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 [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], 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)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • Plant Pathology (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Obesity (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (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 (164)

* 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
ATE410518T1 (de) * 1994-03-23 2008-10-15 Univ Ohio Kompaktnukleinsäure und ihre verabreichung in zellen
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
US6258792B1 (en) 1996-04-12 2001-07-10 University Of Pittsburgh Cationic cholesteryl derivatives containing cyclic polar groups
DK0904282T3 (da) 1996-04-12 2002-03-04 Univ Pittsburgh Hidtil ukendte kationiske cholesterylderivater indeholdende cykliske polære grupper
AU727447B2 (en) * 1996-07-03 2000-12-14 University Of Pittsburgh Emulsion formulations for hydrophilic active agents
CA2268417A1 (en) * 1996-10-09 1998-04-16 Oxford Biomedica (Uk) Limited Mononuclear phagocytes in therapeutic drug delivery
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
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
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
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
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
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
AU2868099A (en) 1998-02-13 1999-08-30 Selective Genetics, Inc. Concurrent flow mixing methods and apparatuses for the preparation of gene therapy vectors and compositions prepared thereby
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
CA2410265A1 (en) * 2000-05-31 2001-12-06 Copernicus Therapeutics, Inc. Lyophilizable and enhanced compacted nucleic acids
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
US7527802B2 (en) * 2001-02-13 2009-05-05 The United States Of America As Represented By The Secretary Of The Army Vaccine for transcutaneous immunization
WO2002075306A1 (en) * 2001-03-16 2002-09-26 Large Scale Biology Corporation 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
US20040014698A1 (en) * 2002-07-18 2004-01-22 Gonzalo Hortelano Oral administration of therapeutic agent coupled to transporting agent
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
US20040014704A1 (en) * 2002-07-18 2004-01-22 Gonzalo Hortelano Oral administration of therapeutic agent coupled to transporting agent induces tolerance
RU2377253C2 (ru) * 2002-12-02 2009-12-27 Амген Фремонт,Инк. Антитела, специфичные к фактору некроза опухолей, и их применение
JP2006517790A (ja) * 2003-01-09 2006-08-03 インヴィトロジェン コーポレーション ポリペプチド−核酸複合体の細胞の送達および活性化
US7824709B2 (en) 2003-02-14 2010-11-02 Children's Hospital And Research Center At Oakland Lipophilic drug delivery vehicle and methods of use thereof
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
EP2287303B1 (de) 2004-11-12 2014-07-02 Asuragen, Inc. Verfahren und Zusammensetzungen, die miRNAs und miRNA-inhibitorischen Molekülen verbunden sind
US20100272769A1 (en) * 2005-08-03 2010-10-28 Amcol International Virus-, Bacteria-, and Fungi-Interacting Layered Phyllosilicates and Methods of Use
US20080184618A1 (en) * 2005-08-03 2008-08-07 Amcol International Virus-Interacting Layered Phyllosilicates and Methods of Use
US20070031512A1 (en) * 2005-08-03 2007-02-08 Amcol International Corporation Virus-interacting layered phyllosilicates and methods of inactivating viruses
US7700541B2 (en) 2006-04-06 2010-04-20 Nitto Denko Corporation Biodegradable cationic polymers
PT2056882E (pt) 2006-08-01 2012-11-19 Univ Texas Identificação de um micro-rna que ativa a expressão da cadeia pesada de beta-miosina
JP2010512313A (ja) 2006-12-06 2010-04-22 メディミューン,エルエルシー 全身性エリテマトーデスの治療方法
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
AU2008218939B2 (en) * 2007-02-23 2014-05-08 The Research Foundation Of 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
EP2068924A4 (de) 2007-05-03 2011-07-20 Medimmune Llc Interferon-alpha-induzierte pharmakodynamische marker
KR101510778B1 (ko) 2007-07-12 2015-04-10 상가모 바이오사이언스 인코포레이티드 알파 1,6 당전이효소(fut8) 유전자 발현을 비활성화시키기 위한 방법 및 조성물
KR101706259B1 (ko) 2007-07-31 2017-02-14 보드 오브 리전츠, 더 유니버시티 오브 텍사스 시스템 섬유증을 조절하는 마이크로-rna 집단 및 이의 용도
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
US8051513B2 (en) * 2007-12-31 2011-11-08 Monster Medic, Inc. Ambulance cot system
CN102036689B (zh) 2008-03-17 2014-08-06 得克萨斯***大学董事会 神经肌肉突触维持和再生中涉及的微小rna的鉴定
