CA3083765A1 - Engineered dna binding proteins - Google Patents

Engineered dna binding proteins Download PDF

Info

Publication number
CA3083765A1
CA3083765A1 CA3083765A CA3083765A CA3083765A1 CA 3083765 A1 CA3083765 A1 CA 3083765A1 CA 3083765 A CA3083765 A CA 3083765A CA 3083765 A CA3083765 A CA 3083765A CA 3083765 A1 CA3083765 A1 CA 3083765A1
Authority
CA
Canada
Prior art keywords
expression cassette
fold
dna binding
etf
expression
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.)
Pending
Application number
CA3083765A
Other languages
English (en)
French (fr)
Inventor
Stephanie TAGLIATELA
Anne TANENHAUS
Kartik RAMAMOORTHI
Andrew Young
David OBERKOFLER
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.)
Encoded Therapeutics Inc
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 CA3083765A1 publication Critical patent/CA3083765A1/en
Pending legal-status Critical Current

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
    • C12N15/63—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
    • 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
    • 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
    • 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
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (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)
CA3083765A 2017-12-01 2018-11-30 Engineered dna binding proteins Pending CA3083765A1 (en)

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
US201762593824P 2017-12-01 2017-12-01
US62/593,824 2017-12-01
US201762610014P 2017-12-22 2017-12-22
US62/610,014 2017-12-22
US201862618966P 2018-01-18 2018-01-18
US62/618,966 2018-01-18
US201862641806P 2018-03-12 2018-03-12
US62/641,806 2018-03-12
US201862646198P 2018-03-21 2018-03-21
US62/646,198 2018-03-21
US201862664814P 2018-04-30 2018-04-30
US201862664817P 2018-04-30 2018-04-30
US62/664,817 2018-04-30
US62/664,814 2018-04-30
PCT/US2018/063498 WO2019109051A1 (en) 2017-12-01 2018-11-30 Engineered dna binding proteins

Publications (1)

Publication Number Publication Date
CA3083765A1 true CA3083765A1 (en) 2019-06-06

Family

ID=66664596

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3083765A Pending CA3083765A1 (en) 2017-12-01 2018-11-30 Engineered dna binding proteins

Country Status (15)

Country Link
US (2) US12083188B2 (enExample)
EP (1) EP3717505A4 (enExample)
JP (3) JP7401432B2 (enExample)
KR (1) KR20200107949A (enExample)
CN (2) CN111727259B (enExample)
AU (2) AU2018375192B2 (enExample)
BR (1) BR112020010883A2 (enExample)
CA (1) CA3083765A1 (enExample)
CL (1) CL2020001451A1 (enExample)
IL (2) IL274926B1 (enExample)
MA (1) MA50942A (enExample)
MX (1) MX2020005561A (enExample)
SG (1) SG11202004926WA (enExample)
TW (3) TWI835761B (enExample)
WO (1) WO2019109051A1 (enExample)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102422625B1 (ko) 2015-10-09 2022-07-20 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
KR102604132B1 (ko) 2015-12-14 2023-11-17 콜드스프링하버러보러토리 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머
MA49153A (fr) * 2017-05-19 2021-03-24 Encoded Therapeutics Inc Éléments régulateurs à haute activité
CN111278991B (zh) 2017-08-25 2022-04-01 斯托克制药公司 用于治疗病况和疾病的反义寡聚体
JP7401432B2 (ja) 2017-12-01 2023-12-19 エンコーデッド セラピューティクス， インコーポレイテッド 操作されたｄｎａ結合タンパク質
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
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
MX2021014460A (es) * 2019-05-29 2022-02-11 Encoded Therapeutics Inc Composiciones y metodos para la regulacion genica selectiva.
US11987791B2 (en) 2019-09-23 2024-05-21 Omega Therapeutics, Inc. Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (HNF4α) gene expression
GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector
JPWO2021141041A1 (enExample) * 2020-01-07 2021-07-15
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
US12203071B2 (en) 2020-05-13 2025-01-21 Hoffmann-La Roche Inc. Oligonucleotide agonists targeting progranulin
AR123041A1 (es) * 2020-07-24 2022-10-26 Univ Massachusetts Transactivadores del dominio de unión al adn y usos de los mismos
US20240024372A1 (en) * 2020-12-09 2024-01-25 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
CN120418436A (zh) * 2022-12-29 2025-08-01 编码治疗公司 用于表征效力的材料和方法

