ATE410518T1 - Kompaktnukleinsäure und ihre verabreichung in zellen - Google Patents

Kompaktnukleinsäure und ihre verabreichung in zellen

Info

Publication number
ATE410518T1
ATE410518T1 AT95914173T AT95914173T ATE410518T1 AT E410518 T1 ATE410518 T1 AT E410518T1 AT 95914173 T AT95914173 T AT 95914173T AT 95914173 T AT95914173 T AT 95914173T AT E410518 T1 ATE410518 T1 AT E410518T1
Authority
AT
Austria
Prior art keywords
nucleic acid
nucleic acids
cells
compacted
administration
Prior art date
Application number
AT95914173T
Other languages
English (en)
Inventor
Richard W Hanson
Jose C Perales
Thomas W Ferkol
Original Assignee
Univ Ohio
Univ Case Western Reserve
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 Univ Ohio, Univ Case Western Reserve filed Critical Univ Ohio
Application granted granted Critical
Publication of ATE410518T1 publication Critical patent/ATE410518T1/de

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Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K19/00Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424Serine endopeptidases (3.4.21)
    • C12N9/644Coagulation factor IXa (3.4.21.22)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • C12Y304/21022Coagulation factor IXa (3.4.21.22)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Obesity (AREA)
  • Pulmonology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Cardiology (AREA)
AT95914173T 1994-03-23 1995-03-23 Kompaktnukleinsäure und ihre verabreichung in zellen ATE410518T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US21653494A 1994-03-23 1994-03-23

Publications (1)

Publication Number Publication Date
ATE410518T1 true ATE410518T1 (de) 2008-10-15

Family

ID=22807432

Family Applications (1)

Application Number Title Priority Date Filing Date
AT95914173T ATE410518T1 (de) 1994-03-23 1995-03-23 Kompaktnukleinsäure und ihre verabreichung in zellen

Country Status (9)

Country Link
US (3) US5877302A (de)
EP (1) EP0752005B1 (de)
JP (2) JP4285766B2 (de)
AT (1) ATE410518T1 (de)
AU (1) AU696455C (de)
CA (1) CA2186118C (de)
DE (1) DE69535853D1 (de)
ES (1) ES2316148T3 (de)
WO (1) WO1995025809A1 (de)

