP2017537611A (ja) 2017-12-21
JP2022068875A (ja) 2022-05-10
EP3209783B1 (en) 2021-11-24
AU2022202407B2 (en) 2024-11-14
CN107109433A (zh) 2017-08-29
JP2025063201A (ja) 2025-04-15
AU2015334469A1 (en) 2017-05-25
CN116789846A (zh) 2023-09-22
CN107109433B (zh) 2023-01-10
PL3209783T3 (pl) 2022-04-19
EP3209783A1 (en) 2017-08-30
AU2022202407A1 (en) 2022-05-05
CA2965591A1 (en) 2016-04-28
DK3209783T3 (da) 2022-02-14
US12152240B2 (en) 2024-11-26
PT3209783T (pt) 2022-02-10
HUE057846T2 (hu) 2022-06-28
WO2016063264A1 (en) 2016-04-28
EP3995584A1 (en) 2022-05-11

Similar Documents

Publication Publication Date Title
AU2022202407B2 (en) Permanent epigenetic gene silencing
US20240139319A1 (en) Gene silencing
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
CA3104948A1 (en) Gene therapy
US20230340471A1 (en) Permanent Epigenetic Gene Silencing
Weber et al. Editing a gglobin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype
WO2023147572A2 (en) Engineered multipartite transcriptional effectors sourced from human protein domains
US20250236870A1 (en) Permanent epigenetic gene silencing
Gutiérrez-Guerrero Gene editing as an alternative to retroviral vectors for Wiskott-Aldrich syndrome gene therapy

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)