JP2024083479A5 - - Google Patents

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JP2024083479A5
JP2024083479A5 JP2024061564A JP2024061564A JP2024083479A5 JP 2024083479 A5 JP2024083479 A5 JP 2024083479A5 JP 2024061564 A JP2024061564 A JP 2024061564A JP 2024061564 A JP2024061564 A JP 2024061564A JP 2024083479 A5 JP2024083479 A5 JP 2024083479A5
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fusion protein
target gene
polypeptide
region
polypeptide domain
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JP2024061564A
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JP2024083479A (ja
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JP2024061564A 2015-02-09 2024-04-05 エピゲノム編集のための組成物および方法 Pending JP2024083479A (ja)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562113569P 2015-02-09 2015-02-09
US62/113,569 2015-02-09
JP2017560481A JP6929791B2 (ja) 2015-02-09 2016-02-09 エピゲノム編集のための組成物および方法
JP2021102533A JP7553110B2 (ja) 2015-02-09 2021-06-21 エピゲノム編集のための組成物および方法

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JP2024083479A JP2024083479A (ja) 2024-06-21
JP2024083479A5 true JP2024083479A5 (enExample) 2025-03-17

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JP2017560481A Active JP6929791B2 (ja) 2015-02-09 2016-02-09 エピゲノム編集のための組成物および方法
JP2021102533A Active JP7553110B2 (ja) 2015-02-09 2021-06-21 エピゲノム編集のための組成物および方法
JP2024061564A Pending JP2024083479A (ja) 2015-02-09 2024-04-05 エピゲノム編集のための組成物および方法

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JP2021102533A Active JP7553110B2 (ja) 2015-02-09 2021-06-21 エピゲノム編集のための組成物および方法

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US (4) US10676726B2 (enExample)
EP (1) EP3256487A4 (enExample)
JP (3) JP6929791B2 (enExample)
WO (1) WO2016130600A2 (enExample)

Families Citing this family (94)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6261500B2 (ja) 2011-07-22 2018-01-17 プレジデント アンド フェローズ オブ ハーバード カレッジ ヌクレアーゼ切断特異性の評価および改善
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
KR102210319B1 (ko) 2013-03-15 2021-02-01 더 제너럴 하스피탈 코포레이션 특정 게놈 좌위에 대한 유전적 및 후성적 조절 단백질의 rna-안내 표적화
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
GB201418965D0 (enExample) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
WO2017015637A1 (en) 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
EA201890565A1 (ru) 2015-08-25 2019-04-30 Дьюк Юниверсити Композиции и способы улучшения специфичности в геномной инженерии с применением рнк-направляемых эндонуклеаз
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
EP3365356B1 (en) 2015-10-23 2023-06-28 President and Fellows of Harvard College Nucleobase editors and uses thereof
WO2017095967A2 (en) 2015-11-30 2017-06-08 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
EP3478313B1 (en) 2016-06-29 2022-05-04 CRISPR Therapeutics AG Materials and methods for treatment of amyotrophic lateral sclerosis (als) and other related disorders
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
JP7231935B2 (ja) 2016-08-03 2023-03-08 プレジデント アンド フェローズ オブ ハーバード カレッジ アデノシン核酸塩基編集因子およびそれらの使用
EP3497214B1 (en) 2016-08-09 2023-06-28 President and Fellows of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
US20190194633A1 (en) * 2016-08-10 2019-06-27 Duke University Compositions, systems and methods for programming immune cell function through targeted gene regulation
AU2017313912B2 (en) * 2016-08-19 2024-01-04 Whitehead Institute For Biomedical Research Methods of editing DNA methylation
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
EP3551218A4 (en) 2016-12-12 2020-12-09 Whitehead Institute for Biomedical Research REGULATION OF TRANSCRIPTION THANKS TO CTCF LOOP ANCHORS
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
EP3565891B1 (en) 2017-01-09 2023-04-12 Whitehead Institute for Biomedical Research Methods of altering gene expression by perturbing transcription factor multimers that structure regulatory loops
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
EP3596117A4 (en) 2017-03-17 2021-01-13 The Johns Hopkins University TARGETED EPIGENETIC THERAPY AGAINST THE DISTAL EXPRESSION REGULATORY ELEMENT OF TGFB2
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
CN111757937A (zh) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 腺苷碱基编辑器的用途
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US20210017544A1 (en) * 2017-12-22 2021-01-21 Novozymes A/S Counter-Selection by Inhibition of Conditionally Essential Genes
WO2019204766A1 (en) 2018-04-19 2019-10-24 The Regents Of The University Of California Compositions and methods for gene editing
JP2021523696A (ja) * 2018-04-23 2021-09-09 デューク ユニバーシティ Dnaメチル化の標的化された編集によるsnca発現の下方調節
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020160125A1 (en) * 2019-01-29 2020-08-06 Flagship Pioneering Innovations V, Inc. Compositions comprising an endonuclease and methods for purifying an endonuclease
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN114599403A (zh) * 2019-08-19 2022-06-07 杜克大学 通过基于crispr/cas9的转录激活物获得的骨骼肌成肌细胞祖细胞谱系的特化
EP4034655A1 (en) * 2019-09-23 2022-08-03 Vigeneron GmbH Method of transactivating a homologous gene of a gene of interest and an in vitro method of diagnosing a disease
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
MX2022014008A (es) 2020-05-08 2023-02-09 Broad Inst Inc Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo.
WO2022008557A2 (en) * 2020-07-08 2022-01-13 UCB Biopharma SRL Modulation of cftr expression
CN112481262B (zh) * 2020-12-04 2022-08-19 中国农业科学院农业基因组研究所 一种基于CRISPR/Cas9基因编辑技术分析增强子细胞生物学功能的方法
MX2023007524A (es) * 2020-12-22 2023-09-19 Chroma Medicine Inc Composiciones y metodos para edicion epigenetica.
