JP2021518139A5 - - Google Patents

Info

Publication number
JP2021518139A5
JP2021518139A5 JP2020550714A JP2020550714A JP2021518139A5 JP 2021518139 A5 JP2021518139 A5 JP 2021518139A5 JP 2020550714 A JP2020550714 A JP 2020550714A JP 2020550714 A JP2020550714 A JP 2020550714A JP 2021518139 A5 JP2021518139 A5 JP 2021518139A5
Authority
JP
Japan
Prior art keywords
seq
sequence
polypeptide
variant
amino acid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
JP2020550714A
Other languages
English (en)
Japanese (ja)
Other versions
JP7550648B2 (ja
JP2021518139A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/023044 external-priority patent/WO2019183150A1/en
Publication of JP2021518139A publication Critical patent/JP2021518139A/ja
Publication of JP2021518139A5 publication Critical patent/JP2021518139A5/ja
Application granted granted Critical
Publication of JP7550648B2 publication Critical patent/JP7550648B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2020550714A 2018-03-19 2019-03-19 新規rnaプログラム可能エンドヌクレアーゼ系およびその使用 Active JP7550648B2 (ja)

Applications Claiming Priority (19)

Application Number Priority Date Filing Date Title
EP18162683 2018-03-19
EP18162681.3 2018-03-19
EP18162681 2018-03-19
EP18162683.9 2018-03-19
EP18172625 2018-05-16
EP18172625.8 2018-05-16
EP18174707.2 2018-05-29
EP18174707 2018-05-29
EP18181680 2018-07-04
EP18181680.2 2018-07-04
US201862745239P 2018-10-12 2018-10-12
US201862745238P 2018-10-12 2018-10-12
US201862745240P 2018-10-12 2018-10-12
US201862745246P 2018-10-12 2018-10-12
US62/745,239 2018-10-12
US62/745,240 2018-10-12
US62/745,246 2018-10-12
US62/745,238 2018-10-12
PCT/US2019/023044 WO2019183150A1 (en) 2018-03-19 2019-03-19 Novel rna-programmable endonuclease systems and uses thereof

Publications (3)

Publication Number Publication Date
JP2021518139A JP2021518139A (ja) 2021-08-02
JP2021518139A5 true JP2021518139A5 (https=) 2022-03-29
JP7550648B2 JP7550648B2 (ja) 2024-09-13

Family

ID=65895066

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020550714A Active JP7550648B2 (ja) 2018-03-19 2019-03-19 新規rnaプログラム可能エンドヌクレアーゼ系およびその使用

Country Status (8)

Country Link
US (2) US12203110B2 (https=)
EP (1) EP3768834B1 (https=)
JP (1) JP7550648B2 (https=)
CN (1) CN112424348B (https=)
AU (1) AU2019239957A1 (https=)
CA (1) CA3092497A1 (https=)
MA (1) MA52074A (https=)
WO (1) WO2019183150A1 (https=)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
EP3555297A1 (en) 2016-12-19 2019-10-23 Editas Medicine, Inc. Assessing nuclease cleavage
JP7229923B2 (ja) 2017-01-06 2023-02-28 エディタス・メディシン、インコーポレイテッド ヌクレアーゼ切断を評価する方法
US11499151B2 (en) 2017-04-28 2022-11-15 Editas Medicine, Inc. Methods and systems for analyzing guide RNA molecules
MX2019014640A (es) 2017-06-09 2020-10-05 Editas Medicine Inc Nucleasas de repeticiones palíndromicas agrupadas y regularmente interespaciadas de proteina asociada 9 (cas9) genomanipuladas.
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
WO2019165168A1 (en) 2018-02-23 2019-08-29 Pioneer Hi-Bred International, Inc. Novel cas9 orthologs
MA52074A (fr) 2018-03-19 2021-01-27 Bayer Healthcare Llc Nouveaux systèmes d'endonucléase à arn programmable et leurs utilisations
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
CN113710799B (zh) * 2018-11-28 2024-11-12 克里斯珀医疗股份公司 用于在LNP中使用的编码CAS9的优化mRNA
WO2020123887A2 (en) 2018-12-14 2020-06-18 Pioneer Hi-Bred International, Inc. Novel crispr-cas systems for genome editing
SG11202109741VA (en) * 2019-03-12 2021-10-28 Crispr Therapeutics Ag Novel high fidelity rna-programmable endonuclease systems and uses thereof
MA56393A (fr) 2019-06-28 2022-05-04 Bayer Healthcare Llc Matériels et méthodes de régulation de l'édition de gènes
WO2021173359A1 (en) * 2020-02-24 2021-09-02 Pioneer Hi-Bred International, Inc. Novel crispr-cas systems for genome editing
WO2022018638A1 (en) 2020-07-21 2022-01-27 Crispr Therapeutics Ag Genome-editing compositions and methods to modulate faah for treatment of neurological disorders
AU2021342057A1 (en) * 2020-09-09 2023-04-06 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of duchenne muscular dystrophy
EP4222264A1 (en) 2020-09-30 2023-08-09 CRISPR Therapeutics AG Materials and methods for treatment of amyotrophic lateral sclerosis
WO2022098933A1 (en) 2020-11-06 2022-05-12 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of dm1 with slucas9 and sacas9
WO2022133246A1 (en) 2020-12-17 2022-06-23 Vertex Pharmaceuticals Incorporated Compositions and methods for editing beta-globin for treatment of hemaglobinopathies
WO2022229851A1 (en) * 2021-04-26 2022-11-03 Crispr Therapeutics Ag Compositions and methods for using slucas9 scaffold sequences
WO2022238958A1 (en) 2021-05-12 2022-11-17 Crispr Therapeutics Ag Multiplex gene editing
EP4512403A1 (en) 2023-08-22 2025-02-26 Friedrich-Schiller-Universität Jena Neuropeptide b and w-receptor as a target for treating mood disorders and/or chronic stress

