MX2020005561A - Proteinas modificadas de union a adn. - Google Patents

Proteinas modificadas de union a adn.

Info

Publication number
MX2020005561A
MX2020005561A MX2020005561A MX2020005561A MX2020005561A MX 2020005561 A MX2020005561 A MX 2020005561A MX 2020005561 A MX2020005561 A MX 2020005561A MX 2020005561 A MX2020005561 A MX 2020005561A MX 2020005561 A MX2020005561 A MX 2020005561A
Authority
MX
Mexico
Prior art keywords
dna binding
binding proteins
engineered dna
engineered
compositions
Prior art date
Application number
MX2020005561A
Other languages
English (en)
Inventor
Kartik Ramamoorthi
Andrew Young
Stephanie Tagliatela
David Oberkofler
Anne Tanenhaus
Original Assignee
Encoded Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Encoded Therapeutics Inc filed Critical Encoded Therapeutics Inc
Publication of MX2020005561A publication Critical patent/MX2020005561A/es

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • 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
    • C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/30—Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/30—Vector systems having a special element relevant for transcription being an enhancer not forming part of the promoter region

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

En la presente se proporcionan composiciones y métodos de uso de las mimas que comprenden una proteína de unión a ADN que no se presenta de manera natural que modula la expresión de un gen endógeno.
MX2020005561A 2017-12-01 2018-11-30 Proteinas modificadas de union a adn. MX2020005561A (es)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201762593824P 2017-12-01 2017-12-01
US201762610014P 2017-12-22 2017-12-22
US201862618966P 2018-01-18 2018-01-18
US201862641806P 2018-03-12 2018-03-12
US201862646198P 2018-03-21 2018-03-21
US201862664814P 2018-04-30 2018-04-30
US201862664817P 2018-04-30 2018-04-30
PCT/US2018/063498 WO2019109051A1 (en) 2017-12-01 2018-11-30 Engineered dna binding proteins

Publications (1)

Publication Number Publication Date
MX2020005561A true MX2020005561A (es) 2020-10-12

Family

ID=66664596

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020005561A MX2020005561A (es) 2017-12-01 2018-11-30 Proteinas modificadas de union a adn.

Country Status (15)

Country Link
US (1) US20200397917A1 (es)
EP (1) EP3717505A4 (es)
JP (3) JP7401432B2 (es)
KR (1) KR20200107949A (es)
CN (2) CN118345115A (es)
AU (2) AU2018375192B2 (es)
BR (1) BR112020010883A2 (es)
CA (1) CA3083765A1 (es)
CL (1) CL2020001451A1 (es)
IL (1) IL274926A (es)
MA (1) MA50942A (es)
MX (1) MX2020005561A (es)
SG (1) SG11202004926WA (es)
TW (2) TW202319538A (es)
WO (1) WO2019109051A1 (es)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6923517B2 (ja) 2015-10-09 2021-08-18 ユニバーシティ・オブ・サザンプトン 遺伝子発現の調節及び脱制御されたタンパク質発現のスクリーニング
CN116059236A (zh) 2015-12-14 2023-05-05 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
EP3624858A4 (en) * 2017-05-19 2021-06-23 Encoded Therapeutics, Inc. HIGH ACTIVITY REGULATORY ELEMENTS
EP3673080B1 (en) 2017-08-25 2023-10-18 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
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
BR112021024055A2 (pt) * 2019-05-29 2022-02-08 Encoded Therapeutics Inc Composições e métodos para a regulação seletiva de gene
CA3147643A1 (en) 2019-09-23 2021-04-01 Omega Therapeutics, Inc. Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (hnf4.alpha.) gene expression
GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector
CA3166986A1 (en) * 2020-01-07 2021-07-15 Sumitomo Pharma Co., Ltd. Therapeutic agent for tauopathies
CA3173647A1 (en) 2020-05-11 2021-11-18 Isabel AZNAREZ Opa1 antisense oligomers for treatment of conditions and diseases
EP4150083A1 (en) * 2020-05-13 2023-03-22 F. Hoffmann-La Roche AG Oligonucleotide agonists targeting progranulin
WO2022125420A1 (en) * 2020-12-09 2022-06-16 City Of Hope Compounds and methods to treat cystic fibrosis
CN115074389A (zh) * 2022-06-13 2022-09-20 华南农业大学 稳定表达鸭坦布苏病毒ns1蛋白的细胞系的构建方法及其应用