CA3227103A1 (en) 2021-07-30 2023-02-02 Matthew P. GEMBERLING Compositions and methods for modulating expression of frataxin (fxn)
US20240252684A1 (en) 2021-07-30 2024-08-01 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
JP7707826B2 (ja) * 2021-10-06 2025-07-15 株式会社三洋物産 遊技機
JP7707821B2 (ja) * 2021-10-06 2025-07-15 株式会社三洋物産 遊技機
JP7707831B2 (ja) * 2021-10-06 2025-07-15 株式会社三洋物産 遊技機
JP7707823B2 (ja) * 2021-10-06 2025-07-15 株式会社三洋物産 遊技機
JP7707829B2 (ja) * 2021-10-06 2025-07-15 株式会社三洋物産 遊技機
JP7707825B2 (ja) * 2021-10-06 2025-07-15 株式会社三洋物産 遊技機
JP7707836B2 (ja) * 2021-10-06 2025-07-15 株式会社三洋物産 遊技機
JP7707828B2 (ja) * 2021-10-06 2025-07-15 株式会社三洋物産 遊技機
JP7707824B2 (ja) * 2021-10-06 2025-07-15 株式会社三洋物産 遊技機
JP7707833B2 (ja) * 2021-10-06 2025-07-15 株式会社三洋物産 遊技機
JP7707820B2 (ja) * 2021-10-06 2025-07-15 株式会社三洋物産 遊技機
JP7707822B2 (ja) * 2021-10-06 2025-07-15 株式会社三洋物産 遊技機
JP7707827B2 (ja) * 2021-10-06 2025-07-15 株式会社三洋物産 遊技機
JP7707835B2 (ja) * 2021-10-06 2025-07-15 株式会社三洋物産 遊技機
JP7707832B2 (ja) * 2021-10-06 2025-07-15 株式会社三洋物産 遊技機
JP7707834B2 (ja) * 2021-10-06 2025-07-15 株式会社三洋物産 遊技機
EP4463549A2 (en) 2022-01-14 2024-11-20 Tune Therapeutics, Inc. Compositions, systems, and methods for programming t cell phenotypes through targeted gene repression
WO2023137471A1 (en) 2022-01-14 2023-07-20 Tune Therapeutics, Inc. Compositions, systems, and methods for programming t cell phenotypes through targeted gene activation
CN114507689B (zh) * 2022-01-27 2024-01-16 山东大学 一种提高真核生物基因编辑效率的方法及其应用
EP4273242A1 (en) * 2022-05-03 2023-11-08 ETH Zurich Crispr-based modification of human hbd gene
JP2025524469A (ja) 2022-06-24 2025-07-30 チューン セラピューティクス インコーポレイテッド 標的を定めた遺伝子抑制を介して低密度リポタンパク質を低減するための組成物、系、及び方法
CA3261865A1 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS
AU2023325407A1 (en) 2022-08-19 2025-02-20 Tune Therapeutics, Inc. Compositions, systems, and methods for regulation of hepatitis b virus through targeted gene repression
EP4590821A2 (en) 2022-09-19 2025-07-30 Tune Therapeutics, Inc. Compositions, systems, and methods for modulating t cell function
AU2023364547A1 (en) * 2022-10-21 2025-04-10 Watchmaker Genomics, Inc. Methods and compositions for sequencing library normalization
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024168154A2 (en) * 2023-02-09 2024-08-15 William Marsh Rice University Engineered lysine acyltransferases and methods of use thereof
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2025029840A1 (en) 2023-07-31 2025-02-06 Tune Therapeutics, Inc. Compositions and methods for multiplexed activation and repression of t cell gene expression
WO2025029835A1 (en) 2023-07-31 2025-02-06 Tune Therapeutics, Inc. Compositions and methods for modulating il-2 gene expression
WO2025038494A1 (en) 2023-08-11 2025-02-20 Tune Therapeutics, Inc. Compositions, systems, and methods for lymphoid cell differentiation using targeted gene activation
WO2025059073A1 (en) 2023-09-11 2025-03-20 Tune Therapeutics, Inc. Epigenetic editing methods and systems for differentiating stem cells
CN118291415B (zh) * 2024-03-14 2024-11-19 内蒙古大学 一种CRISPR::Cr-P300M巴豆酰化修饰编辑工具及其应用
WO2025260068A1 (en) 2024-06-14 2025-12-18 Tune Therapeutics, Inc. Lipid nanoparticle formulation for delivery of nucleic acids to cells

Family Cites Families (428)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US4554101A (en) 1981-01-09 1985-11-19 New York Blood Center, Inc. Identification and preparation of epitopes on antigens and allergens on the basis of hydrophilicity
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
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
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
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
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
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
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5405938A (en) 1989-12-20 1995-04-11 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
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
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
EP0260032B1 (en) 1986-09-08 1994-01-26 Ajinomoto Co., Inc. Compounds for the cleavage at a specific position of RNA, oligomers employed for the formation of said compounds, and starting materials for the synthesis of said oligomers
US5219740A (en) 1987-02-13 1993-06-15 Fred Hutchinson Cancer Research Center Retroviral gene transfer into diploid fibroblasts for gene therapy
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
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
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
CA1340032C (en) 1987-06-24 1998-09-08 Jim Haralambidis Lucleoside 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
US5403711A (en) 1987-11-30 1995-04-04 University Of Iowa Research Foundation Nucleic acid hybridization and amplification method for detection of specific sequences in which a complementary labeled nucleic acid probe is cleaved
WO1989005358A1 (en) 1987-11-30 1989-06-15 University Of Iowa Research Foundation Dna and rna molecules stabilized by modifications of the 3'-terminal phosphodiester linkage and their use as nucleic acid probes and as therapeutic agents to block the expression of specifically targeted genes
US5082830A (en) 1988-02-26 1992-01-21 Enzo Biochem, Inc. End labeled nucleotide probe
WO1989009221A1 (en) 1988-03-25 1989-10-05 University Of Virginia Alumni Patents Foundation Oligonucleotide n-alkylphosphoramidates
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
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
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
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
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
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. 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
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
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5623065A (en) 1990-08-13 1997-04-22 Isis Pharmaceuticals, Inc. Gapped 2' modified oligonucleotides
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5220007A (en) 1990-02-15 1993-06-15 The Worcester Foundation For Experimental Biology Method of site-specific alteration of RNA and production of encoded polypeptides
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
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
DE69032425T2 (de) 1990-05-11 1998-11-26 Microprobe Corp., Bothell, Wash. Teststreifen zum Eintauchen für Nukleinsäure-Hybridisierungsassays und Verfahren zur kovalenten Immobilisierung von Oligonucleotiden
GB9011454D0 (en) 1990-05-22 1990-07-11 Medical Res Council Polynucleotide amplification
CA2086334A1 (en) 1990-06-29 1991-12-30 The Regents Of The University Of Michigan Neurofibromatosis gene
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
