ATE217904T1 - Nukleozyme - Google Patents

Nukleozyme

Info

Publication number
ATE217904T1
ATE217904T1 AT93904752T AT93904752T ATE217904T1 AT E217904 T1 ATE217904 T1 AT E217904T1 AT 93904752 T AT93904752 T AT 93904752T AT 93904752 T AT93904752 T AT 93904752T AT E217904 T1 ATE217904 T1 AT E217904T1
Authority
AT
Austria
Prior art keywords
nucleozymes
degradation
resistant
nucleic acid
further comprise
Prior art date
Application number
AT93904752T
Other languages
English (en)
Inventor
Nassim Usman
Robert J Cedergren
Jean-Pierre Perreault
Jing-Hua Yang
Alexander Rich
Original Assignee
Massachusetts Inst Technology
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=25255389&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=ATE217904(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Massachusetts Inst Technology filed Critical Massachusetts Inst Technology
Application granted granted Critical
Publication of ATE217904T1 publication Critical patent/ATE217904T1/de

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Oncology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Communicable Diseases (AREA)
  • AIDS & HIV (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Compositions Of Macromolecular Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
AT93904752T 1992-01-31 1993-01-28 Nukleozyme ATE217904T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US07/829,729 US5652094A (en) 1992-01-31 1992-01-31 Nucleozymes
PCT/US1993/000833 WO1993015187A1 (en) 1992-01-31 1993-01-28 Nucleozymes

Publications (1)

Publication Number Publication Date
ATE217904T1 true ATE217904T1 (de) 2002-06-15

Family

ID=25255389

Family Applications (1)

Application Number Title Priority Date Filing Date
AT93904752T ATE217904T1 (de) 1992-01-31 1993-01-28 Nukleozyme

Country Status (9)

Country Link
US (4) US5652094A (de)
EP (3) EP0625192B1 (de)
JP (1) JPH07503613A (de)
AT (1) ATE217904T1 (de)
AU (1) AU680405B2 (de)
CA (1) CA2129119A1 (de)
DE (1) DE69331946T2 (de)
MX (1) MX9300499A (de)
WO (1) WO1993015187A1 (de)