CA2726768C (en) 2008-06-10 2016-09-06 Sangamo Biosciences, Inc. Methods and compositions for generation of bax- and bak-deficient cell lines
WO2009158678A1 (en) 2008-06-27 2009-12-30 Children's Hospital & Research Center At Oakland Lipophilic nucleic acid delivery vehicle and methods of use therefor
CA2734235C (en) 2008-08-22 2019-03-26 Sangamo Biosciences, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
EP3156494B8 (de) 2008-12-04 2018-09-19 Sangamo Therapeutics, Inc. Genomänderung bei ratten mithilfe von zink-finger-nukleasen
EP2379720B1 (de) 2009-01-20 2016-08-17 Alona Zilberberg Durch den promoter mir-21 angetriebene gezielte krebstherapie
CA2934285C (en) 2009-02-04 2018-11-27 Sangamo Biosciences, Inc. Methods and compositions for treating neuropathies
AU2010235161B2 (en) 2009-04-09 2015-01-22 Sangamo Therapeutics, Inc. Targeted integration into stem cells
NZ619527A (en) 2009-04-14 2015-06-26 Nestec Sa Inflammatory bowel disease prognostics
CA2769262C (en) 2009-07-28 2019-04-30 Sangamo Biosciences, Inc. Methods and compositions for treating trinucleotide repeat disorders
WO2011060098A1 (en) 2009-11-10 2011-05-19 Prometheus Laboratories Inc. Methods for predicting post-surgery risk associated with ileal pouch-anal anastomosis
EP2526112B1 (de) 2010-01-22 2018-10-17 Dow AgroSciences LLC Gezielte genommodifikation
EP2660318A1 (de) 2010-02-09 2013-11-06 Sangamo BioSciences, Inc. Gezielte Genommodifikation mit partiell einzelsträngigen Donatormolekülen
WO2011108930A1 (en) 2010-03-04 2011-09-09 Interna Technologies Bv A MiRNA MOLECULE DEFINED BY ITS SOURCE AND ITS DIAGNOSTIC AND THERAPEUTIC USES IN DISEASES OR CONDITIONS ASSOCIATED WITH EMT
CA2798988C (en) 2010-05-17 2020-03-10 Sangamo Biosciences, Inc. Tal-effector (tale) dna-binding polypeptides and uses thereof
WO2012005572A1 (en) 2010-07-06 2012-01-12 Interna Technologies Bv Mirna and its diagnostic and therapeutic uses in diseases or conditions associated with melanoma, or in diseases or conditions associated with activated braf pathway
CA2811364C (en) 2010-09-27 2022-01-04 Sangamo Biosciences, Inc. Methods and compositions for inhibiting viral entry into cells
MX2013005346A (es) * 2010-11-15 2013-07-03 Avon Prod Inc Cosmeticos biofuncionales anclados para uso prolongado.
EP2474617A1 (de) 2011-01-11 2012-07-11 InteRNA Technologies BV MIR zur Behandlung von Neoangiogenese
WO2013016446A2 (en) 2011-07-25 2013-01-31 Sangamo Biosciences, Inc. Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (cftr) gene
KR102061557B1 (ko) 2011-09-21 2020-01-03 상가모 테라퓨틱스, 인코포레이티드 이식 유전자 발현의 조절을 위한 방법 및 조성물
EP2769321B1 (de) 2011-10-21 2016-06-01 Nestec S.A. Verfahren zur verbesserung der diagnose einer entzündlichen darmerkrankung
ES2886147T3 (es) 2011-12-22 2021-12-16 Interna Tech B V MiARN para el tratamiento del cáncer de cabeza y de cuello
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
US10883119B2 (en) 2012-07-11 2021-01-05 Sangamo Therapeutics, Inc. Methods and compositions for delivery of biologics
EP3444342B1 (de) 2012-07-11 2020-05-27 Sangamo Therapeutics, Inc. Verfahren und zusammensetzungen zur behandlung von lysosomalen speicherkrankheiten
UA118957C2 (uk) 2012-08-29 2019-04-10 Сангамо Біосайєнсиз, Інк. Спосіб і композиція для лікування генетичного захворювання
CA2886684C (en) 2012-10-10 2023-09-19 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
WO2014072357A1 (en) 2012-11-06 2014-05-15 Interna Technologies B.V. Combination for use in treating diseases or conditions associated with melanoma, or treating diseases or conditions associated with activated b-raf pathway
EP2928303A4 (de) 2012-12-07 2016-07-13 Haplomics Inc Faktor-viii-mutationsreparatur und toleranzinduktion
CA2896070A1 (en) 2012-12-21 2014-06-26 The Trustees Of Columbia University In The City Of New York Biomarkers for chronic traumatic encephalopathy
US8980864B2 (en) * 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
WO2014151551A1 (en) 2013-03-15 2014-09-25 Baylor Research Institute Ulcerative colitis (uc)-associated colorectal neoplasia markers
US9873894B2 (en) 2013-05-15 2018-01-23 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
CA2917569A1 (en) 2013-07-11 2015-01-15 The Trustees Of Columbia University In The City Of New York Micrornas that silence tau expression
AU2014312295C1 (en) 2013-08-28 2020-08-13 Sangamo Therapeutics, Inc. Compositions for linking DNA-binding domains and cleavage domains
EA201690675A1 (ru) 2013-10-03 2016-08-31 Модерна Терапьютикс, Инк. Полинуклеотиды, кодирующие рецептор липопротеинов низкой плотности