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
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5614617A (en) 1990-07-27 1997-03-25 Isis Pharmaceuticals, Inc. Nuclease resistant, pyrimidine modified oligonucleotides that detect and modulate gene expression
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
PT98562B (pt) 1990-08-03 1999-01-29 Sanofi Sa Processo para a preparacao de composicoes que compreendem sequencias de nucleo-sidos com cerca de 6 a cerca de 200 bases resistentes a nucleases
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
AU662298B2 (en) 1990-09-20 1995-08-31 Gilead Sciences, Inc. Modified internucleoside linkages
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
EP0556301B1 (en) 1990-11-08 2001-01-10 Hybridon, Inc. Incorporation of multiple reporter groups on synthetic oligonucleotides
US5510473A (en) 1990-11-09 1996-04-23 The United States Of American As Represented By The Secretary Of Health And Human Services Cloning of the recA gene from thermus aquaticus YT-1
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
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
CA2122030C (en) 1991-10-24 1997-03-04 Muthiah Manoharan Derivatized oligonucleotides having improved uptake and other properties
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US5700922A (en) 1991-12-24 1997-12-23 Isis Pharmaceuticals, Inc. PNA-DNA-PNA chimeric macromolecules
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
GB9206016D0 (en) 1992-03-19 1992-04-29 Sandoz Ltd Improvements in or relating to organic compounds
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
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
IL105914A0 (en) 1992-06-04 1993-10-20 Univ California Methods and compositions for in vivo gene therapy
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
US5652355A (en) 1992-07-23 1997-07-29 Worcester Foundation For Experimental Biology Hybrid oligonucleotide phosphorothioates
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
DE69434469T2 (de) 1993-01-26 2006-06-14 Univ Pennsylvania Zusammensetzungen und verfahren zur verabreichung von genetischem material
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
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
ATE160572T1 (de) 1993-03-31 1997-12-15 Sanofi Sa Oligonucleotide mit amidverkettungen die phosphoesterverkettungen einsetzen
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
CA2178729A1 (en) 1993-12-09 1995-06-15 Eric B. Kmiec Compounds and methods for site-directed mutations in eukaryotic cells
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
US5658784A (en) * 1994-04-14 1997-08-19 Dana-Farber Cancer Institute, Inc. Nucleic acid encoding transcription factor p300 and uses of p300
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
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
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
US5962428A (en) 1995-03-30 1999-10-05 Apollon, Inc. Compositions and methods for delivery of genetic material
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5652356A (en) 1995-08-17 1997-07-29 Hybridon, Inc. Inverted chimeric and hybrid oligonucleotides
DE19608753C1 (de) 1996-03-06 1997-06-26 Medigene Gmbh Transduktionssystem und seine Verwendung
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
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
CA2287478C (en) 1997-04-14 2007-06-19 Richard J. Samulski Methods for increasing the efficiency of recombinant aav product
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
CA2263784A1 (en) 1998-03-23 1999-09-23 Megabios Corporation Dual-tagged proteins and their uses
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6491907B1 (en) 1998-11-10 2002-12-10 The University Of North Carolina At Chapel Hill Recombinant parvovirus vectors and method of making
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US6734291B2 (en) 1999-03-24 2004-05-11 Exiqon A/S Synthesis of [2.2.1]bicyclo nucleosides
CN102180924A (zh) 1999-05-04 2011-09-14 桑塔里斯制药公司 L-核糖-lna类似物
US20040192593A1 (en) 1999-07-26 2004-09-30 Baylor College Of Medicine Protease resistant ti-growth hormone releasing hormone
WO2001011034A2 (en) 1999-08-09 2001-02-15 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
US6287860B1 (en) 2000-01-20 2001-09-11 Isis Pharmaceuticals, Inc. Antisense inhibition of MEKK2 expression
ATE353361T1 (de) 2000-04-28 2007-02-15 Sangamo Biosciences Inc Gezielten modifikation der chromatinstruktur
US20020006397A1 (en) 2000-04-28 2002-01-17 Roberts Bruce L. In VIVO loading of MHC
ES2256265T3 (es) 2000-06-01 2006-07-16 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados.
AU2001268149B2 (en) 2000-06-01 2005-08-18 University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US7879540B1 (en) 2000-08-24 2011-02-01 Promega Corporation Synthetic nucleic acid molecule compositions and methods of preparation
EP1421177A4 (en) 2001-08-20 2006-06-07 Scripps Research Inst ZINC FINGER FASTENING DOMAINS FOR CNN
JP2005514005A (ja) 2001-09-04 2005-05-19 エクシコン エ/エス 新規のlna組成物およびその使用
ATE317916T1 (de) 2001-11-13 2006-03-15 Univ Pennsylvania Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode
EP1453547B1 (en) 2001-12-17 2016-09-21 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
WO2003072788A1 (en) 2002-02-21 2003-09-04 The Wistar Institute Of Anatomy And Biology Methods and compositions for reversibly controlling expression of target genes in cells
US7449561B1 (en) 2002-02-26 2008-11-11 City Of Hope Alterations in the dystrophin gene associated with sporadic dilated cardiomyopathy
US7074596B2 (en) 2002-03-25 2006-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse mRNA cap analogues
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
ATE485031T1 (de) 2002-06-28 2010-11-15 Protiva Biotherapeutics Inc Verfahren und vorrichtung zur herstellung von liposomen
CA2504593C (en) 2002-11-04 2016-08-09 Advisys, Inc. Synthetic muscle promoters with activities exceeding naturally occurring regulatory sequences in cardiac cells
US20070185042A1 (en) 2003-08-08 2007-08-09 The Brigham And Women's Hospital, Inc. President And Fellows Of Harvard College Sirna based methods for treating alzheimer's disease
WO2005028630A2 (en) 2003-09-19 2005-03-31 Sangamo Biosciences, Inc. Engineered zinc finger proteins for regulation of gene expression
ES2874298T3 (es) 2003-09-30 2021-11-04 Univ Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
US20070192880A1 (en) 2003-10-03 2007-08-16 University Of Rochester Horming response element binding transregulators
US8507277B2 (en) 2003-10-24 2013-08-13 Gencia Corporation Nonviral vectors for delivering polynucleotides
EP1766035B1 (en) 2004-06-07 2011-12-07 Protiva Biotherapeutics Inc. Lipid encapsulated interfering rna
EP1781593B1 (en) 2004-06-07 2011-12-14 Protiva Biotherapeutics Inc. Cationic lipids and methods of use
