MX363017B - Métodos y productos para la expresión de proteínas en células. - Google Patents

Métodos y productos para la expresión de proteínas en células.

Info

Publication number
MX363017B
MX363017B MX2015005346A MX2015005346A MX363017B MX 363017 B MX363017 B MX 363017B MX 2015005346 A MX2015005346 A MX 2015005346A MX 2015005346 A MX2015005346 A MX 2015005346A MX 363017 B MX363017 B MX 363017B
Authority
MX
Mexico
Prior art keywords
methods
cells
products
proteins
nucleic acids
Prior art date
Application number
MX2015005346A
Other languages
English (en)
Spanish (es)
Other versions
MX2015005346A (es
Inventor
Angel Matthew
Rohde Christopher
Original Assignee
Factor Bioscience Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Factor Bioscience Inc filed Critical Factor Bioscience Inc
Publication of MX2015005346A publication Critical patent/MX2015005346A/es
Publication of MX363017B publication Critical patent/MX363017B/es

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    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C—CHEMISTRY; METALLURGY
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
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    • C12N9/14—Hydrolases (3)
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    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
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    • C12Y—ENZYMES
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    • C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
    • C12Y301/21004—Type II site-specific deoxyribonuclease (3.1.21.4)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
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    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
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    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/5014—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics for testing toxicity
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    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
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    • C—CHEMISTRY; METALLURGY
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    • C07K—PEPTIDES
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    • C12N2800/00—Nucleic acids vectors
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  • Biochemistry (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
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  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
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  • Neurology (AREA)
  • Urology & Nephrology (AREA)
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  • Gastroenterology & Hepatology (AREA)
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MX2015005346A 2012-11-01 2013-11-01 Métodos y productos para la expresión de proteínas en células. MX363017B (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201261721302P 2012-11-01 2012-11-01
US201361785404P 2013-03-14 2013-03-14
US201361842874P 2013-07-03 2013-07-03
PCT/US2013/068118 WO2014071219A1 (en) 2012-11-01 2013-11-01 Methods and products for expressing proteins in cells

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MX2015005346A MX2015005346A (es) 2015-07-14
MX363017B true MX363017B (es) 2019-03-04

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MX2015005346A MX363017B (es) 2012-11-01 2013-11-01 Métodos y productos para la expresión de proteínas en células.
MX2019002498A MX2019002498A (es) 2012-11-01 2015-04-27 Metodos y productos para la expresion de proteinas en celulas.

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MX2019002498A MX2019002498A (es) 2012-11-01 2015-04-27 Metodos y productos para la expresion de proteinas en celulas.