Family Cites Families (198)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US538A (en) 1837-12-26 Self-separating link for connecting railroad-cars and locomotives
US5552A (en) 1848-05-09 Paul stillman
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4476301A (en) 1982-04-29 1984-10-09 Centre National De La Recherche Scientifique Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon
JPS5927900A (ja) 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
FR2540122B1 (fr) 1983-01-27 1985-11-29 Centre Nat Rech Scient Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application
US4605735A (en) 1983-02-14 1986-08-12 Wakunaga Seiyaku Kabushiki Kaisha Oligonucleotide derivatives
US4948882A (en) 1983-02-22 1990-08-14 Syngene, Inc. Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis
US4824941A (en) 1983-03-10 1989-04-25 Julian Gordon Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems
US4587044A (en) 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5118802A (en) 1983-12-20 1992-06-02 California Institute Of Technology DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside
US5550111A (en) 1984-07-11 1996-08-27 Temple University-Of The Commonwealth System Of Higher Education Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
US4828979A (en) 1984-11-08 1989-05-09 Life Technologies, Inc. Nucleotide analogs for nucleic acid labeling and detection
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US4762779A (en) 1985-06-13 1988-08-09 Amgen Inc. Compositions and methods for functionalizing nucleic acids
US5317098A (en) 1986-03-17 1994-05-31 Hiroaki Shizuya Non-radioisotope tagging of fragments
JPS638396A (ja) 1986-06-30 1988-01-14 Wakunaga Pharmaceut Co Ltd ポリ標識化オリゴヌクレオチド誘導体
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
EP0366685B1 (en) 1987-06-24 1994-10-19 Howard Florey Institute Of Experimental Physiology And Medicine Nucleoside derivatives
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US4924624A (en) 1987-10-22 1990-05-15 Temple University-Of The Commonwealth System Of Higher Education 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
US5525465A (en) 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
DE3738460A1 (de) 1987-11-12 1989-05-24 Max Planck Gesellschaft Modifizierte oligonukleotide
US5082830A (en) 1988-02-26 1992-01-21 Enzo Biochem, Inc. End labeled nucleotide probe
JPH03503894A (ja) 1988-03-25 1991-08-29 ユニバーシィティ オブ バージニア アランミ パテンツ ファウンデイション オリゴヌクレオチド n‐アルキルホスホラミデート
US5278302A (en) 1988-05-26 1994-01-11 University Patents, Inc. Polynucleotide phosphorodithioates
US5109124A (en) 1988-06-01 1992-04-28 Biogen, Inc. Nucleic acid probe linked to a label having a terminal cysteine
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5262536A (en) 1988-09-15 1993-11-16 E. I. Du Pont De Nemours And Company Reagents for the preparation of 5'-tagged oligonucleotides
US5512439A (en) 1988-11-21 1996-04-30 Dynal As Oligonucleotide-linked magnetic particles and uses thereof
US5457183A (en) 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
US5599923A (en) 1989-03-06 1997-02-04 Board Of Regents, University Of Tx Texaphyrin metal complexes having improved functionalization
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5585362A (en) 1989-08-22 1996-12-17 The Regents Of The University Of Michigan Adenovirus vectors for gene therapy
US5451463A (en) 1989-08-28 1995-09-19 Clontech Laboratories, Inc. Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5254469A (en) 1989-09-12 1993-10-19 Eastman Kodak Company Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5292873A (en) 1989-11-29 1994-03-08 The Research Foundation Of State University Of New York Nucleic acids labeled with naphthoquinone probe
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5486603A (en) 1990-01-08 1996-01-23 Gilead Sciences, Inc. Oligonucleotide having enhanced binding affinity
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
AU7579991A (en) 1990-02-20 1991-09-18 Gilead Sciences, Inc. Pseudonucleosides and pseudonucleotides and their polymers
US5214136A (en) 1990-02-20 1993-05-25 Gilead Sciences, Inc. Anthraquinone-derivatives oligonucleotides
US5321131A (en) 1990-03-08 1994-06-14 Hybridon, Inc. Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
US5451513A (en) 1990-05-01 1995-09-19 The State University of New Jersey Rutgers Method for stably transforming plastids of multicellular plants
ES2116977T3 (es) 1990-05-11 1998-08-01 Microprobe Corp Soportes solidos para ensayos de hibridacion de acidos nucleicos y metodos para inmovilizar oligonucleotidos de modo covalente.