Families Citing this family (179)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5218704A (en) * 1989-10-30 1993-06-08 Texas Instruments Real-time power conservation for portable computers
JP4285766B2 (ja) * 1994-03-23 2009-06-24 オハイオ ユニバーシティ 緻密にした核酸および細胞へのデリバリ
US5670347A (en) 1994-05-11 1997-09-23 Amba Biosciences Llc Peptide-mediated gene transfer
US6008202A (en) 1995-01-23 1999-12-28 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US20030069173A1 (en) * 1998-03-16 2003-04-10 Life Technologies, Inc. Peptide-enhanced transfections
US6120794A (en) * 1995-09-26 2000-09-19 University Of Pittsburgh Emulsion and micellar formulations for the delivery of biologically active substances to cells
US5994316A (en) * 1996-02-21 1999-11-30 The Immune Response Corporation Method of preparing polynucleotide-carrier complexes for delivery to cells
WO1997039019A1 (en) * 1996-04-12 1997-10-23 University Of Pittsburgh Novel cationic cholesteryl derivatives containing cyclic polar groups
US6258792B1 (en) 1996-04-12 2001-07-10 University Of Pittsburgh Cationic cholesteryl derivatives containing cyclic polar groups
CA2259179C (en) 1996-07-03 2008-09-23 University Of Pittsburgh Emulsion formulations for hydrophilic active agents
EP0935471B1 (de) * 1996-10-09 2005-03-09 Oxford Biomedica (UK) Limited Mononukleare phagozyten zur verabreichung von therapeutischen arzneimitteln
US6797276B1 (en) 1996-11-14 2004-09-28 The United States Of America As Represented By The Secretary Of The Army Use of penetration enhancers and barrier disruption agents to enhance the transcutaneous immune response
US5980898A (en) 1996-11-14 1999-11-09 The United States Of America As Represented By The U.S. Army Medical Research & Material Command Adjuvant for transcutaneous immunization
US20060002959A1 (en) * 1996-11-14 2006-01-05 Government Of The United States Skin-sctive adjuvants for transcutaneous immuization
US20060002949A1 (en) * 1996-11-14 2006-01-05 Army Govt. Of The Usa, As Rep. By Secretary Of The Office Of The Command Judge Advocate, Hq Usamrmc. Transcutaneous immunization without heterologous adjuvant
US7097829B2 (en) 1996-11-21 2006-08-29 Cedars-Sinai Medical Center Transgenic cells transfected with pituitary tumor transforming gene (PTTG)) expression vectors and uses therefor
US6894031B1 (en) * 1996-11-21 2005-05-17 Cedars-Sinai Medical Center Pituitary tumor transforming gene (PTTG) carboxy-terminal peptides and methods of use thereof to inhibit neoplastic cellular proliferation and/or transformation
US6913926B2 (en) * 1996-11-21 2005-07-05 Cedars-Sinai Medical Center Method of regulating biological activity of pituitary tumor transforming gene (PTTG)1 using PTTG2
US20040258703A1 (en) * 1997-11-14 2004-12-23 The Government Of The Us, As Represented By The Secretary Of The Army Skin-active adjuvants for transcutaneous immunization
WO1999040771A2 (en) 1998-02-13 1999-08-19 Selective Genetics, Inc. Concurrent flow mixing for preparing compositions comprising gene therapy vectors
DE19858005B4 (de) * 1998-12-16 2007-02-08 november Aktiengesellschaft, Gesellschaft für Molekulare Medizin Synthetisches Nucleinsäure-Partikel
AU4188100A (en) * 1999-04-08 2000-11-14 Gregory M. Glenn Dry formulation for transcutaneous immunization
US6281005B1 (en) * 1999-05-14 2001-08-28 Copernicus Therapeutics, Inc. Automated nucleic acid compaction device
US6770740B1 (en) 1999-07-13 2004-08-03 The Regents Of The University Of Michigan Crosslinked DNA condensate compositions and gene delivery methods
EP1268555A2 (de) * 2000-03-27 2003-01-02 The Regents of The University of California Gerichtete liganden gegen die nicht-sekrethorische komponente, und nicht-stengelregion von pigr und methoden zu deren verwendung
US20020042388A1 (en) * 2001-05-01 2002-04-11 Cooper Mark J. Lyophilizable and enhanced compacted nucleic acids