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US (20) US9447395B2 (enExample)
EP (2) EP3786298A1 (enExample)
JP (5) JP6510416B2 (enExample)
KR (4) KR20230154283A (enExample)
CN (1) CN104769112A (enExample)
AU (3) AU2013337651B2 (enExample)
BR (2) BR122019025681B1 (enExample)
CA (2) CA2890110C (enExample)
MX (2) MX363017B (enExample)
RU (2) RU2711249C2 (enExample)
WO (1) WO2014071219A1 (enExample)

Families Citing this family (130)

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Publication number Priority date Publication date Assignee Title
DE10347710B4 (de) 2003-10-14 2006-03-30 Johannes-Gutenberg-Universität Mainz Rekombinante Impfstoffe und deren Verwendung
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2857499A1 (en) 2010-10-01 2015-04-08 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
LT2714071T (lt) 2011-05-24 2019-10-10 Biontech Rna Pharmaceuticals Gmbh Individualizuotos vakcinos nuo vėžio
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
PT3682905T (pt) 2011-10-03 2022-04-07 Modernatx Inc Nucleósidos, nucleótidos e ácidos nucleicos modificados e respetivas utilizações
ES2923757T3 (es) 2011-12-16 2022-09-30 Modernatx Inc Composiciones de ARNm modificado
WO2013151664A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
WO2013143555A1 (en) 2012-03-26 2013-10-03 Biontech Ag Rna formulation for immunotherapy
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
CA2885216A1 (en) 2012-09-17 2014-03-20 The Research Institute At Nationwide Children's Hospital Compositions and methods for treating amyotrophic lateral sclerosis
CA2890110C (en) 2012-11-01 2023-05-02 Factor Bioscience Inc. Methods and products for expressing proteins in cells
PT2922554T (pt) 2012-11-26 2022-06-28 Modernatx Inc Arn modificado nas porções terminais
AU2013351542B2 (en) 2012-11-28 2018-08-09 BioNTech SE Individualized vaccines for cancer
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2014180490A1 (en) 2013-05-10 2014-11-13 Biontech Ag Predicting immunogenicity of t cell epitopes
WO2014197748A2 (en) 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
RS63942B1 (sr) * 2013-08-27 2023-02-28 Res Inst Nationwide Childrens Hospital Proizvodi i postupci za lečenje amiotrofične lateralne skleroze
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
US9725719B2 (en) 2013-11-05 2017-08-08 The Research Institute At Nationwide Children's Hospital Compositions and methods for inhibiting NF-κB and SOD-1 to treat amyotrophic lateral sclerosis
CN111218447B (zh) 2013-11-07 2024-10-11 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
AU2015229299A1 (en) * 2014-03-12 2016-09-22 Precision Biosciences, Inc. Dystrophin gene exon deletion using engineered nucleases
HRP20220070T1 (hr) 2014-04-23 2022-04-01 Modernatx, Inc. Cjepiva nukleinske kiseline
WO2015168800A1 (en) 2014-05-09 2015-11-12 UNIVERSITé LAVAL Prevention and treatment of alzheimer's disease by genome editing using the crispr/cas system
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016045732A1 (en) 2014-09-25 2016-03-31 Biontech Rna Pharmaceuticals Gmbh Stable formulations of lipids and liposomes
US10370673B2 (en) 2014-09-26 2019-08-06 Purecircle Sdn Bhd Single nucleotide polymorphism (SNP) markers for stevia
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
PL3223846T3 (pl) * 2014-11-26 2024-03-11 Huvepharma, Inc. Kompozycje adiuwantowe i powiązane sposoby
US10195261B2 (en) 2014-11-26 2019-02-05 VaxLiant, LLC Adjuvant compositions and related methods
CA2969619A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
WO2016128060A1 (en) 2015-02-12 2016-08-18 Biontech Ag Predicting t cell epitopes useful for vaccination
US11241505B2 (en) * 2015-02-13 2022-02-08 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