DK3441468T3 (da) 2013-10-17 2021-07-26 Sangamo Therapeutics Inc Afgivelsesfremgangsmåder og sammensætninger til nukleasemedieret genom-manipulation
CN105934524A (zh) 2013-11-11 2016-09-07 桑格摩生物科学股份有限公司 用于治疗亨廷顿氏病的方法和组合物
EP3492593B1 (de) 2013-11-13 2021-08-18 Children's Medical Center Corporation Nukleasevermittelte regulierung der genexpression
EP3068783B1 (de) 2013-11-15 2020-09-23 The Board of Trustees of the Leland Stanford Junior University Agonisten des hypokretin-rezeptors 2 zur verwendung zur behandlung von herzversagen
WO2015074010A2 (en) 2013-11-18 2015-05-21 Beth Israel Deaconess Medical Center, Inc. Compositions and methods for cardiac regeneration
CA2931637C (en) 2013-12-09 2023-10-10 Sangamo Biosciences, Inc. Methods and compositions for treating hemophilia
HUE051232T2 (hu) 2014-02-03 2021-03-01 Sangamo Therapeutics Inc Béta-talasszémia kezelésére szolgáló eljárások és készítmények
CA2942762C (en) 2014-03-18 2023-10-17 Sangamo Biosciences, Inc. Methods and compositions for regulation of zinc finger protein expression
RU2691102C2 (ru) 2014-05-08 2019-06-11 Сангамо Байосайенсиз, Инк. Способы и композиции для лечения болезни хантингтона
HUE055583T2 (hu) 2014-09-16 2021-12-28 Sangamo Therapeutics Inc Eljárások és készítmények nukleáz által közvetített genommódosításhoz és -javításhoz hematopoetikus õssejtekben
US20180094243A1 (en) 2015-04-03 2018-04-05 Dana-Farber Cancer Institute, Inc. Composition and methods of genome editing of b-cells
US10450585B2 (en) 2015-07-13 2019-10-22 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
JP7409773B2 (ja) 2015-07-31 2024-01-09 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 改変された細胞および治療の方法
US10435441B2 (en) 2015-09-23 2019-10-08 Sangamo Therapeutics, Inc. HTT repressors and uses thereof
AU2016369490C1 (en) 2015-12-18 2021-12-23 Sangamo Therapeutics, Inc. Targeted disruption of the T cell receptor
IL297018A (en) 2015-12-18 2022-12-01 Sangamo Therapeutics Inc Directed cleavage of cell mhc receptor
CA3010738A1 (en) 2016-01-15 2017-07-20 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
AU2016391970B2 (en) 2016-02-02 2024-02-22 Sangamo Therapeutics, Inc. Compositions for linking DNA-binding domains and cleavage domains
JP7033583B2 (ja) 2016-07-13 2022-03-10 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 ゲノム編集効率を高めるための方法、組成物及びキット
SG10201913948PA (en) 2016-08-24 2020-03-30 Sangamo Therapeutics Inc Engineered target specific nucleases
MY193234A (en) 2016-08-24 2022-09-27 Sangamo Therapeutics Inc Regulation of gene expression using engineered nucleases
EP3528852A4 (de) 2016-10-20 2020-06-03 Sangamo Therapeutics, Inc. Verfahren und zusammensetzungen zur behandlung von morbus fabry
WO2018081775A1 (en) 2016-10-31 2018-05-03 Sangamo Therapeutics, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
WO2018102665A1 (en) 2016-12-01 2018-06-07 Sangamo Therapeutics, Inc. Tau modulators and methods and compositions for delivery thereof
EP4276187A3 (de) 2016-12-08 2024-01-17 Case Western Reserve University Verfahren und zusammensetzungen zur erhöhung der herstellung von funktionalem myelin
WO2018119060A1 (en) 2016-12-20 2018-06-28 Bristol-Myers Squibb Company Methods for increasing the efficiency of homology directed repair (hdr) in the cellular genome
AU2018256877B2 (en) 2017-04-28 2022-06-02 Acuitas Therapeutics, Inc. Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
JP7025537B2 (ja) 2017-10-03 2022-02-24 アプタヘム アクチエボラグ 抗炎症および抗凝固および器官保護特性を有する核酸分子
EP3704250A1 (de) 2017-11-03 2020-09-09 InteRNA Technologies B.V. Mirna-molekül, äquivalent, antagomir oder quelle davon zur behandlung und/oder diagnose eines zustands und/oder einer erkrankung im zusammenhang mit neuronaler defizienz oder neuronaler (re)generation
IL276082B2 (en) 2018-01-17 2024-01-01 Vertex Pharma DNA-PK inhibitor compounds, preparations containing them and their uses
AU2019209293B2 (en) 2018-01-17 2023-07-27 Vertex Pharmaceuticals Incorporated DNA-PK inhibitors
US20200361877A1 (en) 2018-01-17 2020-11-19 Vertex Pharmaceuticals Incorporated Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency
JP2021525058A (ja) * 2018-04-09 2021-09-24 チェックメイト ファーマシューティカルズ ウイルス様粒子中へのオリゴヌクレオチドのパッケージ化
CN113015741A (zh) 2018-09-18 2021-06-22 桑格摩生物治疗股份有限公司 程序性细胞死亡1(pd1)特异性核酸酶
WO2020069029A1 (en) 2018-09-26 2020-04-02 Emendobio Inc. Novel crispr nucleases
US20200270618A1 (en) 2018-10-18 2020-08-27 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
AU2019360270A1 (en) 2018-10-18 2021-05-27 Intellia Therapeutics, Inc. Compositions and methods for expressing factor IX.