Family Cites Families (148)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
CA2181548C (en) 1994-01-18 2009-11-03 Carlos F. Barbas, Iii Zinc finger protein derivatives and methods therefor
CA2187626C (en) 1994-04-13 2009-11-03 Michael G. Kaplitt Aav-mediated delivery of dna to cells of the nervous system
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
USRE45721E1 (en) 1994-08-20 2015-10-06 Gendaq, Ltd. Relating to binding proteins for recognition of DNA
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
US6436708B1 (en) 1997-04-17 2002-08-20 Paola Leone Delivery system for gene therapy to the brain
CA2205076A1 (en) 1997-05-14 1998-11-14 Jim Hu Episomal expression cassettes for gene therapy
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
ATE466952T1 (de) 1998-03-02 2010-05-15 Massachusetts Inst Technology Poly-zinkfinger-proteine mit verbesserten linkern
US6303370B1 (en) 1998-03-24 2001-10-16 Mayo Foundation For Medical Education And Research Tissue-specific regulatory elements
CA2246005A1 (en) 1998-10-01 2000-04-01 Hsc Research And Development Limited Partnership Hybrid genes for gene therapy in erythroid cells
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6503717B2 (en) 1999-12-06 2003-01-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US20030104526A1 (en) * 1999-03-24 2003-06-05 Qiang Liu Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US6649371B1 (en) 1999-06-11 2003-11-18 Neurosearch A/S Potassium channel KCNQ5 and sequences encoding the same
US7329728B1 (en) * 1999-10-25 2008-02-12 The Scripps Research Institute Ligand activated transcriptional regulator proteins
EP1232276B8 (en) 1999-11-16 2007-06-27 Genzyme Corporation Vectors and transgenes with regulatory elements for gene delivery to the liver
EP1103610A1 (en) 1999-11-26 2001-05-30 Introgene B.V. Production of vaccines from immortalised mammalian cell lines
JP4048053B2 (ja) 1999-11-26 2008-02-13 マクギル ユニバーシティー 特発性全身てんかんについてのローカス、当該ローカスのミューテーション、及びてんかんの評価、診断、予後、または治療のための当該ローカスの使用方法
KR20020086508A (ko) 2000-02-08 2002-11-18 상가모 바이오사이언스 인코포레이티드 약물 발견용 세포
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
US6436703B1 (en) 2000-03-31 2002-08-20 Hyseq, Inc. Nucleic acids and polypeptides
ATE353361T1 (de) 2000-04-28 2007-02-15 Sangamo Biosciences Inc Gezielten modifikation der chromatinstruktur
FR2808803B1 (fr) 2000-05-11 2004-12-10 Agronomique Inst Nat Rech Cellules es modifiees et gene specifique de cellules es
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith Methods and compositions for interaction trap assays
PT1287134E (pt) 2000-06-07 2007-10-04 Ortho Mcneil Pharm Inc Subunidade beta-1a humana do canal de sódio dependente de voltagem e métodos de utilização
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US6812339B1 (en) 2000-09-08 2004-11-02 Applera Corporation Polymorphisms in known genes associated with human disease, methods of detection and uses thereof
US6919204B2 (en) 2000-09-29 2005-07-19 Sangamo Biosciences, Inc. Modulation of gene expression using localization domains
USH2191H1 (en) 2000-10-24 2007-06-05 Snp Consortium Identification and mapping of single nucleotide polymorphisms in the human genome
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
ES2409080T3 (es) * 2001-01-22 2013-06-24 Sangamo Biosciences Inc. Proteínas de unión con dedos de zinc modificadas
US20030051266A1 (en) 2001-02-14 2003-03-13 Serafini Tito Andrew Collections of transgenic animal lines (living library)
US7067617B2 (en) 2001-02-21 2006-06-27 The Scripps Research Institute Zinc finger binding domains for nucleotide sequence ANN
ATE441712T1 (de) * 2001-02-21 2009-09-15 Novartis Pharma Gmbh Die nukleotidsequenz ann bindende zink-finger domänen
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
AUPR492201A0 (en) 2001-05-10 2001-06-07 Bionomics Limited Novel mutation
EP1402043A1 (en) 2001-07-03 2004-03-31 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods of administering vectors to synaptically connected neurons
WO2003016496A2 (en) 2001-08-20 2003-02-27 The Scripps Research Institute Zinc finger binding domains for cnn
US6998118B2 (en) 2001-12-21 2006-02-14 The Salk Institute For Biological Studies Targeted retrograde gene delivery for neuronal protection
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
WO2003066828A2 (en) * 2002-02-07 2003-08-14 The Scripps Research Institute Zinc finger libraries
AU2003219933A1 (en) 2002-02-25 2003-09-09 Vanderbilt University Expression system for human brain-specific voltage-gated sodium channel, type 1
FR2836924B1 (fr) 2002-03-08 2005-01-14 Vivalis Lignees de cellules aviaires utiles pour la production de substances d'interet
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
EP2474631B1 (en) 2002-12-20 2014-02-12 Celera Corporation Genetic polymorphisms associated with myocardial infarction, methods of detection and uses thereof