Families Citing this family (307)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA1223831A (en) 1982-06-23 1987-07-07 Dean Engelhardt Modified nucleotides, methods of preparing and utilizing and compositions containing the same
US5646042A (en) * 1992-08-26 1997-07-08 Ribozyme Pharmaceuticals, Inc. C-myb targeted ribozymes
US5891684A (en) * 1992-10-15 1999-04-06 Ribozyme Pharmaceuticals, Inc. Base-modified enzymatic nucleic acid
US5658780A (en) * 1992-12-07 1997-08-19 Ribozyme Pharmaceuticals, Inc. Rel a targeted ribozymes
US5612215A (en) * 1992-12-07 1997-03-18 Ribozyme Pharmaceuticals, Inc. Stromelysin targeted ribozymes
US5811300A (en) * 1992-12-07 1998-09-22 Ribozyme Pharmaceuticals, Inc. TNF-α ribozymes
US5616488A (en) * 1992-12-07 1997-04-01 Ribozyme Pharmaceuticals, Inc. IL-5 targeted ribozymes
US5837542A (en) * 1992-12-07 1998-11-17 Ribozyme Pharmaceuticals, Inc. Intercellular adhesion molecule-1 (ICAM-1) ribozymes
EP1253199A1 (de) 1993-09-02 2002-10-30 Ribozyme Pharmaceuticals, Inc. Enzymatische Nukleinsaüre die abasische Gruppen enthalten
WO1995006764A2 (en) * 1993-09-03 1995-03-09 Vpi Holdings Ltd. Oligonucleotides with rna cleavage activity
US5861288A (en) * 1993-10-18 1999-01-19 Ribozyme Pharmaceuticals, Inc. Catalytic DNA
ATE174600T1 (de) * 1993-10-27 1999-01-15 Ribozyme Pharm Inc 2'-amido-und 2'-peptido-modifizierte oligonukleotide
AU687479B2 (en) * 1993-11-08 1998-02-26 Ribozyme Pharmaceuticals, Inc. Base-modified enzymatic nucleic acid
US5631359A (en) * 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
US5639647A (en) 1994-03-29 1997-06-17 Ribozyme Pharmaceuticals, Inc. 2'-deoxy-2'alkylnucleotide containing nucleic acid
US5693532A (en) * 1994-11-04 1997-12-02 Ribozyme Pharmaceuticals, Inc. Respiratory syncytial virus ribozymes
US5670347A (en) 1994-05-11 1997-09-23 Amba Biosciences Llc Peptide-mediated gene transfer
US6103890A (en) * 1994-05-18 2000-08-15 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids that cleave C-fos
US5633133A (en) * 1994-07-14 1997-05-27 Long; David M. Ligation with hammerhead ribozymes
US5599706A (en) * 1994-09-23 1997-02-04 Stinchcomb; Dan T. Ribozymes targeted to apo(a) mRNA
US5700923A (en) * 1994-09-29 1997-12-23 Hybridon, Inc. Finderons and methods of their preparation and use
US5650502A (en) * 1994-11-09 1997-07-22 Hybridon, Inc. Ribozyme analogs having rigid non-nucleotidic linkers
US5716824A (en) 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
US5672501A (en) * 1994-12-23 1997-09-30 Ribozyme Pharmaceuticals, Inc. Base-modified enzymatic nucleic acid
US5545729A (en) * 1994-12-22 1996-08-13 Hybridon, Inc. Stabilized ribozyme analogs
US5705388A (en) * 1994-12-23 1998-01-06 Ribozyme Pharmaceuticals, Inc. CETP Ribozymes
US5663064A (en) * 1995-01-13 1997-09-02 University Of Vermont Ribozymes with RNA protein binding site
US20030207837A1 (en) * 1995-07-07 2003-11-06 Ribozyme Pharmaceuticals, Inc. Method and reagent for the induction of graft tolerance and reversal of immune responses
US5877021A (en) * 1995-07-07 1999-03-02 Ribozyme Pharmaceuticals, Inc. B7-1 targeted ribozymes
US7034009B2 (en) 1995-10-26 2006-04-25 Sirna Therapeutics, Inc. Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
US6346398B1 (en) 1995-10-26 2002-02-12 Ribozyme Pharmaceuticals, Inc. Method and reagent for the treatment of diseases or conditions related to levels of vascular endothelial growth factor receptor
ATE213019T1 (de) * 1995-11-14 2002-02-15 Vimrx Holdings Ltd Chimäre oligomere mit einer rns- spaltungsaktivität
US5998203A (en) * 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6274369B1 (en) * 1996-02-02 2001-08-14 Invitrogen Corporation Method capable of increasing competency of bacterial cell transformation
US5807743A (en) * 1996-12-03 1998-09-15 Ribozyme Pharmaceuticals, Inc. Interleukin-2 receptor gamma-chain ribozymes
US6248878B1 (en) 1996-12-24 2001-06-19 Ribozyme Pharmaceuticals, Inc. Nucleoside analogs
IL131016A (en) * 1997-01-21 2004-12-15 Gen Hospital Corp Selection of proteins using RNA-protein blends
US6057156A (en) 1997-01-31 2000-05-02 Robozyme Pharmaceuticals, Inc. Enzymatic nucleic acid treatment of diseases or conditions related to levels of epidermal growth factor receptors
US6159951A (en) 1997-02-13 2000-12-12 Ribozyme Pharmaceuticals Inc. 2'-O-amino-containing nucleoside analogs and polynucleotides
US6251666B1 (en) 1997-03-31 2001-06-26 Ribozyme Pharmaceuticals, Inc. Nucleic acid catalysts comprising L-nucleotide analogs
US5942395A (en) * 1997-05-09 1999-08-24 Universite De Montreal Hybrid ribozymes and methods of use
US6280936B1 (en) 1997-06-09 2001-08-28 Ribozyme Pharmaceuticals, Inc. Method for screening nucleic acid catalysts
US6548657B1 (en) 1997-06-09 2003-04-15 Ribozyme Pharmaceuticals, Inc. Method for screening nucleic acid catalysts
US6183959B1 (en) 1997-07-03 2001-02-06 Ribozyme Pharmaceuticals, Inc. Method for target site selection and discovery
US6316612B1 (en) 1997-08-22 2001-11-13 Ribozyme Pharmaceuticals, Inc. Xylofuranosly-containing nucleoside phosphoramidites and polynucleotides
US6656731B1 (en) 1997-09-22 2003-12-02 Max Planck Gesellschaft Zur Forderung Der Wissenschaften E.V. Nucleic acid catalysts with endonuclease activity
CA2304813A1 (en) * 1997-09-22 1999-04-08 Fritz Eckstein Nucleic acid catalysts with endonuclease activity
US6054576A (en) * 1997-10-02 2000-04-25 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA
US6127535A (en) * 1997-11-05 2000-10-03 Ribozyme Pharmaceuticals, Inc. Nucleoside triphosphates and their incorporation into oligonucleotides
US6617438B1 (en) 1997-11-05 2003-09-09 Sirna Therapeutics, Inc. Oligoribonucleotides with enzymatic activity
US20020147143A1 (en) 1998-03-18 2002-10-10 Corixa Corporation Compositions and methods for the therapy and diagnosis of lung cancer