WO2016154432A1 (en) 2015-03-24 2016-09-29 VaxLiant, LLC Adjuvant compositions and related methods
CN107787367B (zh) 2015-04-06 2021-10-26 里兰斯坦福初级大学理事会 用于crispr/cas介导的基因调控的化学修饰的引导rna
ES3009026T3 (en) 2015-07-22 2025-03-25 Univ Duke High-throughput screening of regulatory element function with epigenome editing technologies
WO2017017016A1 (en) * 2015-07-25 2017-02-02 Frost Habib A system, device and a method for providing a therapy or a cure for cancer and other pathological states
KR20240132120A (ko) 2015-08-25 2024-09-02 듀크 유니버시티 Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법
WO2017059902A1 (en) 2015-10-07 2017-04-13 Biontech Rna Pharmaceuticals Gmbh 3' utr sequences for stabilization of rna
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
DK3362461T3 (da) 2015-10-16 2022-05-09 Modernatx Inc Mrna-cap-analoger med modificeret phosphatbinding
US11866754B2 (en) 2015-10-16 2024-01-09 Modernatx, Inc. Trinucleotide mRNA cap analogs
EP3364949A4 (en) 2015-10-22 2019-07-31 ModernaTX, Inc. CANCER VACCINES
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
EP3384038B1 (en) 2015-11-23 2024-07-24 The Regents of The University of Colorado, A Body Corporate Methods and compositions for reprogramming cells
US12214054B2 (en) 2015-11-30 2025-02-04 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
CN105494263B (zh) * 2015-12-25 2018-11-30 哈尔滨医科大学 一种产生ho-1/app/psen1三转基因阿尔茨海默病小鼠模型的方法
EP3411078A1 (en) * 2016-02-02 2018-12-12 Crispr Therapeutics AG Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
WO2017141109A1 (en) * 2016-02-18 2017-08-24 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
US11530253B2 (en) 2016-02-25 2022-12-20 The Children's Medical Center Corporation Customized class switch of immunoglobulin genes in lymphoma and hybridoma by CRISPR/CAS9 technology
US11083799B2 (en) 2016-03-16 2021-08-10 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
KR102312903B1 (ko) * 2016-03-31 2021-10-15 에트리스 게엠베하 신규의 최소 utr 서열
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
WO2017181107A2 (en) * 2016-04-16 2017-10-19 Ohio State Innovation Foundation Modified cpf1 mrna, modified guide rna, and uses thereof
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
WO2018013840A1 (en) 2016-07-13 2018-01-18 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
IL264439B2 (en) * 2016-08-17 2024-08-01 Factor Bioscience Inc non-viral, cell-free composition comprising a synthetic messenger RNA (MRNA) encoding a gene-editing protein for use in treating cancer, and a synthetic RNA encoding a gene-editing protein for use in treatment
CN106370765A (zh) * 2016-08-23 2017-02-01 国家烟草质量监督检验中心 一种基于反相色谱飞行时间质谱的喉癌尿液差异代谢物的测定筛选方法
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CN106381310B (zh) * 2016-08-31 2020-02-04 武汉华美生物工程有限公司 一种用t7噬菌体rna聚合酶和t7启动子系统在哺乳动物细胞表达蛋白的方法
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
CN110214005A (zh) * 2016-11-11 2019-09-06 DNALite治疗学公司 用于基因治疗的结构和方法
KR101908593B1 (ko) * 2016-12-07 2018-10-16 연세대학교 산학협력단 Cftr 유전자 소실 유도 가이드 rna, cftr 변이를 갖는 t84 세포 및 이의 용도
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018154418A1 (en) * 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders
EP3589290A4 (en) * 2017-02-28 2020-12-30 Arcturus Therapeutics, Inc. Translatable molecules and synthesis thereof
US11168319B1 (en) * 2017-02-28 2021-11-09 Inari Agriculture Technology, Inc. Plant cell culture
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
CN107475255B (zh) * 2017-03-21 2021-03-26 河北医科大学第二医院 一种基因载体介导的基于CRISPR/Cas9基因编辑系统的sgRNA及其用途
IL306092A (en) 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
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