EP1289567B1 (de) * 2000-05-31 2008-07-30 Copernicus Therapeutics, Inc. Lyophilisierbare und verbesserte kompaktierte nukleinsäure
GB0027328D0 (en) * 2000-06-23 2000-12-27 Aventis Pharma Inc Bioengineered vehicles for targeted nucleic acid delivery
EP1330516B1 (de) * 2000-11-03 2007-01-17 The Regents Of The University Of Michigan Oberflächentransfektion und expressionsverfahren
US20020081736A1 (en) * 2000-11-03 2002-06-27 Conroy Susan E. Nucleic acid delivery
US6812217B2 (en) 2000-12-04 2004-11-02 Medtronic, Inc. Medical device and methods of use
DE10063112A1 (de) * 2000-12-18 2002-06-20 Bayer Ag Verfahren zur Erhöhung der klinischen Spezifität bei der Detektion von Tumoren und ihren Vorstufen durch simultane Messung von mindestens zwei verschiedenen molekularen Markern
DK1372708T3 (da) * 2001-02-13 2008-10-20 Us Gov Sec Army Vaccine til transkutan immunisering mod rejsediarre
US20020182626A1 (en) * 2001-03-16 2002-12-05 Daniel Tuse Episomal non-transforming nucleic acid elements in functional genomic and antigenic applications
US7294511B2 (en) * 2001-03-22 2007-11-13 Chromos Molecular Systems, Inc. Methods for delivering nucleic acid molecules into cells and assessment thereof
JP2004535388A (ja) * 2001-04-30 2004-11-25 ターゲティッド ジェネティクス コーポレイション 脂質含有薬物送達複合体およびそれらの生成方法
GB0114719D0 (en) * 2001-06-15 2001-08-08 Glaxo Group Ltd Compound
US6909030B2 (en) 2001-10-15 2005-06-21 Cedars-Sinai Medical Center PTTG knockout rodent as a model to study mechanisms for various physiological phenomena, including diabetes
US20040016013A1 (en) * 2002-07-18 2004-01-22 Gonzalo Hortelano Transgenic animals produced using oral administration of a genetic agent coupled to a transporting agent
US20040014698A1 (en) * 2002-07-18 2004-01-22 Gonzalo Hortelano Oral administration of therapeutic agent coupled to transporting agent
US20040014704A1 (en) * 2002-07-18 2004-01-22 Gonzalo Hortelano Oral administration of therapeutic agent coupled to transporting agent induces tolerance
US7285269B2 (en) * 2002-12-02 2007-10-23 Amgen Fremont, Inc. Antibodies directed to tumor necrosis factor
CA2513072A1 (en) * 2003-01-09 2004-07-29 Invitrogen Corporation Cellular delivery and activation polypeptide-nucleic acid complexes
TWI433693B (zh) 2003-02-14 2014-04-11 Childrens Hosp & Res Ct Oak 親脂藥物傳送媒介物及其使用方法
US20050136437A1 (en) * 2003-08-25 2005-06-23 Nastech Pharmaceutical Company Inc. Nanoparticles for delivery of nucleic acids and stable double-stranded RNA
US20050208032A1 (en) * 2004-01-16 2005-09-22 Gonzalo Hortelano Oral administration of therapeutic agent coupled to transporting agent
US7358223B2 (en) 2004-10-04 2008-04-15 Nitto Denko Corporation Biodegradable cationic polymers
EP2302055B1 (de) 2004-11-12 2014-08-27 Asuragen, Inc. Verfahren und Zusammensetzungen, die miRNAs und miRNAa-inhibitorischen Molekülen verbunden sind
US20070031512A1 (en) * 2005-08-03 2007-02-08 Amcol International Corporation Virus-interacting layered phyllosilicates and methods of inactivating viruses
US20080184618A1 (en) * 2005-08-03 2008-08-07 Amcol International Virus-Interacting Layered Phyllosilicates and Methods of Use
US20100272769A1 (en) * 2005-08-03 2010-10-28 Amcol International Virus-, Bacteria-, and Fungi-Interacting Layered Phyllosilicates and Methods of Use
US7700541B2 (en) 2006-04-06 2010-04-20 Nitto Denko Corporation Biodegradable cationic polymers
AU2007281261B2 (en) 2006-08-01 2013-03-21 Board Of Regents Of The University Of Texas System Identification of a micro-RNA that activates expression of beta-myosin heavy chain
EP2712930A3 (de) 2006-12-06 2014-04-23 MedImmune, LLC Interferon-alpha-induzierte pharmakodynamische Marker
EP2591795B1 (de) * 2007-02-23 2018-02-28 The Research Foundation Of State University Of New York RNA-Target-Verbindungen und Verfahren zu ihrer Herstellung und Verwendung