US20200268906A1 (en) 2018-10-18 2020-08-27 Intellia Therapeutics, Inc. Nucleic acid constructs and methods of use
CA3116918A1 (en) 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for transgene expression from an albumin locus
CN113748126A (zh) 2018-11-30 2021-12-03 因提玛生物科学公司 鉴定免疫调节基因的方法
CN113474328A (zh) 2019-01-11 2021-10-01 爱康泰生治疗公司 用于脂质纳米颗粒递送活性剂的脂质
WO2020163379A1 (en) 2019-02-05 2020-08-13 Emendobio Inc. Crispr compositions and methods for promoting gene editing of ribosomal protein s19 (rps19) gene
US11866469B2 (en) 2019-02-06 2024-01-09 Klogenix Llc DNA binding proteins and uses thereof
US20220154157A1 (en) 2019-02-06 2022-05-19 Emendobio Inc. New engineered high fidelity cas9
US20200316116A1 (en) 2019-04-02 2020-10-08 Sangamo Therapeutics, Inc. Method 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
US20210317192A9 (en) 2019-05-29 2021-10-14 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
AU2020376048A1 (en) 2019-11-01 2022-06-02 Sangamo Therapeutics, Inc. Compositions and methods for genome engineering
CN115715203A (zh) 2020-05-06 2023-02-24 塞勒克提斯公司 对细胞进行基因修饰以递送治疗性蛋白质的方法
CA3177093A1 (en) 2020-05-06 2021-11-11 Cellectis S.A. Methods for targeted insertion of exogenous sequences in cellular genomes
EP4182297A1 (de) 2020-07-16 2023-05-24 Acuitas Therapeutics, Inc. Kationische lipide zur verwendung in lipidnanopartikeln
WO2022046760A2 (en) 2020-08-25 2022-03-03 Kite Pharma, Inc. T cells with improved functionality
EP4341300A1 (de) 2021-05-21 2024-03-27 Cellectis S.A. Erhöhung der wirksamkeit von t-zell-vermittelter immuntherapie durch modulation von krebsassoziierten fibroblasten in soliden tumoren
WO2023070072A1 (en) 2021-10-21 2023-04-27 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Retroelement-generated transcription factor decoys
WO2023081756A1 (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
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
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
WO2023183589A1 (en) 2022-03-25 2023-09-28 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Rt-dna fidelity and retron genome editing
WO2023183588A1 (en) 2022-03-25 2023-09-28 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Methods of assessing engineered retron activity, and uses thereof
WO2023196725A1 (en) 2022-04-07 2023-10-12 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Continuous multiplexed phage genome engineering using a retron editing template
WO2024003334A1 (en) 2022-06-30 2024-01-04 Cellectis S.A. Enhancing safety of t-cell-mediated immunotherapy
WO2024044673A1 (en) 2022-08-24 2024-02-29 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Dual cut retron editors for genomic insertions and deletions
WO2024094775A1 (en) 2022-11-03 2024-05-10 Cellectis S.A. Enhancing efficacy and safety of t-cell-mediated immunotherapy

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
US5844107A (en) * 1994-03-23 1998-12-01 Case Western Reserve University Compacted nucleic acids and their delivery to cells
ATE410518T1 (de) * 1994-03-23 2008-10-15 Univ Ohio Kompaktnukleinsäure und ihre verabreichung in zellen

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