WO2018224166A1 (en) 2017-06-09 2018-12-13 Biontech Rna Pharmaceuticals Gmbh Methods for predicting the usefulness of disease specific amino acid modifications for immunotherapy
WO2018227432A1 (en) * 2017-06-14 2018-12-20 Wuhan Institute Of Virology, Chinese Academy Of Sciences Cd2-associated protein (cd2ap) and its interactive proteins
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base 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
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
US11634722B2 (en) 2018-01-22 2023-04-25 Inari Agriculture Technology, Inc. Plant gene editing systems, methods, and compositions
CA3095125A1 (en) * 2018-03-27 2019-10-03 Factor Bioscience Inc. Nucleic acid-based therapeutics
WO2019204643A2 (en) 2018-04-18 2019-10-24 Altius Institute For Biomedical Sciences Animal pathogen-derived polypeptides and uses thereof for genetic engineering
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
CN108624592B (zh) * 2018-05-24 2021-08-17 苏州大学张家港工业技术研究院 针对DJ-1基因编辑的sgRNA筛选及其载体与应用
US20210268086A1 (en) * 2018-06-27 2021-09-02 Modernatx, Inc. Personalized cancer vaccine epitope selection
WO2020006126A1 (en) 2018-06-27 2020-01-02 Altius Institute For Biomedical Sciences Nucleic acid binding domains and methods of use thereof
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
CN108949830B (zh) * 2018-08-03 2021-11-26 福州大学 一种在鱼类中实现基因组编辑、精确定点基因敲入的方法
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
CN109628404B (zh) * 2018-12-18 2020-04-28 浙江大学 猪皮下脂肪前体细胞永生化细胞系的构建方法及用途
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏（ｄｕｃｈｅｎｎｅ）肌肉失養症之大型突變熱點之ａａｖ載體介導之刪除
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN110200973A (zh) * 2019-06-19 2019-09-06 南京市儿童医院 Dna依赖性蛋白激酶特异性抑制剂在制备防治肾纤维化药物中的用途
CA3145425A1 (en) 2019-07-03 2021-01-07 Factor Bioscience Inc. Cationic lipids and uses thereof
US11746354B2 (en) 2019-07-19 2023-09-05 Inari Agriculture Technology, Inc. Homology dependent repair genome editing
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
CN110699381A (zh) * 2019-09-17 2020-01-17 合肥瑞灵生物科技有限公司 地中海贫血病基因治疗载体构建方法及其用途
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
US20210290757A1 (en) 2020-03-23 2021-09-23 Avectas Limited Engineering of dendritic cells for generation of vaccines against sars-cov-2
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
CN113151181B (zh) * 2021-04-14 2022-11-29 中山大学附属第七医院(深圳) 一种脂肪干细胞重编程为神经元的方法及负载该神经元的细胞适应性水凝胶修复脊髓损伤
CN113373214B (zh) * 2021-06-18 2024-03-29 中国人民解放军军事科学院军事医学研究院 Cd30在诊断脑神经相关疾病中的用途
WO2023039586A1 (en) 2021-09-10 2023-03-16 Agilent Technologies, Inc. Guide rnas with chemical modification for prime editing
CN114150017B (zh) * 2021-11-30 2023-05-16 长江大学 一种调节猪未成熟睾丸支持细胞乳酸分泌水平的方法及应用
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression
WO2024032678A1 (zh) * 2022-08-11 2024-02-15 益杰立科(上海)生物科技有限公司 一种表观编辑靶点的方法及用途
WO2025084896A1 (ko) * 2023-10-18 2025-04-24 연세대학교 산학협력단 신경세포 분화 유도용 재조합 벡터 및 이를 포함하는 퇴행성 뇌신경 질환의 예방 또는 치료용 약학적 조성물
CN118667815B (zh) * 2024-07-16 2025-02-14 宁夏大学 一种靶向prdm1改善牛子宫内膜炎的核苷酸序列及其应用

Family Cites Families (123)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3539465A (en) 1968-10-08 1970-11-10 Ncr Co Encapsulation of hydrophilic liquid-in-oil emulsions
US5843780A (en) 1995-01-20 1998-12-01 Wisconsin Alumni Research Foundation Primate embryonic stem cells
US6743771B2 (en) * 1995-12-29 2004-06-01 Novactyl, Inc. Methods and compositions for controlling protein assembly or aggregation
JP2001508302A (ja) 1997-01-10 2001-06-26 ライフ テクノロジーズ，インコーポレイテッド 胚性幹細胞血清置換
EP1021549A2 (en) 1997-09-19 2000-07-26 Sequitur, Inc. SENSE mRNA THERAPY
US20110171185A1 (en) 1999-06-30 2011-07-14 Klimanskaya Irina V Genetically intact induced pluripotent cells or transdifferentiated cells and methods for the production thereof