US9260476B2 (en) 2007-02-23 2016-02-16 The Research Foundation For The State University Of New York RNA targeting compounds and methods for making and using same
WO2008137066A1 (en) * 2007-05-02 2008-11-13 The Board Of Regents Of The University Of Oklahoma Use of compacted nucleic acid nanoparticles in non-viral treatments of ocular diseases
CA2686861A1 (en) 2007-05-03 2008-11-13 Medimmune, Llc Interferon alpha-induced pharmacodynamic markers
EP2171052B1 (de) 2007-07-12 2014-08-27 Sangamo BioSciences, Inc. Verfahren und zusammensetzung zur deaktivierung der alpha-1,6-fucosyltransferase-genexpression
EP2182969B1 (de) 2007-07-31 2016-11-16 The Board of Regents of The University of Texas System miRNA-FAMILIE ALS FIBROSEMODULATOR UND IHRE VERWENDUNG
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
CA2714365A1 (en) * 2007-12-31 2009-07-09 Monster Medic, Inc. Ambulance cot with an elevating mechanism
EP2265291B1 (de) 2008-03-17 2016-10-19 The Board of Regents of The University of Texas System Identifizierung von mikro-rna bei der erhaltung und regeneration von neuromuskulären synapsen
WO2009151591A2 (en) 2008-06-10 2009-12-17 Sangamo Biosciences, Inc. Methods and compositions for generation of bax- and bak-deficient cell lines
US8268796B2 (en) 2008-06-27 2012-09-18 Children's Hospital & Research Center At Oakland Lipophilic nucleic acid delivery vehicle and methods of use thereof
SG191561A1 (en) 2008-08-22 2013-07-31 Sangamo Biosciences Inc Methods and compositions for targeted single-stranded cleavage and targeted integration
EP2352369B1 (de) 2008-12-04 2017-04-26 Sangamo BioSciences, Inc. Genomänderung bei ratten mithilfe von zink-finger-nukleasen
WO2010084488A1 (en) 2009-01-20 2010-07-29 Ramot At Tel-Aviv University Ltd. Mir-21 promoter driven targeted cancer therapy
AU2010211057B2 (en) 2009-02-04 2014-12-18 Sangamo Therapeutics, Inc. Methods and compositions for treating neuropathies
EP2419511B1 (de) 2009-04-09 2018-01-17 Sangamo Therapeutics, Inc. Gezielte integration in stammzellen
WO2010120814A1 (en) 2009-04-14 2010-10-21 Prometheus Laboratories Inc. Inflammatory bowel disease prognostics
CA2769262C (en) 2009-07-28 2019-04-30 Sangamo Biosciences, Inc. Methods and compositions for treating trinucleotide repeat disorders
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
WO2011060098A1 (en) 2009-11-10 2011-05-19 Prometheus Laboratories Inc. Methods for predicting post-surgery risk associated with ileal pouch-anal anastomosis
MX336846B (es) 2010-01-22 2016-02-03 Sangamo Biosciences Inc Alteracion genomica dirigida.
EP2660318A1 (de) 2010-02-09 2013-11-06 Sangamo BioSciences, Inc. Gezielte Genommodifikation mit partiell einzelsträngigen Donatormolekülen
EP2542678B1 (de) 2010-03-04 2017-04-12 InteRNA Technologies B.V. Mirna-molekül, das durch seine quelle definiert ist, und seine therapeutischen verwendungen bei emt assoziierten krebs
EP2591106A1 (de) 2010-07-06 2013-05-15 InteRNA Technologies B.V. Mirna und ihre diagnostischen und therapeutischen verwendungen für mit melanomen assoziierte erkrankungen oder leiden oder für mit dem aktivierten braf-pfad assoziierte erkrankungen oder leiden
AU2011312562B2 (en) 2010-09-27 2014-10-09 Sangamo Therapeutics, Inc. Methods and compositions for inhibiting viral entry into cells
WO2012067837A1 (en) * 2010-11-15 2012-05-24 Avon Products, Inc. Biofunctional anchored extended-wear cosmetics
EP2474617A1 (de) 2011-01-11 2012-07-11 InteRNA Technologies BV MIR zur Behandlung von Neoangiogenese
US9161995B2 (en) 2011-07-25 2015-10-20 Sangamo Biosciences, Inc. Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (CFTR) gene
CA2848417C (en) 2011-09-21 2023-05-02 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