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
JPH0874B2 (ja) 1990-07-27 1996-01-10 アイシス・ファーマシューティカルス・インコーポレーテッド 遺伝子発現を検出および変調するヌクレアーゼ耐性、ピリミジン修飾オリゴヌクレオチド
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
MY107332A (en) 1990-08-03 1995-11-30 Sterling Drug Inc Compounds and methods for inhibiting gene expression.
US5177196A (en) 1990-08-16 1993-01-05 Microprobe Corporation Oligo (α-arabinofuranosyl nucleotides) and α-arabinofuranosyl precursors thereof
US5512667A (en) 1990-08-28 1996-04-30 Reed; Michael W. Trifunctional intermediates for preparing 3'-tailed oligonucleotides
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
JPH06505704A (ja) 1990-09-20 1994-06-30 ギリアド サイエンシズ,インコーポレイテッド 改変ヌクレオシド間結合
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
KR930702373A (ko) 1990-11-08 1993-09-08 안토니 제이. 페이네 합성 올리고누클레오티드에 대한 다중 리포터(Reporter)그룹의 첨합
US5222982A (en) 1991-02-11 1993-06-29 Ommaya Ayub K Spinal fluid driven artificial organ
JPH06505186A (ja) 1991-02-11 1994-06-16 オマーヤ,アユブ ケー. 脊髄液駆動式人工器官
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5371241A (en) 1991-07-19 1994-12-06 Pharmacia P-L Biochemicals Inc. Fluorescein labelled phosphoramidites
US5571799A (en) 1991-08-12 1996-11-05 Basco, Ltd. (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response
DE69233013T2 (de) 1991-08-20 2004-03-04 The Government Of The United States Of America As Represented By The Secretary Of National Institute Of Health, Office Of Technology Transfer Adenovirus vermittelter gentransfer in den gastrointestinaltrakt
DE69233046T2 (de) 1991-10-24 2004-03-04 Isis Pharmaceuticals, Inc., Carlsfeld Derivatisierte oligonukleotide mit verbessertem aufnahmevermögen
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
FR2688514A1 (fr) 1992-03-16 1993-09-17 Centre Nat Rech Scient Adenovirus recombinants defectifs exprimant des cytokines et medicaments antitumoraux les contenant.
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
US7153684B1 (en) 1992-10-08 2006-12-26 Vanderbilt University Pluripotential embryonic stem cells and methods of making same
EP1024198A3 (en) 1992-12-03 2002-05-29 Genzyme Corporation Pseudo-adenoviral vectors for the gene therapy of haemophiliae
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
HU9501974D0 (en) 1993-03-31 1995-09-28 Sterling Winthrop Inc Oligonucleotides with amide linkages replacing phosphodiester linkages
EP0705344B8 (en) 1993-06-24 2006-05-10 Advec Inc. Adenovirus vectors for gene therapy
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
AU687117B2 (en) 1993-10-25 1998-02-19 Canji, Inc. Recombinant adenoviral vector and methods of use
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5576198A (en) 1993-12-14 1996-11-19 Calgene, Inc. Controlled expression of transgenic constructs in plant plastids
US5545817A (en) 1994-03-11 1996-08-13 Calgene, Inc. Enhanced expression in a plant plastid
US5545818A (en) 1994-03-11 1996-08-13 Calgene Inc. Expression of Bacillus thuringiensis cry proteins in plant plastids
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5597696A (en) 1994-07-18 1997-01-28 Becton Dickinson And Company Covalent cyanine dye oligonucleotide conjugates
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US5843780A (en) 1995-01-20 1998-12-01 Wisconsin Alumni Research Foundation Primate embryonic stem cells
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
US6632670B1 (en) 1995-09-08 2003-10-14 Genzyme Corporation AAV vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ/エス オリゴヌクレオチド類似体
CA2307807C (en) 1997-10-23 2008-09-02 Andrea G. Bodnar Methods and materials for the growth of primate-derived primordial stem cells in feeder-free culture
US7410798B2 (en) 2001-01-10 2008-08-12 Geron Corporation Culture system for rapid expansion of human embryonic stem cells
US6667176B1 (en) 2000-01-11 2003-12-23 Geron Corporation cDNA libraries reflecting gene expression during growth and differentiation of human pluripotent stem cells
US7078387B1 (en) 1998-12-28 2006-07-18 Arch Development Corp. Efficient and stable in vivo gene transfer to cardiomyocytes using recombinant adeno-associated virus vectors
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US7229961B2 (en) 1999-08-24 2007-06-12 Cellgate, Inc. Compositions and methods for enhancing drug delivery across and into ocular tissues
US6593292B1 (en) 1999-08-24 2003-07-15 Cellgate, Inc. Compositions and methods for enhancing drug delivery across and into epithelial tissues
EP1083231A1 (en) 1999-09-09 2001-03-14 Introgene B.V. Smooth muscle cell promoter and uses thereof