ES2334231T3 (es) 2003-01-28 2010-03-08 F. Hoffmann-La Roche Ag Uso de secuencias regulatorias para la expresion transitoria especifica en celulas determinadas de manera neuronal.
US20040258666A1 (en) 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
EP1625210B1 (en) 2003-05-21 2010-12-01 Genzyme Corporation Methods for producing preparations of recombinant aav virions substantially free of empty capsids
US7094600B2 (en) 2003-06-26 2006-08-22 The Research Foundation Of State University Of New York Screen for sodium channel modulators
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
EP1528101A1 (en) 2003-11-03 2005-05-04 ProBioGen AG Immortalized avian cell lines for virus production
KR20070052694A (ko) 2004-01-09 2007-05-22 더 리젠트스 오브 더 유니이버시티 오브 캘리포니아 유전자 발현을 위한 세포-유형-특이적 패턴
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
AU2005233550B2 (en) 2004-04-08 2010-11-18 Sangamo Therapeutics, Inc. Treatment of neuropathic pain with zinc finger proteins
JP4564052B2 (ja) 2004-04-08 2010-10-20 サンガモ バイオサイエンシズ インコーポレイテッド 心筋収縮能調節用の方法及び組成物
WO2005099771A2 (en) 2004-04-08 2005-10-27 Sangamo Biosciences, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
EP1747277B1 (en) 2004-04-14 2011-08-10 Agency for Science, Technology and Research Gene delivery to neuronal cells
TWI293307B (en) 2004-09-30 2008-02-11 Ind Tech Res Inst A liver-specific chimeric regulatory sequence and use thereof
JP4988606B2 (ja) 2005-02-28 2012-08-01 サンガモ バイオサイエンシズ インコーポレイテッド 抗血管新生方法及び組成物
FR2884255B1 (fr) 2005-04-11 2010-11-05 Vivalis Utilisation de lignees de cellules souches aviaires ebx pour la production de vaccin contre la grippe
CN101287834B (zh) 2005-05-27 2016-11-09 圣拉法埃莱医院有限公司 基因载体
JP2009504156A (ja) 2005-08-11 2009-02-05 ザ スクリップス リサーチ インスティテュート Ｃｎｎに対するジンクフィンガー結合ドメイン
EP1783645A1 (en) 2005-11-08 2007-05-09 Actigenics Methods for the identification of microRNA and their applications in research and human health
CN101864392B (zh) 2005-12-13 2016-03-23 国立大学法人京都大学 核重新编程因子
US20070161031A1 (en) 2005-12-16 2007-07-12 The Board Of Trustees Of The Leland Stanford Junior University Functional arrays for high throughput characterization of gene expression regulatory elements
JP2009519710A (ja) 2005-12-16 2009-05-21 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 遺伝子発現調節エレメントのハイスループットでの特徴付けのための機能性アレイ
JP4144637B2 (ja) 2005-12-26 2008-09-03 セイコーエプソン株式会社 印刷材収容体、基板、印刷装置および印刷材収容体を準備する方法
CA2645186A1 (en) 2006-01-20 2007-07-26 The Regents Of The University Of California Transplantation of neural cells
EP2046806A4 (en) 2006-07-05 2009-10-21 Scripps Research Inst CHIMERIC RECOMBINASES WITH ZINC FINGERS OPTIMIZED FOR CATALYSIS BY DIRECT EVOLUTION
US7947837B2 (en) 2006-10-25 2011-05-24 Polyera Corporation Organic semiconductor materials and methods of preparing and use thereof
WO2008073303A2 (en) 2006-12-07 2008-06-19 Switchgear Genomics Transcriptional regulatory elements of biological pathways, tools, and methods
CA2684242C (en) 2007-03-23 2019-11-12 Wisconsin Alumni Research Foundation Somatic cell reprogramming
EP1985305A1 (en) 2007-04-24 2008-10-29 Vivalis Duck embryonic derived stem cell lines for the production of viral vaccines
EP1995309A1 (en) 2007-05-21 2008-11-26 Vivalis Recombinant protein production in avian EBx® cells
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
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
ITRM20070523A1 (it) 2007-10-05 2009-04-06 Consiglio Nazionale Ricerche Acido nucleico codificante per una proteina regolatrice specifica della trascrizione dell'utrofina proteina da esso codificata e sue applicazioni
US20100297674A1 (en) 2008-01-22 2010-11-25 Chromocell Corporation NOVEL CELL LINES EXPRESSING NaV AND METHODS USING THEM
US20110165129A1 (en) 2008-05-06 2011-07-07 Arnold Kriegstein Ameliorating Nervous Systems Disorders
US9267151B2 (en) 2008-08-20 2016-02-23 Brainco Biopharma, S.L. STXBP1 overexpressing mouse and its uses in screening of treatments for neuropsychiatric illness
WO2010037143A1 (en) 2008-09-29 2010-04-01 The University Of Montana Vectors and methods of treating brain seizures
ES2881050T3 (es) 2009-06-16 2021-11-26 Genzyme Corp Métodos mejorados para la purificación de vectores de AAV recombinantes
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
US8815779B2 (en) 2009-09-16 2014-08-26 SwitchGear Genomics, Inc. Transcription biomarkers of biological responses and methods
US20110135611A1 (en) 2009-12-03 2011-06-09 The J. David Gladstone Institutes Methods for treating apolipoprotein e4-associated disorders
US20110203007A1 (en) 2010-02-16 2011-08-18 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Assays of neurodegenerative disorders, including frontotemporal dementia and amyotrophic lateral sclerosis
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
WO2011139349A1 (en) 2010-05-03 2011-11-10 Sangamo Biosciences, Inc. Compositions for linking zinc finger modules
WO2011146121A1 (en) 2010-05-17 2011-11-24 Sangamo Biosciences, Inc. Novel dna-binding proteins and uses thereof