JP2015502740A (ja) 2011-10-21 2015-01-29 ネステク ソシエテ アノニム 炎症性腸疾患の診断を改善するための方法
EP3369818B1 (de) 2011-12-22 2021-06-09 InteRNA Technologies B.V. Mirna zur behandlung von kopf-hals-karzinom
WO2013130824A1 (en) 2012-02-29 2013-09-06 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
US9486540B2 (en) 2012-03-09 2016-11-08 Northeastern University Methods for delivery to the central nervous system of nucleic acid nanoparticles to treat central nervous system disorders
EP3196301B1 (de) 2012-07-11 2018-10-17 Sangamo Therapeutics, Inc. Verfahren und zusammensetzungen zur behandlung von monogener krankheiten
JP6329537B2 (ja) 2012-07-11 2018-05-23 サンガモ セラピューティクス, インコーポレイテッド 生物学的薬剤の送達のための方法および組成物
SG10201701601WA (en) 2012-08-29 2017-04-27 Sangamo Biosciences Inc Methods and compositions for treatment of a genetic condition
AU2013329186B2 (en) 2012-10-10 2019-02-14 Sangamo Therapeutics, Inc. T cell modifying compounds and uses thereof
EP2917348A1 (de) 2012-11-06 2015-09-16 InteRNA Technologies B.V. Kombination zur behandlung von erkrankungen oder zuständen im zusammenhang mit melanom oder behandlung von erkrankungen und zuständen im zusammenhang mit aktivierten b-raf-signalweg
EP2928303A4 (de) 2012-12-07 2016-07-13 Haplomics Inc Faktor-viii-mutationsreparatur und toleranzinduktion
EP2935627A4 (de) 2012-12-21 2016-09-28 Univ Columbia Biomarker für chronisch traumatische enzephalopathie
US8980864B2 (en) * 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
US9758835B2 (en) 2013-03-15 2017-09-12 Baylor Research Institute Ulcerative colitis (UC)-associated colorectal neoplasia markers
CN105683376A (zh) 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物
US9951330B2 (en) 2013-07-11 2018-04-24 The Trustees Of Columbia University In The City Of New York Micrornas that silence tau expression
CA3131284C (en) 2013-08-28 2023-09-19 David Paschon Compositions for linking dna-binding domains and cleavage domains
EP3052521A1 (de) 2013-10-03 2016-08-10 Moderna Therapeutics, Inc. Polynukleotide zur codierung von low-density-lipoprotein-rezeptor
EP3441468B1 (de) 2013-10-17 2021-05-19 Sangamo Therapeutics, Inc. Freisetzungsverfahren und zusammensetzungen für nukleasevermitteltes genomengineering
WO2015070212A1 (en) 2013-11-11 2015-05-14 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
DK3492593T3 (da) 2013-11-13 2021-11-08 Childrens Medical Center Nukleasemedieret regulering af genekspression
CA2930693A1 (en) 2013-11-15 2015-05-21 The Board Of Trustees Of The Leland Stanford Junior Unversity Methods of treating heart failure with agonists of hypocretin receptor 2
US10772974B2 (en) 2013-11-18 2020-09-15 Beth Israel Deaconess Medical Center, Inc. Compositions and methods for cardiac regeneration
CN105940013B (zh) 2013-12-09 2020-03-27 桑格摩生物科学股份有限公司 用于治疗血友病的方法和组合物
US10072066B2 (en) 2014-02-03 2018-09-11 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a beta thalessemia
CN106459894B (zh) 2014-03-18 2020-02-18 桑格摩生物科学股份有限公司 用于调控锌指蛋白表达的方法和组合物
WO2015171932A1 (en) 2014-05-08 2015-11-12 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
DK3194570T3 (da) 2014-09-16 2021-09-13 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til nukleasemedieret genommodificering og -korrektion i hæmatopoietiske stamceller
CA2981077A1 (en) 2015-04-03 2016-10-06 Dana-Farber Cancer Institute, Inc. Composition and methods of genome editing of b-cells
AU2016261927B2 (en) 2015-05-12 2022-04-07 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
AU2016291778B2 (en) 2015-07-13 2021-05-06 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
GB2592821B (en) 2015-07-31 2022-01-12 Univ Minnesota Modified cells and methods of therapy
JP6853257B2 (ja) 2015-09-23 2021-03-31 サンガモ セラピューティクス, インコーポレイテッド Httリプレッサー及びその使用
BR112018012235A2 (pt) 2015-12-18 2018-12-04 Sangamo Therapeutics Inc rompimento alvejado do receptor de células de mhc