US7256286B2 (en) 1999-11-30 2007-08-14 The Board Of Trustees Of The Leland Stanford Junior University Bryostatin analogues, synthetic methods and uses
US6287860B1 (en) 2000-01-20 2001-09-11 Isis Pharmaceuticals, Inc. Antisense inhibition of MEKK2 expression
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
DE60233137D1 (de) 2001-02-16 2009-09-10 Univ R Transporter mit beabstandeten arginin-teilchen
US20030158403A1 (en) 2001-07-03 2003-08-21 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
US7169874B2 (en) 2001-11-02 2007-01-30 Bausch & Lomb Incorporated High refractive index polymeric siloxysilane compositions
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
CN101267805A (zh) 2005-07-27 2008-09-17 普洛体维生物治疗公司 制造脂质体的系统和方法
US8278104B2 (en) 2005-12-13 2012-10-02 Kyoto University Induced pluripotent stem cells produced with Oct3/4, Klf4 and Sox2
US8048999B2 (en) 2005-12-13 2011-11-01 Kyoto University Nuclear reprogramming factor
US20090227032A1 (en) 2005-12-13 2009-09-10 Kyoto University Nuclear reprogramming factor and induced pluripotent stem cells
DK2019683T4 (da) 2006-04-25 2022-08-29 Univ California Indgivelse af vækstfaktorer til behandling af CNS-lidelser
WO2008060360A2 (en) 2006-09-28 2008-05-22 Surmodics, Inc. Implantable medical device with apertures for delivery of bioactive agents
JP2008307007A (ja) 2007-06-15 2008-12-25 Bayer Schering Pharma Ag 出生後のヒト組織由来未分化幹細胞から誘導したヒト多能性幹細胞
US9683232B2 (en) 2007-12-10 2017-06-20 Kyoto University Efficient method for nuclear reprogramming
WO2009097468A2 (en) 2008-01-29 2009-08-06 Kliman Gilbert H Drug delivery devices, kits and methods therefor
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
WO2013080784A1 (ja) 2011-11-30 2013-06-06 シャープ株式会社 メモリ回路とその駆動方法、及び、これを用いた不揮発性記憶装置、並びに、液晶表示装置
US9802715B2 (en) 2012-03-29 2017-10-31 The Boeing Company Fastener systems that provide EME protection
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
AU2013266968B2 (en) * 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
WO2014008334A1 (en) 2012-07-06 2014-01-09 Alnylam Pharmaceuticals, Inc. Stable non-aggregating nucleic acid lipid particle formulations
CN113355357B (zh) 2012-12-12 2024-12-03 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
CN120574876A (zh) * 2013-08-22 2025-09-02 纳幕尔杜邦公司 使用向导rna/cas内切核酸酶系统的植物基因组修饰及其使用方法
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
US9074199B1 (en) * 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
EP3470089A1 (en) 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
EP3083556B1 (en) 2013-12-19 2019-12-25 Novartis AG Lipids and lipid compositions for the delivery of active agents
EP3089989B1 (en) 2013-12-31 2020-06-24 The Regents of The University of California Cas9 crystals and methods of use thereof
US10821175B2 (en) 2014-02-25 2020-11-03 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
WO2015141521A1 (ja) 2014-03-21 2015-09-24 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及び記録媒体
EP3540061A1 (en) 2014-04-02 2019-09-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
AU2016263026A1 (en) * 2015-05-15 2017-11-09 Pioneer Hi-Bred International, Inc. Guide RNA/Cas endonuclease systems
TWI906646B (zh) 2015-06-18 2025-12-01 美商博得學院股份有限公司 降低脫靶效應的crispr酶突變
US11008555B2 (en) 2015-09-17 2021-05-18 The Regents Of The University Of California Variant Cas9 polypeptides comprising internal insertions
EA201891000A1 (ru) 2015-10-22 2018-12-28 МОДЕРНАТиЭкс, ИНК. Вакцина против респираторно-синцитиального вируса
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
PL3386484T3 (pl) 2015-12-10 2022-07-25 Modernatx, Inc. Kompozycje i sposoby dostarczania środków terapeutycznych
US20190032088A1 (en) 2016-02-26 2019-01-31 Cellectis Micelle based system nuclease encapsulation for in-vivo gene editing
LT3436077T (lt) 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams
US11427838B2 (en) 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
AU2017286835B2 (en) 2016-06-29 2023-12-14 Crispr Therapeutics Ag Compositions and methods for gene editing
JP2019524149A (ja) 2016-08-20 2019-09-05 アベリノ ラボ ユーエスエー インコーポレイテッドAvellino Lab USA, Inc. 一本鎖ガイドRNA、CRISPR/Cas9システム、及びそれらの使用方法
US20180127786A1 (en) 2016-09-23 2018-05-10 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing
EP3532103B1 (en) 2016-10-26 2025-12-03 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
AU2017374042C1 (en) 2016-12-09 2024-07-11 Acuitas Therapeutics, Inc. Delivery of target specific nucleases
MA52074A (fr) 2018-03-19 2021-01-27 Bayer Healthcare Llc Nouveaux systèmes d'endonucléase à arn programmable et leurs utilisations
CN113710799B (zh) * 2018-11-28 2024-11-12 克里斯珀医疗股份公司 用于在LNP中使用的编码CAS9的优化mRNA