EP2292632A3 (en) 2000-09-26 2012-07-25 Idera Pharmaceuticals, Inc. Modulation of immunostimulatory activity of immunostimulatory oligonucleotide analogs by positional chemical changes
WO2003018767A2 (en) 2001-08-27 2003-03-06 Advanced Cell Technology, Inc. Trans-differentiation and re-differentiation of somatic cells and production of cells for cell therapies
WO2003027277A1 (en) 2001-09-21 2003-04-03 Japan Science And Technology Corporation Method of screening reporgramming factor, reprogramming factor screened by the method, method of using the reprogramming factor, method of differentiating undifferentiated fused cells and method of constructing cell, tissues and organs
US20050053588A1 (en) 2001-10-18 2005-03-10 Li Yin Conversion of liver stem and progenitor cells to pancreatic functional cells
US7276489B2 (en) 2002-10-24 2007-10-02 Idera Pharmaceuticals, Inc. Modulation of immunostimulatory properties of oligonucleotide-based compounds by optimal presentation of 5′ ends
GB0202149D0 (en) 2002-01-30 2002-03-20 Univ Edinburgh Pluripotency determining factors and uses thereof
JP4579911B2 (ja) 2003-06-03 2010-11-10 アイシス・ファーマシューティカルズ・インコーポレイテッド スルビビン発現の調節
US7682828B2 (en) 2003-11-26 2010-03-23 Whitehead Institute For Biomedical Research Methods for reprogramming somatic cells
BRPI0514778B1 (pt) 2004-09-08 2018-07-10 Wisconsin Alumni Research Foundation Meio para cultivar células tronco embrionárias
BRPI0514641A (pt) 2004-09-08 2008-06-17 Wisconsin Alumni Res Found cultivação das células tronco embriÈnica humana
CA2595695A1 (en) * 2005-01-25 2006-08-03 Cell Therapeutics, Inc. Biologically active protein conjugates comprising (nn[s/t]) peptide repeats and having increased plasma half-life
JP5147692B2 (ja) * 2005-07-13 2013-02-20 ノボ ノルディスク ヘルス ケア アーゲー 治療用タンパク質生成のための宿主細胞タンパク質ノックアウト細胞
EP1913149A4 (en) * 2005-07-26 2009-08-05 Sangamo Biosciences Inc TARGETED INTEGRATION AND EXPRESSION OF EXOGENOUS NUCLEIC ACID SEQUENCES
US8323666B2 (en) 2005-08-01 2012-12-04 Allergan, Inc. Botulinum toxin compositions
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
PL2578685T3 (pl) 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
WO2007026353A2 (en) 2005-08-29 2007-03-08 Technion Research & Development Foundation Ltd. Media for culturing stem cells
EP2206724A1 (en) 2005-12-13 2010-07-14 Kyoto University Nuclear reprogramming factor
US8129187B2 (en) 2005-12-13 2012-03-06 Kyoto University Somatic cell reprogramming by retroviral vectors encoding Oct3/4. Klf4, c-Myc and Sox2
US8278104B2 (en) 2005-12-13 2012-10-02 Kyoto University Induced pluripotent stem cells produced with Oct3/4, Klf4 and Sox2
WO2007079886A1 (de) 2005-12-22 2007-07-19 Csl Behring Gmbh Oktanoatreduziertes human albumin
DE102006051516A1 (de) * 2006-10-31 2008-05-08 Curevac Gmbh (Basen-)modifizierte RNA zur Expressionssteigerung eines Proteins
GB0623635D0 (en) 2006-11-27 2007-01-03 Stem Cell Sciences Uk Ltd Pluripotent cell growth media
WO2008073856A2 (en) 2006-12-08 2008-06-19 Massachusetts Institute Of Technology Delivery of nanoparticles and/or agents to cells
US10829733B2 (en) 2007-01-04 2020-11-10 Biolamina Ab Composition and method for enabling proliferation of pluripotent human stem cells
US20100172882A1 (en) 2007-01-11 2010-07-08 Glazer Peter M Compositions and methods for targeted inactivation of hiv cell surface receptors
WO2008097926A2 (en) 2007-02-02 2008-08-14 Yale University Transient transfection with rna
US9249423B2 (en) 2007-02-02 2016-02-02 Yale University Method of de-differentiating and re-differentiating somatic cells using RNA
DK3399025T3 (da) 2007-03-23 2025-06-16 Wisconsin Alumni Res Found Omprogrammering af somatiske celler
MX348010B (es) 2007-04-07 2017-05-23 Whitehead Inst Biomedical Res Reprogramacion de celulas somaticas.