EP3389677B1 (de) 2015-12-18 2024-06-26 Sangamo Therapeutics, Inc. Gezielte zerstörung des t-zell-rezeptors
WO2017123757A1 (en) 2016-01-15 2017-07-20 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
KR20180101442A (ko) 2016-02-02 2018-09-12 상가모 테라퓨틱스, 인코포레이티드 Dna-결합 도메인 및 절단 도메인을 연결시키기 위한 조성물
WO2017222911A1 (en) 2016-06-20 2017-12-28 The Board Of Trustees Of The Leland Stanford Junior University Circular rnas and their use in immunomodulation
MA45670A (fr) 2016-07-13 2019-05-22 Vertex Pharma Procédés, compositions et kits pour augmenter l'efficacité d'édition du génome
EP3995574A1 (de) 2016-08-24 2022-05-11 Sangamo Therapeutics, Inc. Regulierung der genexpression mit manipulierten nukleasen
KR102455249B1 (ko) 2016-08-24 2022-10-17 상가모 테라퓨틱스, 인코포레이티드 가공된 표적 특이적 뉴클레아제
KR102712926B1 (ko) 2016-10-20 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 방법 및 조성물
WO2018081775A1 (en) 2016-10-31 2018-05-03 Sangamo Therapeutics, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
IL266862B2 (en) 2016-12-01 2024-01-01 Sangamo Therapeutics Inc Tau modulators and methods and preparations for their administration
EP3551754B1 (de) 2016-12-08 2023-08-30 Case Western Reserve University Verfahren und zusammensetzungen zur erhöhung der herstellung von funktionalem myelin
ES2962711T3 (es) 2016-12-20 2024-03-20 Bristol Myers Squibb Co Métodos para aumentar la eficacia de la reparación dirigida por homología (HDR) en el genoma celular
EP3609519B1 (de) 2017-04-11 2025-11-26 University of Maryland, Baltimore Zusammensetzungen und verfahren zur behandlung von entzündungen und krebs
LT3615674T (lt) 2017-04-28 2024-09-25 The Regents Of The University Of Colorado, A Body Corporate Reumatoidinio artrito gydymo būdai, naudojant nukreipiančios rnr hla geno redagavimą genome
CN110799492B (zh) 2017-04-28 2023-06-27 爱康泰生治疗公司 用于递送核酸的新型羰基脂质和脂质纳米颗粒制剂
CN111344403B (zh) 2017-09-15 2025-05-06 利兰斯坦福初级大学董事会 遗传工程细胞的多元性产生和条形码编制
IL273759B2 (en) 2017-10-03 2024-08-01 Aptahem Ab A nucleic acid molecule with anti-inflammatory, anti-coagulant and organ-protecting properties
CN111566212A (zh) 2017-11-03 2020-08-21 因特尔纳技术有限公司 miRNA分子,等同物,安塔够妙或其来源用于治疗和/或诊断与神经元缺陷相关的病症和/或疾病或用于神经元生成和/或再生
BR112020013626A2 (pt) 2018-01-17 2020-12-01 Vertex Pharmaceuticals Incorporated compostos de quinoxalinona, composições, métodos e kits para aumentar a eficiência de edição de genoma
JP7466448B2 (ja) 2018-01-17 2024-04-12 バーテックス ファーマシューティカルズ インコーポレイテッド Dna-pk阻害剤
MA51619A (fr) 2018-01-17 2021-04-14 Vertex Pharma Inhibiteurs de la protéine kinase dépendante de l'adn
WO2019160866A2 (en) 2018-02-13 2019-08-22 Checkmate Pharmaceuticals, Inc. Compositions and methods for tumor immunotherapy
BR112020020479A2 (pt) * 2018-04-09 2021-01-12 Checkmate Pharmaceuticals Empacotamento de oligonucleotídeos em partículas similares a vírus
EP3790980A4 (de) 2018-05-06 2022-03-23 Emendobio Inc. Differentielles knockout eines allels eines heterozygoten elan-gens
KR20210060533A (ko) 2018-09-18 2021-05-26 상가모 테라퓨틱스, 인코포레이티드 프로그램화된 세포 사멸 1 (pd1) 특이적 뉴클레아제
IL281615B2 (en) 2018-09-21 2026-01-01 Acuitas Therapeutics Inc Systems and methods for producing lipid nanoparticles and liposomes
WO2020069029A1 (en) 2018-09-26 2020-04-02 Emendobio Inc. Novel crispr nucleases
WO2020072677A1 (en) 2018-10-02 2020-04-09 Sangamo Therapeutics, Inc. Methods and compositions for modulation of tau proteins
CA3116576A1 (en) 2018-10-18 2020-04-23 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
AU2019360270B2 (en) 2018-10-18 2025-08-07 Intellia Therapeutics, Inc. Compositions and methods for expressing factor IX.
WO2020082047A1 (en) 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiencey
US20200270617A1 (en) 2018-10-18 2020-08-27 Intellia Therapeutics, Inc. Compositions and methods for transgene expression from an albumin locus