PT2585596T (pt) 2010-06-23 2021-03-23 Curna Inc Tratamento de doenças relacionadas com a subunidade alfa de canais de sódio dependentes de voltagem (scna) por inibição da transcrição antissentido natural para scna
EP2634253B1 (en) 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
WO2012061828A2 (en) 2010-11-05 2012-05-10 The Regents Of The University Of California Neuronal specific targeting of caveolin expression to restore synaptic signaling and improve cognitive function in the neurodegenerative brain and motor function in spinal cord
CA2817256A1 (en) 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas
CA2822462A1 (en) 2010-12-20 2012-06-28 The General Hospital Corporation Polycomb-associated non-coding rnas
WO2012112578A2 (en) 2011-02-14 2012-08-23 The Children's Hospital Of Philadelphia Improved aav8 vector with enhanced functional activity and methods of use thereof
WO2013033627A2 (en) 2011-09-01 2013-03-07 The Regents Of The University Of California Diagnosis and treatment of arthritis using epigenetics
JP6125505B2 (ja) * 2011-09-06 2017-05-10 カッパーアールエヌエー，インコーポレイテッド 小分子による電位開口型ナトリウムチャネル・アルファサブユニット関連疾患の治療
KR20120087860A (ko) 2012-02-03 2012-08-07 주식회사 툴젠 신규한 징크 핑거 뉴클레아제 및 이의 용도
AU2013221212B2 (en) 2012-02-17 2018-08-09 The Children's Hospital Of Philadelphia AAV vector compositions and methods for gene transfer to cells, organs and tissues
US9284575B2 (en) 2012-03-06 2016-03-15 Duke University Synthetic regulation of gene expression
WO2013155222A2 (en) 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
EP3808844A1 (en) 2012-07-25 2021-04-21 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
CA2922005A1 (en) 2012-09-27 2014-04-03 Population Diagnostics, Inc. Methods and compositions for screening and treating developmental disorders
EP2909324B1 (en) 2012-10-25 2020-02-26 Tocagen Inc. Retroviral vector with mini-promoter cassette
CA2904156C (en) 2013-03-15 2023-01-10 The Children's Hospital Of Philadelphia Vectors comprising stuffer/filler polynucleotide sequences and methods of use
TW201514202A (zh) * 2013-04-03 2015-04-16 Aliophtha Ag 經工程改造以克服內體捕獲之人工轉錄因子
AR095984A1 (es) * 2013-04-03 2015-11-25 Aliophtha Ag Factores artificiales de transcripción que regulan receptores nucleares y su uso terapéutico
CA3209821A1 (en) 2013-05-15 2014-11-20 Regents Of The University Of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
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
AU2015231353B2 (en) 2014-03-18 2020-11-05 Sangamo Therapeutics, Inc. Methods and compositions for regulation of zinc finger protein expression
WO2015153760A2 (en) * 2014-04-01 2015-10-08 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a nervous system disorder
WO2015188065A1 (en) 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design
WO2016016119A1 (en) 2014-07-26 2016-02-04 Consiglio Nazionale Delle Ricerche Compositions and methods for treatment of muscular dystrophy
DK3233130T3 (da) 2014-12-17 2021-06-21 Fundacion Para La Investig Medica Aplicada Nukleinsyrekonstruktioner og genterapivektorer til anvendelse i behandlingen af wilson-sygdom
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
CA3019315A1 (en) 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
CN104846015B (zh) 2015-05-27 2018-03-27 深圳先进技术研究院 特异性兴奋伏隔核中的gaba能神经元的组合物及其在改善精神分裂症异样行为中的应用
WO2017048466A1 (en) * 2015-09-15 2017-03-23 The Regents Of The University Of California Compositions and methods for delivering biotherapeutics
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
AU2016343979A1 (en) 2015-10-29 2018-05-10 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
KR102604132B1 (ko) * 2015-12-14 2023-11-17 콜드스프링하버러보러토리 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머
CA3016314A1 (en) 2016-03-02 2017-09-08 Julianne REIDERS Therapy for frontotemporal dementia
US20190127713A1 (en) * 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
US20190255106A1 (en) 2016-09-07 2019-08-22 Flagship Pioneering Inc. Methods and compositions for modulating gene expression
JP2018088888A (ja) 2016-12-06 2018-06-14 国立研究開発法人理化学研究所 Ｓｃｎ１ａ遺伝子の発現増強法
JP7128190B2 (ja) 2016-12-30 2022-08-30 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ウィルソン病を処置するための遺伝子療法
EP3579858A4 (en) 2017-02-07 2020-12-23 The Regents of The University of California GENE THERAPY AGAINST HAPLOINSUFFICIENCY
EP4403642A3 (en) 2017-04-03 2024-10-09 Encoded Therapeutics, Inc. Tissue selective transgene expression
MA49153A (fr) 2017-05-19 2021-03-24 Encoded Therapeutics Inc Éléments régulateurs à haute activité
JP7401432B2 (ja) * 2017-12-01 2023-12-19 エンコーデッド セラピューティクス， インコーポレイテッド 操作されたｄｎａ結合タンパク質
WO2019224864A1 (ja) 2018-05-21 2019-11-28 国立研究開発法人理化学研究所 Ｓｃｎ１ａ遺伝子の発現増強法とそれによるドラベ症候群の治療法
EP3931336A4 (en) 2019-02-28 2023-01-04 Encoded Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF LAMINOPATHIES
MX2021014460A (es) 2019-05-29 2022-02-11 Encoded Therapeutics Inc Composiciones y metodos para la regulacion genica selectiva.