US6307041B1 (en) 1998-03-28 2001-10-23 University Of Utah Research Foundation Circular, hairpin, circular/hairpin, lariat, and hairpin-lariat hammerhead ribozymes
CA2326823A1 (en) 1998-04-20 1999-10-28 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
US6995259B1 (en) 1998-10-23 2006-02-07 Sirna Therapeutics, Inc. Method for the chemical synthesis of oligonucleotides
JP2002542805A (ja) 1999-04-30 2002-12-17 ユニバーシティ オブ フロリダ アデノ随伴ウイルス送達リボザイム組成物および使用方法
US6831171B2 (en) 2000-02-08 2004-12-14 Yale University Nucleic acid catalysts with endonuclease activity
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US8273866B2 (en) 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
US20040002068A1 (en) 2000-03-01 2004-01-01 Corixa Corporation Compositions and methods for the detection, diagnosis and therapy of hematological malignancies
EP1319069B1 (de) 2000-06-28 2008-05-21 Corixa Corporation Zusammensetzungen und verfahren für therapie und diagnose von lungenkrebs
US8568766B2 (en) 2000-08-24 2013-10-29 Gattadahalli M. Anantharamaiah Peptides and peptide mimetics to treat pathologies associated with eye disease
US7125660B2 (en) * 2000-09-13 2006-10-24 Archemix Corp. Nucleic acid sensor molecules and methods of using same
CA2418724A1 (en) * 2000-09-13 2002-03-21 Archemix Corporation Target activated nucleic acid biosensor and methods of using same
JP5305553B2 (ja) 2000-10-12 2013-10-02 ユニバーシティー オブ ロチェスター 癌細胞の増殖を阻害する組成物
AU2002257067A1 (en) 2001-03-14 2002-09-24 Myriad Genetics, Inc Tsg101-gag interaction and use thereof
EP1386004A4 (de) 2001-04-05 2005-02-16 Ribozyme Pharm Inc Modulation der mit der entzündungsausbreitung und dem neuritenauswuchs assoziierten genexpression unter verwendung von technologien auf nukleinsäurebasis
CA2446788A1 (en) 2001-05-09 2002-11-14 Corixa Corporation Compositions and methods for the therapy and diagnosis of prostate cancer
US20050148530A1 (en) 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US7517864B2 (en) 2001-05-18 2009-04-14 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
EP3231445A1 (de) 2001-05-18 2017-10-18 Sirna Therapeutics, Inc. Konjugate und zusammensetzungen zur zellulären verabreichung
US7205399B1 (en) 2001-07-06 2007-04-17 Sirna Therapeutics, Inc. Methods and reagents for oligonucleotide synthesis
EP1581119B1 (de) 2001-12-17 2013-01-30 Corixa Corporation Zusammensetzungen und verfahren zur behandlung und diagnose von entzündlicher darmerkrankung
EP1572168B1 (de) 2002-02-06 2010-05-26 Vicor Technologies, Inc. Anti-infarkt-moleküle
US20050042632A1 (en) * 2002-02-13 2005-02-24 Sirna Therapeutics, Inc. Antibodies having specificity for nucleic acids
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
EP1432724A4 (de) 2002-02-20 2006-02-01 Sirna Therapeutics Inc Durch rna-interferenz vermittelte inhibierung von map-kinase-genen
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
EP1534729A2 (de) * 2002-02-26 2005-06-01 University of Utah Research Foundation Mit bluthochdruck und virusknospung assoziierte nedd4l-varianten
US20030195164A1 (en) * 2002-04-16 2003-10-16 Veli-Matti Kahari Novel ribozyme and its use
US7655790B2 (en) 2002-07-12 2010-02-02 Sirna Therapeutics, Inc. Deprotection and purification of oligonucleotides and their derivatives
US6989442B2 (en) 2002-07-12 2006-01-24 Sirna Therapeutics, Inc. Deprotection and purification of oligonucleotides and their derivatives
US20050287648A1 (en) 2002-08-05 2005-12-29 University Of Rochester Protein Transducing Domain/Deaminase Chimeric Proteins, Related Compounds, and Uses Thereof
US7923547B2 (en) 2002-09-05 2011-04-12 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US20080260744A1 (en) 2002-09-09 2008-10-23 Omeros Corporation G protein coupled receptors and uses thereof
CA2507863A1 (en) * 2002-12-04 2004-06-17 Algos Therapeutics, Inc. Methods and materials for modulating trpm2
US20040209353A1 (en) * 2002-12-12 2004-10-21 Chiron Corporation Biological sample storage device and method for biological sample contamination testing
EP3222294A1 (de) 2003-04-30 2017-09-27 Sirna Therapeutics, Inc. Konjugate und zusammensetzungen zur zellulären verabreichung
JP2005017116A (ja) * 2003-06-26 2005-01-20 Sharp Corp 光学式エンコーダ用受光素子
WO2005078848A2 (en) 2004-02-11 2005-08-25 University Of Tennessee Research Foundation Inhibition of tumor growth and invasion by anti-matrix metalloproteinase dnazymes
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
CA2557532A1 (en) 2004-04-23 2005-11-10 Angela M. Christiano Inhibition of hairless protein mrna
US20060040882A1 (en) 2004-05-04 2006-02-23 Lishan Chen Compostions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
US10508277B2 (en) 2004-05-24 2019-12-17 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
WO2006020684A2 (en) 2004-08-10 2006-02-23 Institute For Multiple Myeloma And Bone Cancer Research Methods of regulating differentiation and treating of multiple myeloma
US7568565B2 (en) * 2004-08-17 2009-08-04 Nes Technologies, Inc Device, a system and a method for transferring vibrational energy
US8101745B2 (en) 2004-12-16 2012-01-24 The Regents Of The University Of California Lung-targeted drugs
BRPI0606791A2 (pt) 2005-02-02 2010-02-09 The Uab Research Foundation métodos de reverter ou prevenir a resistência de uma célula a um agonista do receptor da morte, de triar uma célula quanto a um biomarcador da resistência a um agonista do receptor da morte, de monitorar a resistência a um agonista do receptor da morte em um indivìduo, de induzir seletivamente a apoptose em uma célula alvo expressando um receptor da morte, de tratar de um indivìduo com cáncer, de tratar de um indivìduo com uma doença inflamatória ou autoimune, de bloquear a ligação da proteìna contendo card a um receptor da morte em uma célula, de bloquear a associação de um iap com o receptor da morte e de triar um modulador de uma proteìna contendo card, composição, ácido nucleico isolado, vetor, célula, e, polipeptìdeo isolado