CN113272428A (zh) 2018-10-18 2021-08-17 英特利亚治疗股份有限公司 核酸构建体和使用方法
WO2020112860A1 (en) 2018-11-30 2020-06-04 Intima Bioscience, Inc. Methods of identifying immunomodulatory genes
PT3908568T (pt) 2019-01-11 2024-09-30 Acuitas Therapeutics Inc Lípidos para a administração de agentes ativos por nanopartículas lipídicas
US20220098621A1 (en) 2019-02-05 2022-03-31 Emendobio Inc. Crispr compositions and methods for promoting gene editing of ribosomal protein s19 (rps19) gene
US20220154157A1 (en) 2019-02-06 2022-05-19 Emendobio Inc. New engineered high fidelity cas9
US11866469B2 (en) 2019-02-06 2024-01-09 Klogenix Llc DNA binding proteins and uses thereof
CA3132167A1 (en) 2019-04-02 2020-10-08 Weston P. MILLER IV Methods for the treatment of beta-thalassemia
US20220202957A1 (en) 2019-04-09 2022-06-30 The Regents Of The University Of California Long-lasting analgesia via targeted in vivo epigenetic repression
TW202521561A (zh) 2019-04-23 2025-06-01 美商聖加莫治療股份有限公司 染色體9開放讀框72基因表現之調節子及其用途
WO2020243485A1 (en) 2019-05-29 2020-12-03 Massachusetts Institute Of Technology Hiv-1 specific immunogen compositions and methods of use
JP7284473B2 (ja) * 2019-06-24 2023-05-31 学校法人東京理科大学 血液成分分離方法及びマイクロ流体デバイスの目詰まり防止剤
WO2021087361A1 (en) 2019-11-01 2021-05-06 Sangamo Therapeutics, Inc. Zinc finger nuclease variants for treating or preventing lysosomal storage diseases
CN115803435A (zh) 2020-05-06 2023-03-14 塞勒克提斯公司 用于在细胞基因组中靶向插入外源序列的方法
EP4146284A1 (de) 2020-05-06 2023-03-15 Cellectis S.A. Verfahren zur genetischen modifizierung von zellen zur freisetzung therapeutischer proteine
ES3054438T3 (en) 2020-07-16 2026-02-03 Acuitas Therapeutics Inc Cationic lipids for use in lipid nanoparticles
US12152251B2 (en) 2020-08-25 2024-11-26 Kite Pharma, Inc. T cells with improved functionality
AU2022277649A1 (en) 2021-05-21 2023-11-30 Cellectis S.A. Enhancing efficacy of t-cell-mediated immunotherapy by modulating cancer-associated fibroblasts in solid tumors
US20250011787A1 (en) 2021-10-21 2025-01-09 The J. David Gladstone Institutes, a testamentary trust established under the Will of J. David Glads Retroelement-generated transcription factor decoys
CA3237482A1 (en) 2021-11-03 2023-05-11 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Precise genome editing using retrons
CA3242402A1 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
EP4499822A1 (de) 2022-03-25 2025-02-05 The J. David Gladstone Institutes, A Testamentary Trust Established under The Will of J. David Gladstone Rt-dna-fidelity- und retron-genomeditierung
WO2023183627A1 (en) 2022-03-25 2023-09-28 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Production of reverse transcribed dna (rt-dna) using a retron reverse transcriptase from exogenous rna
EP4499839A1 (de) 2022-03-25 2025-02-05 The J. David Gladstone Institutes, A Testamentary Trust Established under The Will of J. David Gladstone Verfahren zur beurteilung der manipulierten retron-aktivität und verwendungen davon
US20250230429A1 (en) 2022-04-07 2025-07-17 The J. David Gladstone Institutes, a testamentary trust established under the Will of J. David Glads Continuous Multiplexed Phage Genome Engineering Using a Retron Editing Template
AU2023298102A1 (en) 2022-06-30 2024-11-14 Cellectis S.A. Enhancing safety of t-cell-mediated immunotherapy
US20250243516A1 (en) 2022-08-24 2025-07-31 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Dual Cut Retron Editors for Genomic Insertions and Deletions
JP2025536578A (ja) 2022-11-03 2025-11-07 セレクティス ソシエテ アノニム T細胞媒介免疫療法の有効性及び安全性の増進
CN121729237A (zh) 2023-06-30 2026-03-24 武田药品工业株式会社 Htt阻遏物及其用途
AU2024357861A1 (en) 2023-10-12 2026-04-23 Renagade Therapeutics Management Inc. Nickase-retron template-based precision editing system and methods of use