USRE47769E1 (en) 2004-06-28 2019-12-17 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US7728118B2 (en) 2004-09-17 2010-06-01 Promega Corporation Synthetic nucleic acid molecule compositions and methods of preparation
ES2428218T3 (es) 2005-04-07 2013-11-06 The Trustees Of The University Of Pennsylvania Cápsides de AAV rh48 modificadas, composiciones que las contienen y usos de las mismas
EP1777906A1 (en) 2005-06-09 2007-04-25 Matsushita Electric Industrial Co., Ltd. Amplitude error compensating apparatus and orthogonality error compensating apparatus
EP1937213B1 (en) 2005-07-27 2017-10-25 Protiva Biotherapeutics Inc. Systems and methods for manufacturing liposomes
KR20080031504A (ko) 2005-08-05 2008-04-08 미시간 스테이트 유니버시티 악티노바실러스 숙시노제네스의 c4-경로로부터 화학물질을생산하기 위해 악티노바실러스 숙시노제네스130z(atcc 55618)로부터 얻은 유전자
US7943374B2 (en) 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
HUE043492T2 (hu) 2005-08-23 2019-08-28 Univ Pennsylvania Módosított nukleozidokat tartalmazó RNS és eljárások az alkalmazására
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
AU2007269048A1 (en) 2006-07-05 2008-01-10 The Scripps Research Institute Chimeric zinc finger recombinases optimized for catalysis by directed evolution
WO2008070859A2 (en) 2006-12-07 2008-06-12 The Government Of The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Treatment of skin conditions by dickkopf1 (dkk1)
US20090018031A1 (en) 2006-12-07 2009-01-15 Switchgear Genomics Transcriptional regulatory elements of biological pathways tools, and methods
US8048998B2 (en) 2007-01-19 2011-11-01 Exiqon A/S Mediated cellular delivery of LNA oligonucleotides
WO2008151631A2 (en) 2007-06-15 2008-12-18 Exiqon A/S Use of short oligonucleotides for reagent redundancy experiments in rna functional analysis
HUE028662T2 (en) 2007-10-26 2016-12-28 Academisch Ziekenhuis Leiden Preparations and methods for controlling muscle disorders
EP2620161A1 (en) 2008-05-13 2013-07-31 University of Washington Diblock copolymers and polynucleotide complexes thereof for delivery into cells
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
EP2743265B1 (en) 2008-10-09 2017-03-15 Arbutus Biopharma Corporation Improved amino lipids and methods for the delivery of nucleic acids
EP2998402A1 (en) 2008-10-17 2016-03-23 Joule Unlimited Technologies, Inc. Ethanol production by microorganisms
TR201902952T4 (tr) 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
MX2011004242A (es) 2008-11-06 2011-07-20 Univ Washington Copolimeros multibloque.
US8969353B2 (en) 2008-11-07 2015-03-03 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
WO2010075424A2 (en) 2008-12-22 2010-07-01 The Regents Of University Of California Compositions and methods for downregulating prokaryotic genes
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
WO2010108126A2 (en) 2009-03-19 2010-09-23 Fate Therapeutics, Inc. Reprogramming compositions and methods of using the same
HRP20211619T1 (hr) 2009-06-10 2022-02-04 Arbutus Biopharma Corporation Poboljšana formulacija lipida
EP2281579A1 (en) 2009-08-05 2011-02-09 BioNTech AG Vaccine composition comprising 5'-Cap modified RNA
WO2011036640A2 (en) 2009-09-24 2011-03-31 Cellectis Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations
MX390753B (es) 2009-10-31 2025-03-21 Genesis Tech Limited Metodos para reprogramar celulas y usos de los mismos.
ES2696825T3 (es) 2009-12-10 2019-01-18 Univ Minnesota Modificación del ADN inducida por el efector TAL
EP2533629B1 (en) 2010-02-11 2018-11-28 Recombinetics, Inc. Methods and materials for producing transgenic artiodactyls
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US20130023033A1 (en) 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US20130145487A1 (en) 2010-05-12 2013-06-06 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
EP2571512B1 (en) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
IT1400425B1 (it) 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
EP2517731A1 (en) 2011-04-07 2012-10-31 Ludwig-Maximilians-Universität München Method of activating a target gene in a cell
JP5756171B2 (ja) 2011-05-12 2015-07-29 日立建機株式会社 建設機械
JP6184945B2 (ja) 2011-06-08 2017-08-23 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド mRNA送達のための脂質ナノ粒子組成物および方法
WO2013009525A1 (en) 2011-07-08 2013-01-17 Cellectis S.A. Method for increasing the efficiency of double-strand break-induced mutagenssis
CA2854819C (en) 2011-11-16 2022-07-19 Sangamo Biosciences, Inc. Modified dna-binding proteins and uses thereof
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
CN111893117B (zh) 2012-01-27 2024-06-04 比奥马林技术公司 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
WO2013143555A1 (en) 2012-03-26 2013-10-03 Biontech Ag Rna formulation for immunotherapy
US20130274129A1 (en) 2012-04-04 2013-10-17 Geneart Ag Tal-effector assembly platform, customized services, kits and assays
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
US9738879B2 (en) 2012-04-27 2017-08-22 Duke University Genetic correction of mutated genes
MY189533A (en) 2012-05-25 2022-02-16 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
US9890364B2 (en) 2012-05-29 2018-02-13 The General Hospital Corporation TAL-Tet1 fusion proteins and methods of use thereof
DK2858677T3 (da) 2012-06-08 2020-08-31 Ethris Gmbh Pulmonær levering af messenger rna
KR20230065381A (ko) 2012-07-25 2023-05-11 더 브로드 인스티튜트, 인코퍼레이티드 유도 dna 결합 단백질 및 게놈 교란 도구 및 이의 적용
US9567581B2 (en) 2012-08-07 2017-02-14 The General Hospital Corporation Selective reactivation of genes on the inactive X chromosome
EP2906602B1 (en) 2012-10-12 2019-01-16 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
WO2014065596A1 (en) 2012-10-23 2014-05-01 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
BR122019025681B1 (pt) 2012-11-01 2023-04-18 Factor Bioscience Inc Método para inserir uma sequência de ácido nucleico em uma localização segura de um genoma de uma célula
US20140140969A1 (en) 2012-11-20 2014-05-22 Sangamo Biosciences, Inc. Methods and compositions for muscular dystrophies
AU2013355214B2 (en) 2012-12-06 2017-06-15 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
CN119752887A (zh) 2012-12-12 2025-04-04 布罗德研究所有限公司 用于序列操纵的系统、方法和优化的指导组合物的工程化
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
CA2894668A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas systems and methods for altering expression of gene products in eukaryotic cells
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP4299741A3 (en) 2012-12-12 2024-02-28 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
EP4481048A3 (en) 2012-12-17 2025-02-26 President and Fellows of Harvard College Rna-guided human genome engineering
WO2014204578A1 (en) * 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
MX373959B (es) 2013-03-15 2020-07-13 Sarepta Therapeutics Inc Composición para usarse en el tratamiento de la distrofia muscular de duchenne (dmd).