Also Published As

Publication number Publication date
JP7401432B2 (ja) 2023-12-19
IL274926B1 (en) 2025-12-01
AU2018375192A1 (en) 2020-07-02
EP3717505A1 (en) 2020-10-07
CN118345115A (zh) 2024-07-16
AU2023285810A1 (en) 2024-01-18
BR112020010883A2 (pt) 2020-11-10
WO2019109051A1 (en) 2019-06-06
TW201925474A (zh) 2019-07-01
JP7634507B2 (ja) 2025-02-21
EP3717505A4 (en) 2021-12-01
US20240408242A1 (en) 2024-12-12
JP2021505133A (ja) 2021-02-18
US12083188B2 (en) 2024-09-10
SG11202004926WA (en) 2020-06-29
KR20200107949A (ko) 2020-09-16
JP2022171792A (ja) 2022-11-11
AU2018375192B2 (en) 2023-11-09
TW202319538A (zh) 2023-05-16
MA50942A (fr) 2020-10-07
CN111727259A (zh) 2020-09-29
IL274926A (en) 2020-07-30
TW202421790A (zh) 2024-06-01
MX2020005561A (es) 2020-10-12
CN111727259B (zh) 2024-04-19
JP2024069591A (ja) 2024-05-21
US20200397917A1 (en) 2020-12-24
CL2020001451A1 (es) 2020-11-06
TWI835761B (zh) 2024-03-21
IL323220A (en) 2025-11-01

Similar Documents

Publication Publication Date Title
US20240408242A1 (en) Engineered dna binding proteins
JP7477675B2 (ja) 組織選択的導入遺伝子発現
TWI872078B (zh) 用於選擇性基因調節的組合物和方法
JP2024109708A (ja) 非症候性感音性聴力喪失の治療方法
US12516350B2 (en) Compositions and methods for selective gene regulation
EA049067B1 (ru) Тканеселективная экспрессия трансгена

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20220912

EEER Examination request

Effective date: 20220912

EEER Examination request

Effective date: 20220912

EEER Examination request

Effective date: 20220912

EEER Examination request

Effective date: 20220912