EP2551282A3 (de) 2005-03-23 2013-02-13 Genmab A/S Antikörper gegen CD38 zur Behandling von multiplem Myelom
US7476733B2 (en) 2005-03-25 2009-01-13 The United States Of America As Represented By The Department Of Health And Human Services Development of a real-time PCR assay for detection of pneumococcal DNA and diagnosis of pneumococccal disease
PL1866414T3 (pl) 2005-03-31 2012-10-31 Calando Pharmaceuticals Inc Inhibitory podjednostki 2 reduktazy rybonukleotydowej i ich zastosowania
ATE522626T1 (de) 2005-06-28 2011-09-15 Medtronic Inc Verfahren und nukleotid-sequenzen, die bevorzugt die expression eines mutierten huntingtin-genes unterdrücken
AU2006302245A1 (en) * 2005-10-06 2007-04-19 Emthrax, Llc Methods and compositions relating to anthrax spore glycoproteins as vaccines
US8080534B2 (en) 2005-10-14 2011-12-20 Phigenix, Inc Targeting PAX2 for the treatment of breast cancer
EP2395076A1 (de) 2005-10-14 2011-12-14 MUSC Foundation For Research Development Targeting von PAX2 zur Induktion der DEFB1-vermittelten Tumorimmunität und Krebstherapie
EP2526968A3 (de) 2006-01-27 2013-05-22 Biogen Idec MA Inc. Nogo-Rezeptorantagonisten
JP5704741B2 (ja) 2006-03-31 2015-04-22 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Ｅｇ５遺伝子発現の抑制のための組成物および方法
US8524454B2 (en) 2006-04-07 2013-09-03 The Research Foundation Of State University Of New York Transcobalamin receptor polypeptides, nucleic acids, and modulators thereof, and related methods of use in modulating cell growth and treating cancer and cobalamin deficiency
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
WO2008054561A2 (en) 2006-07-11 2008-05-08 University Of Medicine And Dentistry Of New Jersey Proteins, nucleic acids encoding the same and associated methods of use
EP2066812A4 (de) 2006-09-21 2010-04-28 Univ Rochester Zusammensetzungen und verfahren in zusammenhang mit proteinverschiebungstherapie für myotone dystrophie
CN105061580B (zh) 2006-10-03 2020-06-05 罗格斯新泽西州立大学 多肽和药物组合物及其应用
US20100166743A1 (en) 2006-10-06 2010-07-01 University Of Utah Research Foundation Method of detecting ocular diseases and pathologic conditions and treatment of same
WO2008052774A2 (en) 2006-10-31 2008-05-08 Noxxon Pharma Ag Methods for detection of a single- or double-stranded nucleic acid molecule
WO2008136852A2 (en) 2006-11-01 2008-11-13 University Of Rochester Methods and compositions related to the structure and function of apobec3g
JP2010512327A (ja) 2006-12-11 2010-04-22 ユニヴァーシティー オブ ユタ リサーチ ファウンデーション 病的血管形成および脈管透過性の処置用の組成物および方法
US20100129358A1 (en) 2006-12-22 2010-05-27 University Of Utah Research Foundation Method of detecting ocular diseases and pathologic conditions and treatment of same
EP3434259A1 (de) 2007-05-04 2019-01-30 Marina Biotech, Inc. Aminosäurelipide und verwendungen davon
AU2008296487A1 (en) 2007-08-28 2009-03-12 The Uab Research Foundation Synthetic apolipoprotein E mimicking polypeptides and methods of use
JP2010538005A (ja) 2007-08-28 2010-12-09 ユーエービー リサーチ ファウンデーション 合成アポリポ蛋白質ｅ模倣ポリペプチドおよび使用方法
WO2009033027A2 (en) 2007-09-05 2009-03-12 Medtronic, Inc. Suppression of scn9a gene expression and/or function for the treatment of pain
WO2010070380A2 (en) * 2007-12-03 2010-06-24 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health Of Human Services, National Institutes Of Health Doc1 compositions and methods for treating cancer
EP2229186A2 (de) 2007-12-04 2010-09-22 Alnylam Pharmaceuticals Inc. Kohlenhydrat-konjugate als mittel zur abgabe von oligonukleotiden
EP3100718B1 (de) 2008-01-02 2019-11-27 Arbutus Biopharma Corporation Verbesserte zusammensetzungen und verfahren zur freisetzung von nukleinsäuren
US20090233993A1 (en) * 2008-03-06 2009-09-17 Burnham Institute For Medical Research Compositions and methods for inhibiting gsk3 activity and uses thereof
JP2011516065A (ja) 2008-04-04 2011-05-26 カランド ファーマシューティカルズ， インコーポレイテッド Ｅｐａｓ１阻害剤の組成物および使用
EP2279254B1 (de) 2008-04-15 2017-07-05 Protiva Biotherapeutics Inc. Neue lipidformulierungen zur nukleinsäurezuführung
US20090274696A1 (en) * 2008-04-29 2009-11-05 Wyeth Methods for treating inflammation
US20110262395A1 (en) 2008-05-08 2011-10-27 University Of Utah Research Foundation Sensory receptors for chronic fatigue and pain and uses thereof
US20110212021A1 (en) 2008-05-30 2011-09-01 Slack Frank J Targeted oligonucleotide compositions for modifying gene expression
EP2323679A4 (de) 2008-07-25 2012-08-22 Univ Colorado Clip-hemmer und verfahren zur modulierung der immunfunktion
EP2341924A4 (de) 2008-10-02 2013-01-23 David Gladstone Inst Verfahren zur behandlung von hepatitis-c-virusinfektionen
AU2009303345B2 (en) 2008-10-09 2015-08-20 Arbutus Biopharma Corporation Improved amino lipids and methods for the delivery of nucleic acids
CN104382853A (zh) 2008-10-16 2015-03-04 玛瑞纳生物技术有限公司 基因沉默治疗剂的脂质体有效递送方法和组合物
ES2732504T3 (es) 2008-10-29 2019-11-22 Circular Commitment Company Procedimientos y agentes para el diagnóstico y tratamiento del carcinoma hepatocelular
EP3699172A3 (de) 2008-11-10 2020-11-18 Arbutus Biopharma Corporation Neuartige lipide und zusammensetzungen zur verabreichung von therapeutika
US20120070443A1 (en) 2008-12-02 2012-03-22 University Of Utah Research Foundation Pde1 as a target therapeutic in heart disease
EP2391343B1 (de) 2009-01-29 2017-03-01 Arbutus Biopharma Corporation Verbesserte lipidformulierung zur verabreichung von nukleinsaueren
US20100215660A1 (en) 2009-02-23 2010-08-26 Sarwar Hashmi Kruppel-like factors and fat regulation
JP6010458B2 (ja) 2009-04-03 2016-10-19 ダイセルナ ファーマシューティカルズ， インコーポレイテッドＤｉｃｅｒｎａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 非対称二本鎖ｒｎａによる特異的阻害のための、方法および組成物