WO2014145838A2 (en) 2013-03-15 2014-09-18 Britt Edward J Energy conversion device and method for making and using same
KR102210319B1 (ko) 2013-03-15 2021-02-01 더 제너럴 하스피탈 코포레이션 특정 게놈 좌위에 대한 유전적 및 후성적 조절 단백질의 rna-안내 표적화
US10760064B2 (en) * 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2014172470A2 (en) * 2013-04-16 2014-10-23 Whitehead Institute For Biomedical Research Methods of mutating, modifying or modulating nucleic acid in a cell or nonhuman mammal
AU2014265331B2 (en) 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
EP3309248B1 (en) 2013-05-29 2021-06-09 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
MY177814A (en) 2013-06-04 2020-09-23 Harvard College Rna-guided transcriptional regulation
US9267135B2 (en) 2013-06-04 2016-02-23 President And Fellows Of Harvard College RNA-guided transcriptional regulation
KR102733409B1 (ko) 2013-06-05 2024-11-21 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
ES2767318T3 (es) 2013-06-17 2020-06-17 Broad Inst Inc Suministro, modificación y optimización de sistemas, métodos y composiciones para generar modelos y actuar sobre enfermedades y trastornos de células posmitóticas
EP3011031B1 (en) 2013-06-17 2020-09-30 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
EP3011035B1 (en) 2013-06-17 2020-05-13 The Broad Institute, Inc. Assay for quantitative evaluation of target site cleavage by one or more crispr-cas guide sequences
DK3019619T3 (da) 2013-07-11 2021-10-11 Modernatx Inc Sammensætninger, der omfatter syntetiske polynukleotider, som koder for crispr-beslægtede proteiner, og syntetiske sgrna'er, og anvendelsesfremgangsmåder
US10421957B2 (en) 2013-07-29 2019-09-24 Agilent Technologies, Inc. DNA assembly using an RNA-programmable nickase
US9867885B2 (en) 2013-07-30 2018-01-16 Phaserx, Inc. Block copolymers
US20150044772A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. Crispr/cas system-based novel fusion protein and its applications in genome editing
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
US10265347B2 (en) 2013-08-29 2019-04-23 Norimasa Miura Biomolecular group related to cell anti-aging
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
WO2015048690A1 (en) 2013-09-27 2015-04-02 The Regents Of The University Of California Optimized small guide rnas and methods of use
PT3066201T (pt) 2013-11-07 2018-06-04 Massachusetts Inst Technology Métodos e composições relacionadas com crispr com arng reguladores
KR20250068794A (ko) 2013-12-12 2025-05-16 더 브로드 인스티튜트, 인코퍼레이티드 게놈 편집을 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료적 응용
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
WO2015089419A2 (en) 2013-12-12 2015-06-18 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
KR20160097327A (ko) 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 유전자 산물, 구조 정보 및 유도성 모듈형 cas 효소의 발현의 변경을 위한 crispr-cas 시스템 및 방법
CA2932479A1 (en) 2013-12-12 2015-06-18 The Rockefeller University Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
CN106103705A (zh) 2013-12-12 2016-11-09 布罗德研究所有限公司 核苷酸重复障碍中crispr‑cas系统的组合物和使用方法
EP3099786A1 (en) 2014-01-29 2016-12-07 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Trans-differentiation of differentiated cells
RU2016136977A (ru) 2014-02-18 2018-03-20 Дьюк Юниверсити Композиции для инактивации репликации вируса и способы их получения и применения
EP3116997B1 (en) 2014-03-10 2019-05-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
US11439712B2 (en) 2014-04-08 2022-09-13 North Carolina State University Methods and compositions for RNA-directed repression of transcription using CRISPR-associated genes
SG10201809157VA (en) 2014-04-18 2018-11-29 Editas Medicine Inc Crispr-cas-related methods, compositions and components for cancer immunotherapy
JP6930834B2 (ja) 2014-06-16 2021-09-01 ザ・ジョンズ・ホプキンス・ユニバーシティー H1プロモーターを用いるcrisprガイドrnaの発現のための組成物および方法
ES3047792T3 (en) 2014-07-14 2025-12-04 Univ California Crispr/cas transcriptional modulation
EP3169702A4 (en) 2014-07-14 2018-04-18 The Regents of The University of California A protein tagging system for in vivo single molecule imaging and control of gene transcription
WO2016022866A1 (en) 2014-08-07 2016-02-11 Agilent Technologies, Inc. Cis-blocked guide rna
JP6837429B2 (ja) 2014-08-11 2021-03-03 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム Crispr/cas9媒介遺伝子編集による筋ジストロフィーの予防
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
GB201418965D0 (enExample) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
MA40880A (fr) 2014-10-30 2017-09-05 Temple Univ Of The Commonwealth Éradication guidée par l'arn du virus jc humain et d'autres polyomavirus
WO2016081924A1 (en) 2014-11-20 2016-05-26 Duke University Compositions, systems and methods for cell therapy
IL301900A (en) 2014-11-21 2023-06-01 Regeneron Pharma Methods and compositions for targeted genetic modification using paired guide rnas
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
WO2016094867A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Protected guide rnas (pgrnas)
US10190106B2 (en) * 2014-12-22 2019-01-29 Univesity Of Massachusetts Cas9-DNA targeting unit chimeras