KR101532442B1 (ko) 2007-12-10 2015-06-29 고쿠리츠 다이가쿠 호진 교토 다이가쿠 효율적인 핵 초기화 방법
EP2072618A1 (en) 2007-12-14 2009-06-24 Johannes Gutenberg-Universität Mainz Use of RNA for reprogramming somatic cells
JP5951254B2 (ja) 2008-03-17 2016-07-13 ザ スクリプス リサーチ インスティテュート 人工多能性幹細胞を作製するための化学的手法と遺伝的手法の組み合わせ法
US20110045001A1 (en) 2008-03-28 2011-02-24 Biontex Laboratories Gmbh Transfection results of non-viral gene delivery systems by influencing of the innate immune system
CA2720903C (en) * 2008-04-14 2019-01-15 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
ES2587395T3 (es) 2008-06-04 2016-10-24 Cellular Dynamics International, Inc. Procedimientos para la producción de células IPS usando un enfoque no vírico
WO2009147400A1 (en) 2008-06-05 2009-12-10 Iti Scotland Limited Stem cell culture media and methods
WO2010008486A2 (en) 2008-06-24 2010-01-21 Parkinsons Institute Pluripotent cell lines and methods of use thereof
US20100184033A1 (en) 2008-07-16 2010-07-22 West Michael D Methods to accelerate the isolation of novel cell strains from pluripotent stem cells and cells obtained thereby
EP2326711A2 (en) 2008-08-20 2011-06-01 VIRxSYS Corporation Compositions and methods for generation of pluripotent stem cells
EP3450545B1 (en) 2008-10-24 2023-08-23 Wisconsin Alumni Research Foundation Pluripotent stem cells obtained by non-viral reprogramming
EP2881461A1 (en) * 2008-11-21 2015-06-10 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Reprogramming cells toward a pluripotent state
US20110023141A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved with parkinson's disease
WO2010079340A2 (en) 2009-01-08 2010-07-15 Eisai R & D Management Co., Ltd. Assay
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
EP2213990B1 (fr) 2009-01-22 2016-08-17 Johnson Controls Technology Company Procédé de calibration et/ou de correction d'un dispositif d'affichage ayant une aiguille, l'aiguille étant mobile en rotation autour d'un axe de rotation
US20100209404A1 (en) 2009-02-10 2010-08-19 University Of Dayton Enhanced method for producing stem-like cells from somatic cells
US10894944B2 (en) 2009-04-10 2021-01-19 Monash University Cell culture media
WO2010123501A1 (en) 2009-04-22 2010-10-28 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long rna molecules
ES2849181T3 (es) 2009-04-22 2021-08-16 Viacyte Inc Composiciones celulares derivadas de células reprogramadas desdiferenciadas
US10837020B2 (en) 2009-04-22 2020-11-17 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long RNA molecules
US8496941B2 (en) 2009-06-03 2013-07-30 National Institute Of Advanced Industrial Science And Technology Vectors for generating pluripotent stem cells and methods of producing pluripotent stem cells using the same
EA022786B1 (ru) 2009-07-31 2016-03-31 Этрис Гмбх Рнк с комбинацией из немодифицированных и модифицированных нуклеотидов для экспрессии белков
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
AU2010312291A1 (en) * 2009-10-29 2012-06-21 Mcmaster University Generating induced pluripotent stem cells and progenitor cells from fibroblasts
KR101775926B1 (ko) 2009-11-04 2017-09-07 셀룰러 다이내믹스 인터내셔널, 인코포레이티드 화학 물질을 이용한 에피솜 재프로그래밍
EP2499239A1 (en) 2009-11-11 2012-09-19 Sanford-Burnham Medical Research Institute METHOD FOR GENERATION AND REGULATION OF iPS CELLS AND COMPOSITIONS THEREOF
US20130189741A1 (en) 2009-12-07 2013-07-25 Cellscript, Inc. Compositions and methods for reprogramming mammalian cells