Similar Documents

Publication Publication Date Title
JP2021518139A5 (https=)
JP2021500072A5 (https=)
JP2022516130A5 (https=)
US11702643B2 (en) System and method for genome editing
KR102021585B1 (ko) 두 개의 벡터로부터 발현된 Cas9 단백질을 이용한 유전자 발현 조절 방법
EP3178935B1 (en) Genome editing using campylobacter jejuni crispr/cas system-derived rgen
CN113373130A (zh) Cas12蛋白、含有Cas12蛋白的基因编辑系统及应用
DK3155099T3 (en) NUCLEASE MEDIATED DNA COLLECTION
JP2023541400A5 (https=)
US20190119678A1 (en) Means and methods for inactivating therapeutic dna in a cell
JP2021522783A5 (https=)
JP2021526858A5 (https=)
JP2022542839A5 (https=)
JP2023502473A5 (https=)
JP2019536425A5 (https=)
WO2012172277A4 (en) Simian adenovirus and hybrid adenoviral vectors
JP2016165307A5 (https=)
WO2019127087A1 (zh) 基因组编辑系统和方法
JP2021536250A5 (https=)
RU2022103641A (ru) Искусственная модификация генома для регуляции экспрессии гена
WO2023206872A1 (zh) 一种工程优化的核酸酶、向导rna、编辑系统和应用
JP2024531607A5 (https=)
JP2020511931A5 (https=)
JP2023525263A5 (https=)
CN104152414A (zh) 对细胞基因组的预定位点进行基因改造的方法