EP2756845B1 (de) 2009-04-03 2017-03-15 Dicerna Pharmaceuticals, Inc. Verfahren und Zusammensetzungen für spezifische KRAS-Hemmung mit asymmetrischer Doppelstrang-RNA
CA2758189C (en) 2009-04-10 2020-12-29 Association Institut De Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
CN107252489A (zh) 2009-04-13 2017-10-17 法国健康和医学研究院 Hpv颗粒及其用途
CA2760706C (en) 2009-05-05 2019-08-06 Alnylam Pharmaceuticals, Inc. Methods of delivering oligonucleotides to immune cells
CA3042927C (en) 2009-05-05 2022-05-17 Arbutus Biopharma Corporation Lipid compositions for the delivery of therapeutic agents
CA2762095A1 (en) 2009-05-16 2010-11-25 Kunyuan Cui Compositions comprising cationic amphiphiles and colipids for delivering therapeutic molecules
KR20220038506A (ko) 2009-06-10 2022-03-28 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
ES2613498T3 (es) 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
WO2011031974A1 (en) 2009-09-10 2011-03-17 Southern Research Institute Acridine analogs in the treatment of gliomas
WO2011039646A2 (en) 2009-09-30 2011-04-07 Inserm (Institut National De La Sante Et De La Recherche Medicale) Papilloma virus -like particles for targeted gene delivery
US8450090B2 (en) 2009-10-06 2013-05-28 The Regents Of The University Of Colorado, A Body Corporate Compositions and methods for promoting fatty acid production in plants
US20120270930A1 (en) 2009-10-29 2012-10-25 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Methods and compositions for dysferlin exon-skipping
CA3044884A1 (en) 2009-12-07 2011-06-16 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
US20130017223A1 (en) 2009-12-18 2013-01-17 The University Of British Columbia Methods and compositions for delivery of nucleic acids
MX2012009318A (es) 2010-02-10 2012-09-07 Novartis Ag Metodos y compuestos para el crecimiento muscular.
ES2647390T3 (es) 2010-02-11 2017-12-21 Nanostring Technologies, Inc Composiciones y métodos para la detección preferencial de ARN pequeños mediante hibridación con puente y ligación
US20110207789A1 (en) 2010-02-19 2011-08-25 Ye Fang Methods related to casein kinase ii (ck2) inhibitors and the use of purinosome-disrupting ck2 inhibitors for anti-cancer therapy agents
EP2550361B1 (de) 2010-03-25 2017-02-08 The J. David Gladstone Institutes Zusammensetzungen und verfahren zur behandlung neurologischer störungen
WO2011120023A1 (en) 2010-03-26 2011-09-29 Marina Biotech, Inc. Nucleic acid compounds for inhibiting survivin gene expression uses thereof
WO2011133584A2 (en) 2010-04-19 2011-10-27 Marina Biotech, Inc. Nucleic acid compounds for inhibiting hras gene expression and uses thereof
HUE041571T2 (hu) 2010-04-23 2019-05-28 Univ Florida RAAV-guanilát-cikláz-készítmények és ejárások 1-es típusú Leber-féle veleszületett vakság (LCA1) kezelésére
US20130190383A1 (en) 2010-04-26 2013-07-25 Marina Biotech, Inc. Nucleic acid compounds with conformationally restricted monomers and uses thereof
WO2011139842A2 (en) 2010-04-28 2011-11-10 Marina Biotech, Inc. Nucleic acid compounds for inhibiting fgfr3 gene expression and uses thereof
DK2591105T3 (en) 2010-07-06 2017-07-31 Dicerna Pharmaceuticals Inc METHODS AND COMPOSITIONS FOR SPECIFIC INHIBITION OF BETA-CATENIN USING DOUBLE-STRENGTH RNA
US10064885B2 (en) 2010-07-09 2018-09-04 Massachusetts Institute Of Technology Metabolic gene, enzyme, and flux targets for cancer therapy
WO2012016188A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
JP6106085B2 (ja) 2010-08-24 2017-03-29 サーナ・セラピューティクス・インコーポレイテッドＳｉｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 内部非核酸スペーサーを含む一本鎖ＲＮＡｉ剤
WO2012044992A2 (en) 2010-09-30 2012-04-05 Agency For Science, Technology And Research (A*Star) Methods and reagents for detection and treatment of esophageal metaplasia
WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
WO2012068463A2 (en) 2010-11-18 2012-05-24 Beth Israel Deaconess Medicall Center, Inc. Methods of treating obesity by inhibiting nicotinamide n-methyl transferase (nnmt)
US9999673B2 (en) 2011-01-11 2018-06-19 Alnylam Pharmaceuticals, Inc. PEGylated lipids and their use for drug delivery
CN103476791A (zh) 2011-01-11 2013-12-25 菲特治疗公司 新型wnt组合物和该组合物的治疗用途
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
EP2753631A1 (de) 2011-09-07 2014-07-16 Marina Biotech, Inc. Synthese und verwendung von nukleinsäureverbindungen mit konformationseingeschränkten monomeren
JP6250543B2 (ja) 2011-09-27 2017-12-20 アルニラム・ファーマシューティカルズ・インコーポレーテッド ジ脂肪族置換ｐｅｇ化脂質
EP2766501B1 (de) 2011-10-14 2020-01-01 Accugenomics, Inc. Quantitative Nukleinsäureamplifikation
PE20141562A1 (es) 2011-10-14 2014-11-12 Genentech Inc Anticuerpos anti-htra1 y metodos de uso
JP2015502365A (ja) 2011-12-12 2015-01-22 オンコイミューニン，インコーポレイティド オリゴヌクレオチドのイン−ビボ送達
EP2797948B1 (de) 2011-12-27 2019-02-20 The J. David Gladstone Institutes Zusammensetzungen zur verwendung in der regulierung des glukosemetabolismus
WO2013119877A1 (en) 2012-02-07 2013-08-15 Aura Biosciences, Inc. Virion-derived nanospheres for selective delivery of therapeutic and diagnostic agents to cancer cells
US9352042B2 (en) 2012-02-24 2016-05-31 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
US20150267260A1 (en) 2012-05-25 2015-09-24 Accugenomics, Inc. Nucleic acid amplification and use thereof
KR101520383B1 (ko) 2012-08-02 2015-05-15 에이비온 주식회사 Ｈｐｖ 감염과 관련된 암의 치료용 조성물
KR20150130355A (ko) 2013-03-15 2015-11-23 더 리전트 오브 더 유니버시티 오브 캘리포니아 비환식 뉴클레오사이드 포스포네이트 디에스테르
CA2916499A1 (en) 2013-07-03 2015-01-08 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna
WO2015006740A2 (en) 2013-07-11 2015-01-15 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
WO2015042325A1 (en) 2013-09-18 2015-03-26 Aura Biosciences, Inc. Virus-like particle conjugates for diagnosis and treatment of tumors