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5120657A (en) * 1986-12-05 1992-06-09 Agracetus, Inc. Apparatus for genetic transformation
US5166320A (en) * 1987-04-22 1992-11-24 University Of Connecticut Carrier system and method for the introduction of genes into mammalian cells
US5149782A (en) * 1988-08-19 1992-09-22 Tanox Biosystems, Inc. Molecular conjugates containing cell membrane-blending agents
US5354844A (en) * 1989-03-16 1994-10-11 Boehringer Ingelheim International Gmbh Protein-polycation conjugates
US5108921A (en) * 1989-04-03 1992-04-28 Purdue Research Foundation Method for enhanced transmembrane transport of exogenous molecules
US5122466A (en) * 1989-06-13 1992-06-16 North Carolina State University Ballistic transformation of conifers
AU1922092A (en) * 1991-05-03 1992-12-21 Albert Einstein College Of Medicine Of Yeshiva University Targeted delivery of genes encoding cell surface receptors
US5521291A (en) * 1991-09-30 1996-05-28 Boehringer Ingelheim International, Gmbh Conjugates for introducing nucleic acid into higher eucaryotic cells
JP4285766B2 (ja) * 1994-03-23 2009-06-24 オハイオ ユニバーシティ 緻密にした核酸および細胞へのデリバリ
US5844107A (en) * 1994-03-23 1998-12-01 Case Western Reserve University Compacted nucleic acids and their delivery to cells

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US5877302A (en) 1999-03-02
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