ES2769129T3 (es) 2009-12-07 2020-06-24 Univ Pennsylvania Preparaciones de ARN que comprenden ARN modificado purificado para reprogramar células
PT2816112T (pt) * 2009-12-10 2018-11-20 Univ Iowa State Res Found Inc Modificação do adn modificada pelo efector tal
US8557972B2 (en) 2009-12-21 2013-10-15 University Of Washington Through Its Center For Commercialization Compositions and methods for transfection of RNA and controlled stabilization of transfected RNA
WO2011094738A1 (en) * 2010-02-01 2011-08-04 The Board Of Trustees Of The Leland Stanford Junior University Enhanced efficiency of induced pluripotent stem cell generation
WO2011110886A1 (en) 2010-03-09 2011-09-15 Biolamina Ab Composition and method for enabling proliferation of pluripotent human stem cells
WO2011130624A2 (en) 2010-04-16 2011-10-20 Immune Disease Institute, Inc. Sustained polypeptide expression from synthetic, modified rnas and uses thereof
US9725695B2 (en) 2010-05-05 2017-08-08 The Regents Of The University Of California Stem cell defined media for xeno-free and feeder free conditions and uses thereof
EP2569424A1 (en) * 2010-05-12 2013-03-20 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
US8048675B1 (en) 2010-05-12 2011-11-01 Ipierian, Inc. Integration-free human induced pluripotent stem cells from blood
AU2011256838B2 (en) * 2010-05-17 2014-10-09 Sangamo Therapeutics, Inc. Novel DNA-binding proteins and uses thereof
EP2392208B1 (en) * 2010-06-07 2016-05-04 Helmholtz Zentrum München Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Fusion proteins comprising a DNA-binding domain of a Tal effector protein and a non-specific cleavage domain of a restriction nuclease and their use
WO2011159369A1 (en) * 2010-06-14 2011-12-22 Iowa State University Research Foundation, Inc. Nuclease activity of tal effector and foki fusion protein
CA2802249A1 (en) * 2010-06-15 2011-12-22 Cellular Dynamics International, Inc. Generation of induced pluripotent stem cells from small volumes of peripheral blood
CN108531444A (zh) 2010-08-05 2018-09-14 威斯康星校友研究基金会 用于人多能细胞培养的简化基础培养基
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
US20140127814A1 (en) 2010-08-10 2014-05-08 Srinivasan Chandrasegaran Generation and use of pluripotent stem cells
GB201014169D0 (en) * 2010-08-25 2010-10-06 Cambridge Entpr Ltd In vitro hepatic differentiation
US9447408B2 (en) 2010-09-14 2016-09-20 Kyoto University Method of efficiently establishing induced pluripotent stem cells
EP3511420B1 (en) * 2010-09-27 2021-04-07 Sangamo Therapeutics, Inc. Compositions for inhibiting viral entry into cells
EP2857499A1 (en) 2010-10-01 2015-04-08 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2625278A1 (en) * 2010-10-08 2013-08-14 Regents of the University of Minnesota A method to increase gene targeting frequency
WO2012060473A1 (en) 2010-11-04 2012-05-10 Kyoto University Method of efficiently establishing induced pluripotent stem cells
WO2012094132A1 (en) * 2011-01-05 2012-07-12 Sangamo Biosciences, Inc. Methods and compositions for gene correction
US9499592B2 (en) * 2011-01-26 2016-11-22 President And Fellows Of Harvard College Transcription activator-like effectors
KR20120096395A (ko) * 2011-02-22 2012-08-30 주식회사 툴젠 뉴클레아제에 의해 유전자 변형된 세포를 농축시키는 방법
MX363013B (es) * 2011-02-25 2019-03-04 Recombinetics Inc Animales genéticamente modificados y métodos para su obtención.