WO2015048531A1 (en) 2013-09-26 2015-04-02 Beth Israel Deaconess Medical Center, Inc. Inhibition of sgk1 in the treatment of heart conditions
CN110003066B (zh) 2013-11-18 2021-09-03 阿克丘勒斯治疗公司 用于rna递送的可电离的阳离子脂质
WO2015074010A2 (en) 2013-11-18 2015-05-21 Beth Israel Deaconess Medical Center, Inc. Compositions and methods for cardiac regeneration
US9365610B2 (en) 2013-11-18 2016-06-14 Arcturus Therapeutics, Inc. Asymmetric ionizable cationic lipid for RNA delivery
JP6772062B2 (ja) 2013-12-02 2020-10-21 フィオ ファーマシューティカルズ コーポレーションＰｈｉｏ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｃｏｒｐ． 癌の免疫療法
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
US20150176076A1 (en) 2013-12-20 2015-06-25 Acetylon Pharmaceuticals, Inc. Histone deacetylase 6 (hdac6) biomarkers in multiple myeloma
SI3581654T1 (sl) 2013-12-27 2021-08-31 Dicerna Pharmaceuticals, Inc. Postopki in sestavki za specifično inhibicijo glikolat-oksidaze (HA01) z dvoverižno RNA
US9458464B2 (en) 2014-06-23 2016-10-04 The Johns Hopkins University Treatment of neuropathic pain
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
JP7081923B2 (ja) 2014-07-31 2022-06-07 ユーエイビー リサーチ ファンデーション アポｅ模倣ペプチド及び血漿コレステロールを取り除くためのより高い効果
MX377983B (es) 2014-09-15 2025-03-04 Univ California Análogos de nucleótidos.
US10351854B2 (en) 2014-10-10 2019-07-16 Dicerna Pharmaceuticals, Inc. Therapeutic inhibition of lactate dehydrogenase and agents therefor
WO2016061131A1 (en) 2014-10-14 2016-04-21 The J. David Gladstone Institutes Compositions and methods for reactivating latent immunodeficiency virus
DK3221293T3 (da) 2014-11-18 2023-04-03 Arcturus Therapeutics Inc Ioniserbar kationisk lipid til RNA-afgivelse
EP3865576A1 (de) 2014-12-15 2021-08-18 Dicerna Pharmaceuticals, Inc. Ligandenmodifizierte doppelsträngige nukleinsäuren
EP3331536A4 (de) 2015-08-03 2019-03-27 The Regents of The University of California Zusammensetzungen und verfahren zur modulierung der abhd2-aktivität
EP3875462A1 (de) 2015-09-15 2021-09-08 The Regents of The University of California Nukleotidanaloga
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
KR20180095511A (ko) 2015-10-30 2018-08-27 더 유나이티드 스테이츠 오브 어메리카, 애즈 리프리젠티드 바이 더 세크러테리, 디파트먼트 오브 헬쓰 앤드 휴먼 서비씨즈 표적화된 암 요법
BR112018007703A2 (pt) 2015-10-30 2018-11-06 Genentech Inc anticorpos, ácido nucleico isolado, métodos de produção de um anticorpo e de tratamento de um distúrbio, composição farmacêutica, terapia de combinação e uso do anticorpo
US10876121B2 (en) 2016-04-01 2020-12-29 Avidity Biosciences, Inc. EGFR nucleic acids and uses thereof
WO2017173297A1 (en) 2016-04-01 2017-10-05 Avidity Biosciences Llc Beta-catenin nucleic acids and uses thereof
MA45471A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques de phosphatidylinositol-3-kinase et leurs utilisations
KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
US10858656B2 (en) 2016-04-01 2020-12-08 Avidity Biosciences, Inc. KRAS nucleic acids and uses thereof
MA45468A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques myc et utilisations
EP3463388B1 (de) 2016-05-24 2024-09-11 Emory University Partikel mit rna-spaltenden nukleobasenpolymeren und verwendungen zur behandlung von entzündlichen erkrankungen
EP3496736A4 (de) 2016-08-03 2020-05-13 H. Lee Moffitt Cancer Center And Research Institute, Inc. Gegen tlr9 gerichtete therapeutika
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
EP3500696A4 (de) 2016-08-16 2020-04-08 Bluebird Bio, Inc. Il-10-rezeptor-alpha-homing-endonuklease-varianten, zusammensetzungen und verfahren zur verwendung
US20190262398A1 (en) 2016-08-23 2019-08-29 Bluebird Bio, Inc. Tim3 homing endonuclease variants, compositions, and methods of use
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
SG11201901757YA (en) 2016-09-08 2019-03-28 Bluebird Bio Inc Pd-1 homing endonuclease variants, compositions, and methods of use
SG11201903230QA (en) 2016-10-11 2019-05-30 Bluebird Bio Inc TCRa HOMING ENDONUCLEASE VARIANTS
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
US11654158B2 (en) 2016-11-17 2023-05-23 2Seventy Bio, Inc. TGFBeta signal convertor
WO2018119163A1 (en) 2016-12-21 2018-06-28 Payne Joseph E Ionizable cationic lipid for rna delivery
US10383952B2 (en) 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US10526284B2 (en) 2016-12-21 2020-01-07 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
KR20240172236A (ko) 2017-01-06 2024-12-09 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
AU2018221730B2 (en) 2017-02-15 2024-06-20 Novo Nordisk A/S Donor repair templates multiplex genome editing
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
AU2018251788B2 (en) 2017-04-12 2025-04-10 Aura Biosciences, Inc. Targeted combination therapy
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
EP3658573A1 (de) 2017-07-28 2020-06-03 President and Fellows of Harvard College Verfahren und zusammensetzungen zur entwicklung von basiseditoren unter verwendung von phagenunterstützter kontinuierlicher entwicklung (pace)
EP3676376B1 (de) 2017-08-30 2025-01-15 President and Fellows of Harvard College Hocheffiziente baseneditoren mit gam
US11306312B2 (en) 2017-09-01 2022-04-19 Thomas Jefferson University Compositions and methods for MYC messenger RNA inhibitors
WO2019051355A1 (en) 2017-09-08 2019-03-14 Ohio State Innovation Foundation NEW MICROARN INHIBITOR THERAPY FOR SYSTEMIC LUPUS ERYTHEMATOSUS
SG11202002517RA (en) 2017-09-22 2020-04-29 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
SG11202003126VA (en) 2017-10-04 2020-05-28 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and uses thereof
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
CN118638787A (zh) 2017-12-06 2024-09-13 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
EP3820495A4 (de) 2018-07-09 2022-07-20 The Broad Institute Inc. Rna-programmierbare epigenetische rna-modifikatoren und deren verwendungen