EP3245232B1 (en) 2015-01-12 2021-04-21 The Regents of The University of California Heterodimeric cas9 and methods of use thereof
EP3250693B2 (en) 2015-01-30 2023-12-20 The Regents of The University of California Protein delivery in primary hematopoietic cells
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
US9944912B2 (en) 2015-03-03 2018-04-17 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with altered PAM specificity
US20160281166A1 (en) 2015-03-23 2016-09-29 Parabase Genomics, Inc. Methods and systems for screening diseases in subjects
HK1244838A1 (zh) 2015-04-01 2018-08-17 Editas Medicine, Inc. 用於治疗杜兴氏肌肉营养不良症和贝克型肌肉营养不良症的与crispr/cas相关的方法及组合物
WO2016170348A2 (en) 2015-04-22 2016-10-27 Mina Therapeutics Limited Sarna compositions and methods of use
WO2016172727A1 (en) 2015-04-24 2016-10-27 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
WO2016187717A1 (en) 2015-05-26 2016-12-01 Exerkine Corporation Exosomes useful for genome editing
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
KR102840885B1 (ko) 2015-06-18 2025-07-30 더 브로드 인스티튜트, 인코퍼레이티드 표적외 효과를 감소시키는 crispr 효소 돌연변이
WO2017015637A1 (en) 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
EP3325464B1 (en) 2015-07-24 2022-04-20 Basf Se Pyridine compounds useful for combating phytopathogenic fungi
US20200123533A1 (en) 2015-07-31 2020-04-23 The Trustees Of Columbia University In The City Of New York High-throughput strategy for dissecting mammalian genetic interactions
EA201890565A1 (ru) 2015-08-25 2019-04-30 Дьюк Юниверсити Композиции и способы улучшения специфичности в геномной инженерии с применением рнк-направляемых эндонуклеаз
EP3350315A4 (en) 2015-09-18 2019-07-17 The Regents of The University of California METHOD FOR THE AUTOCATALYTIC GENOMIC PROCESSING AND NEUTRALIZATION OF AUTO CATALYTIC GENOMIC PROCESSING AND COMPOSITIONS THEREFOR
EP3332008A4 (en) 2015-09-23 2019-01-16 Université Laval CHANGE OF THE DYSTROPHINE GENE AND USES THEREOF
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
EP3365356B1 (en) 2015-10-23 2023-06-28 President and Fellows of Harvard College Nucleobase editors and uses thereof
EP3368689B1 (en) 2015-10-28 2020-06-17 The Broad Institute, Inc. Composition for modulating immune responses by use of immune cell gene signature
EP3368063B1 (en) 2015-10-28 2023-09-06 Vertex Pharmaceuticals Inc. Materials and methods for treatment of duchenne muscular dystrophy
WO2017081288A1 (en) 2015-11-11 2017-05-18 Lonza Ltd Crispr-associated (cas) proteins with reduced immunogenicity
US11306308B2 (en) 2015-11-13 2022-04-19 Massachusetts Institute Of Technology High-throughput CRISPR-based library screening
WO2017095967A2 (en) 2015-11-30 2017-06-08 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
CN109312335B (zh) 2016-01-11 2023-10-24 斯坦福大学托管董事会 嵌合蛋白和调节基因表达的方法
EP3199632A1 (en) 2016-01-26 2017-08-02 ACIB GmbH Temperature-inducible crispr/cas system
EP3413908B1 (en) 2016-02-11 2024-06-19 The Regents of The University of California Methods and compositions for modifying a mutant dystrophin gene in a cell's genome
WO2017165859A1 (en) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
AU2017248656B2 (en) 2016-04-15 2023-07-27 The Trustees Of The University Of Pennsylvania Novel AAV8 mutant capsids and compositions containing same
JP7079733B2 (ja) 2016-04-15 2022-06-02 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル β-サルコグリカン及びマイクロRNA-29のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療
EP3452498B1 (en) 2016-05-05 2023-07-05 Duke University Crispr/cas-related compositions for treating duchenne muscular dystrophy
US11278550B2 (en) 2016-05-17 2022-03-22 Duke University Compositions and methods for the treatment of Prader-Willi syndrome
US20170362635A1 (en) 2016-06-20 2017-12-21 University Of Washington Muscle-specific crispr/cas9 editing of genes
WO2018002812A1 (en) 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of myotonic dystrophy type 1 (dm1) and other related disorders
JP2019525742A (ja) 2016-06-30 2019-09-12 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマー
SG10202109385QA (en) 2016-07-05 2021-10-28 Univ Johns Hopkins Crispr/cas9-based compositions and methods for treating retinal degenerations
AU2017295898B2 (en) 2016-07-15 2023-08-17 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells
CN109715218A (zh) 2016-07-18 2019-05-03 嘉安生物治疗有限责任公司 用于治疗心脏疾病的组合物和方法
US20190235624A1 (en) 2016-07-19 2019-08-01 Supereye, Inc. Systems and methods for predictive visual rendering
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
JP7231935B2 (ja) 2016-08-03 2023-03-08 プレジデント アンド フェローズ オブ ハーバード カレッジ アデノシン核酸塩基編集因子およびそれらの使用
US20190194633A1 (en) 2016-08-10 2019-06-27 Duke University Compositions, systems and methods for programming immune cell function through targeted gene regulation
EP4485466A3 (en) 2016-08-17 2025-04-02 The Broad Institute Inc. Novel crispr enzymes and systems
JP7050215B2 (ja) 2016-08-19 2022-04-08 ツールゲン インコーポレイテッド 人工的に操作された血管新生調節系
AU2017313912B2 (en) 2016-08-19 2024-01-04 Whitehead Institute For Biomedical Research Methods of editing DNA methylation
EP3500677A4 (en) 2016-08-20 2020-04-01 Avellino Lab USA, Inc. Single guide rna, crispr/cas9 systems, and methods of use thereof