AU2012225497A1 (en) * 2011-03-07 2013-10-24 Massachusetts Institute Of Technology Methods for transfecting cells with nucleic acids
CA2831613A1 (en) * 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
US10086043B2 (en) 2011-04-03 2018-10-02 The General Hospital Corporation Efficient protein expression in vivo using modified RNA (MOD-RNA)
US20140115726A1 (en) * 2011-04-05 2014-04-24 Cellectis New tale-protein scaffolds and uses thereof
US9862926B2 (en) 2011-06-27 2018-01-09 Cellscript, Llc. Inhibition of innate immune response
WO2013009825A1 (en) 2011-07-11 2013-01-17 Cellular Dynamics International, Inc. Methods for cell reprogramming and genome engineering
EP2758529B1 (en) * 2011-09-21 2019-03-20 Sangamo Therapeutics, Inc. Methods and compositions for regulation of transgene expression
PT3682905T (pt) 2011-10-03 2022-04-07 Modernatx Inc Nucleósidos, nucleótidos e ácidos nucleicos modificados e respetivas utilizações
US8497124B2 (en) 2011-12-05 2013-07-30 Factor Bioscience Inc. Methods and products for reprogramming cells to a less differentiated state
EP3260140B1 (en) * 2011-12-05 2021-02-03 Factor Bioscience Inc. Methods and products for transfecting cells
ES2923757T3 (es) 2011-12-16 2022-09-30 Modernatx Inc Composiciones de ARNm modificado
AU2012358384A1 (en) 2011-12-21 2014-07-31 Moderna Therapeutics, Inc. Methods of increasing the viability or longevity of an organ or organ explant
DK3421601T3 (da) 2011-12-30 2020-02-24 Cellscript Llc Fremstilling og anvendelse af in-vitro-syntetiseret ssRNA til indføring i pattedyrceller med henblik på at inducere en biologisk eller biokemisk effekt
WO2013151664A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
WO2013152220A2 (en) * 2012-04-04 2013-10-10 Life Technologies Corporation Tal-effector assembly platform, customized services, kits and assays
AU2013246419A1 (en) 2012-04-10 2014-10-30 City Of Hope RNA aptamers for therapeutic and diagnostic delivery to pancreatic cancer cells
AU2013251649B2 (en) 2012-04-24 2019-04-04 Vcell Therapeutics, Inc. Generating pluripotent cells de novo
US9738879B2 (en) * 2012-04-27 2017-08-22 Duke University Genetic correction of mutated genes
US10119150B2 (en) 2012-05-13 2018-11-06 Allele Biotechnology & Pharmaceuticals, Inc. Feeder-free Derivation of human-induced pluripotent stem cells with synthetic messenger RNA
US10155929B2 (en) 2012-05-13 2018-12-18 Allele Biotechnology & Pharmaceuticals, Inc. Feeder-free derivation of human-induced pluripotent stem cells with synthetic messenger RNA
CN104520424B (zh) 2012-05-13 2019-06-28 美国绿阳生物技术及医药公司 使用合成的信使rna无饲养细胞地衍生人类诱导性多能干细胞
HUE051612T2 (hu) * 2012-07-11 2021-03-01 Sangamo Therapeutics Inc Eljárások és készítmények lizoszomális tárolási betegségek kezelésére
EP2684892A1 (en) * 2012-07-13 2014-01-15 Association Française contre les Myopathies Compositions and methods for duchenne muscular dystrophy gene therapy
WO2014059173A2 (en) * 2012-10-10 2014-04-17 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
CA2890110C (en) * 2012-11-01 2023-05-02 Factor Bioscience Inc. Methods and products for expressing proteins in cells
US20140140969A1 (en) * 2012-11-20 2014-05-22 Sangamo Biosciences, Inc. Methods and compositions for muscular dystrophies
CA2892448A1 (en) * 2012-12-05 2014-06-12 Sangamo Biosciences, Inc. Methods and compositions for regulation of metabolic disorders
US10525075B2 (en) 2013-02-22 2020-01-07 The Board Of Trustees Of The Leland Stanford Junior University Compounds, compositions, methods, and kits relating to telomere extension
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
CN104878020B (zh) 2015-04-29 2017-09-15 华中农业大学 一种控制水稻叶片直立性发育的基因及其应用
IL264439B2 (en) * 2016-08-17 2024-08-01 Factor Bioscience Inc non-viral, cell-free composition comprising a synthetic messenger RNA (MRNA) encoding a gene-editing protein for use in treating cancer, and a synthetic RNA encoding a gene-editing protein for use in treatment

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