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
CA3122080A1 (en) 2018-12-06 2020-06-11 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
WO2020123938A1 (en) 2018-12-14 2020-06-18 Bluebird Bio, Inc. Dimerizing agent regulated immunoreceptor complexes
IL312067B2 (en) 2018-12-21 2025-08-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020210751A1 (en) 2019-04-12 2020-10-15 The Broad Institute, Inc. System for genome editing
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020219820A1 (en) 2019-04-25 2020-10-29 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
WO2020247782A1 (en) 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
JP7581252B2 (ja) 2019-06-06 2024-11-12 アビディティー バイオサイエンシーズ，インク． Ｕｎａアミダイトおよびその使用
GB2600859B (en) 2019-08-14 2024-04-03 Acuitas Therapeutics Inc Improved lipid nanoparticles for delivery of nucleic acids
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4058032A4 (de) 2019-12-19 2024-01-10 Entrada Therapeutics, Inc. Zusammensetzungen zur verabreichung von antisense-verbindungen
EP4114470A4 (de) 2020-03-03 2024-04-17 Arcturus Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von ornithintranscarbamylase-mangel
JP7709981B2 (ja) 2020-03-17 2025-07-17 ジェネヴァント サイエンシズ ゲーエムベーハー 肝星細胞への治療薬の脂質ナノ粒子送達のためのカチオン性脂質
TW202144572A (zh) 2020-03-19 2021-12-01 美商亞維代堤生物科學公司 治療臉肩胛肱骨肌肉失養症之組合物及方法
KR102927945B1 (ko) 2020-03-27 2026-02-23 어비디티 바이오사이언시스 인크. 근이영양증의 치료용 조성물 및 방법
WO2021222801A2 (en) 2020-05-01 2021-11-04 Arcturus Therapeutics, Inc. Nucleic acids and methods of treatment for cystic fibrosis
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
IL301253A (en) 2020-09-13 2023-05-01 Arcturus Therapeutics Inc As a mass of large RNA lipid nanoparticles
WO2022097157A2 (en) 2020-11-09 2022-05-12 1E Therapeutics, Ltd. Catalytic sequence based methods of treating or preventing bacterial infections
JP7880877B2 (ja) 2020-12-18 2026-06-26 イェダ リサーチ アンド ディベロップメント カンパニー リミテッド Ｃｈｄ２ハプロ不全の処置における使用のための組成物及びその同定方法
AU2021403156A1 (en) 2020-12-18 2023-07-13 Genevant Sciences Gmbh Peg lipids and lipid nanoparticles
AU2021416356B2 (en) 2020-12-28 2026-02-26 1E Therapeutics, Ltd. P21 mrna targeting dnazymes
EP4267741A2 (de) 2020-12-28 2023-11-01 1E Therapeutics, Ltd. P21-mrna-zielbereiche zum silencing
KR20240009393A (ko) 2021-03-31 2024-01-22 엔트라다 테라퓨틱스, 인크. 사이클릭 세포 침투 펩티드
AU2022270611A1 (en) 2021-05-03 2023-10-12 Astellas Institute For Regenerative Medicine Methods of generating mature corneal endothelial cells
IL308353A (en) 2021-05-10 2024-01-01 Entrada Therapeutics Inc Compositions and methods for modulating tissue distribution of intracellular therapies
US20250051393A1 (en) 2021-05-10 2025-02-13 Entrada Therapeutics, Inc. Compositions and methods for modulating interferon regulatory factor-5 (irf-5) activity
JP2024518476A (ja) 2021-05-10 2024-05-01 エントラーダ セラピューティクス，インコーポレイティド ｍＲＮＡスプライシングを調節するための組成物及び方法
EP4355875A1 (de) 2021-06-14 2024-04-24 2seventy bio, Inc. Verfahren zur aufreinigung einzelsträngiger rna
AU2022298774A1 (en) 2021-06-23 2023-12-14 Entrada Therapeutics, Inc. Antisense compounds and methods for targeting cug repeats
CA3227661A1 (en) 2021-08-05 2023-02-09 Weimin Wang 1'-alkyl modified ribose derivatives and methods of use
EP4401792A4 (de) 2021-09-16 2025-12-03 Avidity Biosciences Inc Zusammensetzungen und verfahren zur behandlung von fazioskapulohumeraler muskeldystrophie
CA3233113A1 (en) 2021-09-22 2023-03-30 Sanegene Bio Usa Inc. 2'-alkyl or 3'-alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides
US20230227480A1 (en) 2021-10-05 2023-07-20 Sanegene Bio Usa Inc. Polyhydroxylated cyclopentane derivatives and methods of use
KR20240146674A (ko) 2022-01-31 2024-10-08 제네반트 사이언시즈 게엠베하 지질 나노입자를 위한 이온화 가능한 양이온성 지질
WO2023144792A1 (en) 2022-01-31 2023-08-03 Genevant Sciences Gmbh Poly(alkyloxazoline)-lipid conjugates and lipid particles containing same
IL314850A (en) 2022-02-22 2024-10-01 Sanegene Bio Usa Inc Ribonucleotide derivatives substituted at the 5' position in the carboxyl group and methods of use
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
EP4524244A4 (de) 2022-05-13 2026-07-08 Seoul Nat Univ R&Db Foundation Sequenzdeterminanten der dsrna-verarbeitung durch dicer
IL317824A (en) 2022-07-11 2025-02-01 Sanegene Bio Usa Inc Ribose derivatives modified at the '2' position and methods of use
KR20250052378A (ko) 2022-07-21 2025-04-18 안티바 바이오사이언시즈, 인크. Hpv 감염 및 hpv-유도 신생물의 치료를 위한 조성물 및 투여 형태
EP4705312A1 (de) 2023-05-03 2026-03-11 Sanegene Bio USA Inc. Lipidbasierte konjugate für systemisches, zentrales nervensystem, peripheres nervensystem und okulare freisetzung
US12491257B2 (en) 2023-06-27 2025-12-09 Avidity Biosciences, Inc. Compositions and methods of using PRKAG2-targeting antibody-oligonucleotide conjugates
AU2024308906A1 (en) 2023-06-30 2026-02-05 Atrium Therapeutics, Inc. Compositions and methods of using pln-targeting antibody-oligonucleotide conjugates
AU2024301344A1 (en) 2023-07-24 2026-01-29 Sanegene Bio Usa Inc. Lipid-based enhancement agent for rna delivery and therapy
WO2025052278A1 (en) 2023-09-05 2025-03-13 Genevant Sciences Gmbh Pyrrolidine based cationic lipids for lipid nanoparticle delivery of therapeutics to hepatic stellate cells
WO2025076006A2 (en) 2023-10-02 2025-04-10 Avidity Biosciences, Inc. Antibody-oligonucleotide conjugate compositions and methods of inducing dmd exon 45 skipping
WO2025133951A1 (en) 2023-12-21 2025-06-26 Genevant Sciences Gmbh Ionizable lipids suitable for lipid nanoparticles
AU2025209576A1 (en) 2024-01-16 2026-06-11 Novo Nordisk A/S Albumin-targeted endonucleases, compositions, and methods of use
WO2026117631A2 (en) 2024-11-26 2026-06-04 City Therapeutics, Inc. Factor xi rnai agent compositions and methods of use thereof