CA3037635A1 (en) 2016-09-23 2018-03-29 Universite Laval Methods of modifying the dystrophin gene and restoring dystrophin expression and uses thereof
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
EP3532616A1 (en) 2016-10-28 2019-09-04 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus
EP3545090A1 (en) 2016-11-28 2019-10-02 The Board of Regents of The University of Texas System Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing
CA3045335A1 (en) 2016-12-01 2018-06-07 Universite Laval Crispr-based treatment of friedreich ataxia
CA3046220A1 (en) 2016-12-08 2018-06-14 The Board Of Regents Of The University Of Texas System Dmd reporter models containing humanized duschene muscular dystrophy mutations
JOP20190166A1 (ar) 2017-01-05 2019-07-02 Univ Texas استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي
CN110612113B (zh) 2017-02-07 2024-03-26 加利福尼亚大学董事会 单倍体机能不全的基因疗法
JP2018143239A (ja) 2017-03-01 2018-09-20 エリクサジェン,エルエルシー. 多能性幹細胞を所望の細胞型へ効率的に分化する方法
US20210322577A1 (en) 2017-03-03 2021-10-21 Flagship Pioneering Innovations V, Inc. Methods and systems for modifying dna
US10687520B2 (en) 2017-03-07 2020-06-23 The Board Of Regents Of The University Of Texas System Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44
WO2018162702A1 (en) 2017-03-10 2018-09-13 Institut National De La Sante Et De La Recherche Medicale (Inserm) Nuclease fusions for enhancing genome editing by homology-directed transgene integration
EP3600428A4 (en) 2017-03-23 2020-08-26 Dnarx SYSTEMS AND METHODS FOR THE EXPRESSION OF NUCLEIC ACIDS IN VIVO
WO2018179578A1 (ja) 2017-03-30 2018-10-04 国立大学法人京都大学 ゲノム編集によるエクソンスキッピング誘導方法
KR20240155360A (ko) 2017-04-12 2024-10-28 더 브로드 인스티튜트, 인코퍼레이티드 신규 타입 vi crispr 오르소로그 및 시스템
US20180305719A1 (en) 2017-04-19 2018-10-25 The Board Of Trustees Of The University Of Illinois Vectors For Integration Of DNA Into Genomes And Methods For Altering Gene Expression And Interrogating Gene Function
WO2018208998A1 (en) 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9
EP3420811A1 (en) 2017-06-29 2019-01-02 Paris Sciences et Lettres - Quartier Latin Non-human model for neurofibromatosis type 1
CA3069296A1 (en) 2017-07-07 2019-01-10 Toolgen Incorporated Target-specific crispr mutant
WO2019014122A1 (en) 2017-07-08 2019-01-17 The Brigham And Women's Hospital, Inc. METHODS FOR ENHANCING ANTIANGIOGENIC THERAPY AND IMMUNOTHERAPY
CA3106738C (en) 2017-07-25 2025-02-11 Shanghai Institutes For Biological Sciences, Chinese Academy Of Sciences METHOD FOR MODULATING RNA SPLICING BY INDUCING A BASE MUTATION AT A SPLICE SITE OR A BASE SUBSTITUTION IN A POLYPYRIMIDIN REGION
WO2019023291A2 (en) 2017-07-25 2019-01-31 Dana-Farber Cancer Institute, Inc. COMPOSITIONS AND METHODS FOR PRODUCTION AND DECODING OF GUIDE RNA LIBRARIES AND USES THEREOF
EP3441461A1 (en) 2017-08-11 2019-02-13 Baylor College of Medicine Cd1d-restricted nkt cells as a platform for off-the-shelf cancer immunotherapy
WO2019036599A1 (en) 2017-08-18 2019-02-21 The Board Of Regents Of The University Of Texas System EXON DELETION CORRECTION OF MUTATIONS OF DUCHENNE MUSCLE DYSTROPHY IN ACTINE DYSTROPHINE BINDING DOMAIN 1 Using a GENOME CRISPR EDITION
WO2019046755A1 (en) 2017-08-31 2019-03-07 Sarepta Therapeutics, Inc. METHODS FOR TREATING MUSCLE DYSTROPHY
WO2019067786A1 (en) 2017-09-29 2019-04-04 The Regents Of The University Of California MULTI-ARM POLYROTAXANE PLATFORM FOR PROTECTED ADMINISTRATION OF NUCLEIC ACIDS
US11987790B2 (en) 2017-10-17 2024-05-21 Massachusetts Institute Of Technology Methods for high-resolution genome-wide functional dissection of transcriptional regulatory regions
JP2021501572A (ja) 2017-10-19 2021-01-21 キュアバック アーゲー 新規な人工核酸分子
GB2610100B (en) 2017-10-23 2023-08-16 Stoke Therapeutics Inc Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases
US20210032622A1 (en) 2017-11-09 2021-02-04 Crispr Therapeutics Ag Self-inactivating (sin) crispr/cas or crispr/cpf1 systems and uses thereof
US12018255B2 (en) 2017-12-08 2024-06-25 University Of Connecticut Compositions and methods for treating disorders of genomic imprinting
WO2019118902A2 (en) 2017-12-15 2019-06-20 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for inhibiting t cell exhaustion
BR112020012064A2 (pt) 2017-12-22 2020-12-01 KWS SAAT SE & Co. KGaA regulação transcricional direcionada usando fatores de transcrição sintéticos
CN111684069A (zh) 2017-12-22 2020-09-18 G+Flas生命科学有限公司 嵌合基因组工程分子和方法
US12031130B2 (en) 2017-12-28 2024-07-09 Takeda Pharmaceutical Company Limited Composition for modifying target gene
EP3735462A1 (en) 2018-01-05 2020-11-11 The Board of Regents of The University of Texas System Therapeutic crispr/cas9 compositions and methods of use
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOMIC ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYOTES
CA3091267A1 (en) 2018-02-23 2019-08-29 Pioneer Hi-Bred International, Inc. Novel cas9 orthologs
US20210254049A1 (en) 2018-04-20 2021-08-19 Cellino Biotech, Inc. Directed cell fate specification and targeted maturation
JP7498499B2 (ja) 2018-05-03 2024-06-12 プレジデント アンド フェローズ オブ ハーバード カレッジ 心臓、骨格筋、及び筋幹細胞におけるインビボ相同組換え修復