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US20030104526A1 (en) * 1999-03-24 2003-06-05 Qiang Liu Position dependent recognition of GNN nucleotide triplets by zinc fingers
US7329728B1 (en) * 1999-10-25 2008-02-12 The Scripps Research Institute Ligand activated transcriptional regulator proteins
ES2409080T3 (es) * 2001-01-22 2013-06-24 Sangamo Biosciences Inc. Proteínas de unión con dedos de zinc modificadas
ATE441712T1 (de) * 2001-02-21 2009-09-15 Novartis Pharma Gmbh Die nukleotidsequenz ann bindende zink-finger domänen
EP1481087A4 (en) * 2002-02-07 2005-12-21 Scripps Research Inst ZINC FINGER LIBRARIES
WO2009060316A2 (en) * 2007-09-14 2009-05-14 The Governors Of The University Of Alberta Hepatitis b virus-binding polypeptides and methods of use thereof
BR112014005234A2 (pt) * 2011-09-06 2017-04-11 Curna Inc tratamento de doenças relacionadas com subunididades alfa dos canais de sódio dependentes de voltagem (scnxa) com pequenas moléculas
KR102530118B1 (ko) * 2012-07-25 2023-05-08 더 브로드 인스티튜트, 인코퍼레이티드 유도 ｄｎａ 결합 단백질 및 게놈 교란 도구 및 이의 적용
AR095982A1 (es) * 2013-04-03 2015-11-25 Aliophtha Ag Factores de transcripción artificiales modificados genéticamente para superar el atrapamiento endosómico
TW201514201A (zh) * 2013-04-03 2015-04-16 Aliophtha Ag 調控核受體之人工轉錄因子及其治療用途
US10076574B2 (en) * 2013-10-25 2018-09-18 Wayne State University Methods, systems and compositions relating to cell conversion via protein-induced in-vivo cell reprogramming
WO2015153760A2 (en) * 2014-04-01 2015-10-08 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a nervous system disorder
US20180346531A1 (en) * 2015-09-15 2018-12-06 Regents Of The University Of California Compositions and methods for delivering biotherapeutics
CN116059236A (zh) * 2015-12-14 2023-05-05 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
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Also Published As

Publication number Publication date
CA3083765A1 (en) 2019-06-06
CN111727259B (zh) 2024-04-19
US20200397917A1 (en) 2020-12-24
KR20200107949A (ko) 2020-09-16
WO2019109051A1 (en) 2019-06-06
AU2018375192A1 (en) 2020-07-02
CL2020001451A1 (es) 2020-11-06
TW201925474A (zh) 2019-07-01
BR112020010883A2 (pt) 2020-11-10
TWI835761B (zh) 2024-03-21
MA50942A (fr) 2020-10-07
JP2024069591A (ja) 2024-05-21
EP3717505A4 (en) 2021-12-01
CN118345115A (zh) 2024-07-16
CN111727259A (zh) 2020-09-29
IL274926A (en) 2020-07-30
AU2023285810A1 (en) 2024-01-18
SG11202004926WA (en) 2020-06-29
EP3717505A1 (en) 2020-10-07
AU2018375192B2 (en) 2023-11-09
JP2021505133A (ja) 2021-02-18
TW202319538A (zh) 2023-05-16
JP7401432B2 (ja) 2023-12-19
JP2022171792A (ja) 2022-11-11

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