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4987071A (en) * 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
CA2039718C (en) * 1989-08-31 2003-02-25 John J. Rossi Chimeric dna-rna catalytic sequences
US5149796A (en) * 1989-08-31 1992-09-22 City Of Hope Chimeric DNA-RNA catalytic sequences
WO1991019789A1 (en) * 1990-06-19 1991-12-26 Commonwealth Scientific And Industrial Research Organisation Endonucleases
AU649074B2 (en) * 1990-10-12 1994-05-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US6204027B1 (en) * 1992-02-26 2001-03-20 University Of Massachusetts Worcester Ribozymes having 2′-O substituted nucleotides in the flanking sequences

Also Published As

Publication number Publication date
WO1993015187A1 (en) 1993-08-05
US6713456B1 (en) 2004-03-30
US7074601B2 (en) 2006-07-11
EP1162263A1 (de) 2001-12-12
JPH07503613A (ja) 1995-04-20
EP1219709A1 (de) 2002-07-03
AU680405B2 (en) 1997-07-31
DE69331946T2 (de) 2003-01-02
CA2129119A1 (en) 1993-08-05
US6140491A (en) 2000-10-31
US5652094A (en) 1997-07-29
EP0625192A1 (de) 1994-11-23
AU3600193A (en) 1993-09-01
US20020137718A1 (en) 2002-09-26
MX9300499A (es) 1994-07-29
EP0625192B1 (de) 2002-05-22
DE69331946D1 (de) 2002-06-27

Similar Documents

Publication Publication Date Title
DE69331946D1 (de) Nukleozyme
ES2186690T3 (es) Acido nucleico enzimatico que contiene no-nucleotidos.
DE69403642D1 (de) 7-deazapurin modifizierte oligonukleotide
NO975731L (no) Katalytisk DNA
DE69736598D1 (de) Ribonukleaseresistente rna präparation und seine verwendung
ATE228534T1 (de) Struktur höherer ordnung und bindung von peptidnukleinsäuren
DE3787356D1 (de) Expression von höheren eukaryotischen genen in aspergillus.
NL971024I2 (nl) Acrylzuuramiden, hun bereiding en hun toepassing als fungiciden.
FI864331A7 (fi) Transformaatio ja vieraiden geenien ilmaiseminen puulajeilla.
DE3673244D1 (de) Methode um acrylprodukten antimikrobielle aktivitaet zu verleihen.
EP0585257A4 (de) Für natürliche killerzellen spezifische dna- und aminosäuresequenz.
FR2715847B1 (fr) Composition contenant des acides nucléiques, préparation et utilisations.
BE854916A (fr) Procede de purification possedant notamment une activite interferon, preparations purifiees ainsi obtenues et leur application
KR910008132A (ko) 자기 가공 활성을 갖는 다기능 rna, 이의 제조방법 및 용도
EP0707653A4 (de) Methode der spezifischen abschaltung von genen durch dna-methylierung
MA23475A1 (fr) Acide 5-vinyl et 5-ethinyl -quinolone et naphtyridone -carboxyliques.
DE69937959D1 (de) Verwendung von substanzen mit oxytocin aktivität mit dem ziel der zellregeneration
DE3879030D1 (de) Methode, um metallstrukturen korrosionsbestaendig, schalldicht und erschuetterungssicher zu machen.
EP0567785A3 (de) Protein mit Alpha-Glukosidase-Aktivität, DNS mit dessen genetischer Information und Herstellung von Alpha-Glukosidase.
UA15920A (uk) Аміди циклометілеh-1, 2-дикарбоhових кислот, які мають гіпотеhзивhу активhість
DE3686893D1 (de) Gaerung zur gewinnung von d-milchsaeure.
ATE137263T1 (de) Erhöhung der katalytischen aktivität von ribozymen mit einem benachbartem oligonukleotid als heffer
ES2187939T3 (es) Moleculas aisladas de acido nucleico codificantes de miembros de la familia ssx y utilizaciones de las mismas.
ATE209683T1 (de) Planze resistent gegen mindestens zwei viren und dessen preparation
FR2680797B1 (de)

Legal Events

Date Code Title Description
UEP Publication of translation of european patent specification
REN Ceased due to non-payment of the annual fee