US20210299174A1 (en) 2018-05-30 2021-09-30 M2X2 Therapeutics, Inc. Cell therapy
WO2020018918A1 (en) 2018-07-19 2020-01-23 The Board Of Trustees Of The University Of Illinois Methods for exon skipping and gene knockout using base editors
KR20210069692A (ko) 2018-10-02 2021-06-11 상가모 테라퓨틱스, 인코포레이티드 조작된 유전자 조정제
WO2020132226A1 (en) 2018-12-20 2020-06-25 Ohio State Innovation Foundation Compositions and methods for reprogramming diseased musculoskeletal cells
BR102019009665A2 (pt) 2018-12-21 2022-02-08 Jacques P. Tremblay Modificação da proteína precursora beta amiloide (app) através da edição de base usando o sistema crispr/cas9
EP3921417A4 (en) 2019-02-04 2022-11-09 The General Hospital Corporation VARIANTS OF ADENINE DNA BASES WITH REDUCED OFF-TARGET RNA EDIT
CN114096666B (zh) 2019-02-13 2025-03-18 比姆医疗股份有限公司 治疗血红素病变的组合物和方法
WO2020210776A1 (en) 2019-04-12 2020-10-15 Duke University Crispr/cas-based base editing composition for restoring dystrophin function
JP2022529424A (ja) 2019-04-14 2022-06-22 デューク ユニバーシティ ジストロフィン機能を修復するためのCRISPR/Casをベースにしたゲノム編集組成物
KR20210151916A (ko) 2019-04-14 2021-12-14 듀크 유니버시티 뒤시엔느 근육 이영양증의 치료를 위한 aav 벡터-매개된 큰 돌연변이 핫스팟의 결실
JP2022537384A (ja) 2019-06-20 2022-08-25 セルジーン コーポレーション ベネトクラクス、ギルテリチニブ、ミドスタウリン、または白血病もしくは骨髄異形成症候群を治療するための他の化合物との組み合わせにおけるアザシチジン
WO2021026516A1 (en) 2019-08-08 2021-02-11 Duke University High-throughput screening platform for engineering next-generation gene therapy vectors
CN114599403A (zh) 2019-08-19 2022-06-07 杜克大学 通过基于crispr/cas9的转录激活物获得的骨骼肌成肌细胞祖细胞谱系的特化
CN114555805A (zh) 2019-08-19 2022-05-27 杜克大学 用于鉴定细胞类型命运特化的调控物的组合物和方法
US20230060409A1 (en) 2019-09-20 2023-03-02 The Uab Research Foundation Methods of Treatment of Neurofibromatosis Type 1 (NF1) and NF-1 Mediated Conditions and Compositions for Use in Such Methods
EP4038204A4 (en) 2019-10-02 2023-09-13 Duke University EPIGENETIC MODULATION OF GENOMIC TARGETS TO REGULATE THE EXPRESSION OF GENES ASSOCIATED WITH PWS
US20230032846A1 (en) 2019-12-03 2023-02-02 Duke University Systems and methods for lipid nanoparticle delivery of gene editing machinery
US20230349888A1 (en) 2020-04-27 2023-11-02 Duke University A high-throughput screening method to discover optimal grna pairs for crispr-mediated exon deletion
WO2021222268A1 (en) 2020-04-27 2021-11-04 Duke University Crispr/cas9 therapies for correcting duchenne muscular dystrophy by targeted genomic integration
WO2021222328A1 (en) 2020-04-27 2021-11-04 Duke University Targeted genomic integration to restore neurofibromin coding sequence in neurofibromatosis type 1 (nf1)
US20230348870A1 (en) 2020-04-27 2023-11-02 Duke University Gene editing of satellite cells in vivo using aav vectors encoding muscle-specific promoters
US20230159927A1 (en) 2020-05-08 2023-05-25 Duke University Chromatin remodelers to enhance targeted gene activation
EP4200406A1 (en) 2020-08-21 2023-06-28 Julius-Maximilians-Universität Würzburg Modified lymphocytes
WO2022087321A1 (en) 2020-10-21 2022-04-28 Duke University Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
EP4244344A4 (en) 2020-11-11 2024-10-16 The Trustees Of Columbia University In The City Of New York MULTIPLEX EPIGENOME EDITION
US20240026352A1 (en) 2020-11-13 2024-01-25 Duke University Targeted gene regulation of human immune cells with crispr-cas systems
WO2022133062A1 (en) 2020-12-16 2022-06-23 Epicrispr Biotechnologies, Inc. Systems and methods for engineering characteristics of a cell
WO2022187288A2 (en) 2021-03-01 2022-09-09 Duke University Systems and methods for genome-wide annotation of gene regulatory elements linked to cell fitness
US20240252684A1 (en) 2021-07-30 2024-08-01 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
CA3227103A1 (en) 2021-07-30 2023-02-02 Matthew P. GEMBERLING Compositions and methods for modulating expression of frataxin (fxn)
WO2023137471A1 (en) 2022-01-14 2023-07-20 Tune Therapeutics, Inc. Compositions, systems, and methods for programming t cell phenotypes through targeted gene activation
EP4463549A2 (en) 2022-01-14 2024-11-20 Tune Therapeutics, Inc. Compositions, systems, and methods for programming t cell phenotypes through targeted gene repression
US20240058425A1 (en) 2022-03-08 2024-02-22 Duke University Systems and methods for genome-wide annotation of gene regulatory elements linked to cell fitness
JP2025524469A (ja) 2022-06-24 2025-07-30 チューン セラピューティクス インコーポレイテッド 標的を定めた遺伝子抑制を介して低密度リポタンパク質を低減するための組成物、系、及び方法
CA3261865A1 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS
WO2024040253A1 (en) 2022-08-18 2024-02-22 Duke University Epigenetic modulation of genomic targets to control expression of pws-associated genes
AU2023325407A1 (en) 2022-08-19 2025-02-20 Tune Therapeutics, Inc. Compositions, systems, and methods for regulation of hepatitis b virus through targeted gene repression
EP4590821A2 (en) 2022-09-19 2025-07-30 Tune Therapeutics, Inc. Compositions, systems, and methods for modulating t cell function
WO2024081937A2 (en) 2022-10-13 2024-04-18 Duke University Cas12a fusion proteins and methods of using same

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