BR112016025519A2 - engenharia genética in vivo com vetores de adenovírus - Google Patents
engenharia genética in vivo com vetores de adenovírusInfo
- Publication number
- BR112016025519A2 BR112016025519A2 BR112016025519A BR112016025519A BR112016025519A2 BR 112016025519 A2 BR112016025519 A2 BR 112016025519A2 BR 112016025519 A BR112016025519 A BR 112016025519A BR 112016025519 A BR112016025519 A BR 112016025519A BR 112016025519 A2 BR112016025519 A2 BR 112016025519A2
- Authority
- BR
- Brazil
- Prior art keywords
- genetic engineering
- adenovirus vectors
- vivo genetic
- helper
- expression cassettes
- Prior art date
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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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
- A61P31/18—Antivirals for RNA viruses for HIV
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P5/00—Drugs for disorders of the endocrine system
- A61P5/48—Drugs for disorders of the endocrine system of the pancreatic hormones
- A61P5/50—Drugs for disorders of the endocrine system of the pancreatic hormones for increasing or potentiating the activity of insulin
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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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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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- 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
- C12N15/87—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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10041—Use of virus, viral particle or viral elements as a vector
- C12N2710/10045—Special targeting system for viral vectors
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10051—Methods of production or purification of viral material
- C12N2710/10052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
- C12N2710/10341—Use of virus, viral particle or viral elements as a vector
- C12N2710/10345—Special targeting system for viral vectors
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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
- C12N2710/10351—Methods of production or purification of viral material
- C12N2710/10352—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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- C12N2800/00—Nucleic acids vectors
- C12N2800/24—Vectors characterised by the absence of particular element, e.g. selectable marker, viral origin of replication
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- C12N2800/00—Nucleic acids vectors
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- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- C12N2800/00—Nucleic acids vectors
- C12N2800/90—Vectors containing a transposable element
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- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
- C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
- C12N2810/6009—Vectors comprising as targeting moiety peptide derived from defined protein from viruses dsDNA viruses
- C12N2810/6018—Adenoviridae
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- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific
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- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation
- C12N2840/102—Vectors comprising a special translation-regulating system regulates levels of translation inhibiting translation
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- C12N2999/00—Further aspects of viruses or vectors not covered by groups C12N2710/00 - C12N2796/00 or C12N2800/00
- C12N2999/007—Technological advancements, e.g. new system for producing known virus, cre-lox system for production of transgenic animals
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
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- Chemical & Material Sciences (AREA)
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Abstract
a presente invenção fornece cassetes de expressão de ácidos nucleicos recombinantes e adenovírus dependente de auxiliar, onde os cassetes de expressão utilizam um sistema de mirna com base para controlar a expressão de nucleases em células produtoras de adenovírus dependentes de auxiliar, permitindo assim a produção e utilização para em edição de gene in vivo em células cd34 +.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201461987340P | 2014-05-01 | 2014-05-01 | |
PCT/US2015/028789 WO2015168547A2 (en) | 2014-05-01 | 2015-05-01 | In vivo gene engineering with adenoviral vectors |
Publications (1)
Publication Number | Publication Date |
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BR112016025519A2 true BR112016025519A2 (pt) | 2018-01-16 |
Family
ID=54359500
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112016025519A BR112016025519A2 (pt) | 2014-05-01 | 2015-05-01 | engenharia genética in vivo com vetores de adenovírus |
Country Status (7)
Country | Link |
---|---|
US (1) | US20170037431A1 (pt) |
EP (1) | EP3137120A4 (pt) |
JP (1) | JP2017514476A (pt) |
CN (1) | CN107405411A (pt) |
BR (1) | BR112016025519A2 (pt) |
CA (1) | CA2947466A1 (pt) |
WO (1) | WO2015168547A2 (pt) |
Families Citing this family (50)
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EP2734621B1 (en) | 2011-07-22 | 2019-09-04 | President and Fellows of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
US9163284B2 (en) | 2013-08-09 | 2015-10-20 | 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 |
US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
AU2016261358B2 (en) | 2015-05-11 | 2021-09-16 | Editas Medicine, Inc. | Optimized CRISPR/Cas9 systems and methods for gene editing in stem cells |
EP3307887A1 (en) | 2015-06-09 | 2018-04-18 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for improving transplantation |
IL310721A (en) | 2015-10-23 | 2024-04-01 | Harvard College | Nucleobase editors and their uses |
EP4424829A2 (en) * | 2016-04-18 | 2024-09-04 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
JP7362097B2 (ja) * | 2016-06-27 | 2023-10-17 | 国立大学法人京都大学 | 細胞特異的にヌクレアーゼを制御する方法 |
EP3494215A1 (en) | 2016-08-03 | 2019-06-12 | President and Fellows of Harvard College | Adenosine nucleobase editors and uses thereof |
EP3497214B1 (en) | 2016-08-09 | 2023-06-28 | President and Fellows of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
KR102572759B1 (ko) | 2016-08-24 | 2023-08-29 | 상가모 테라퓨틱스, 인코포레이티드 | 조작된 뉴클레아제를 이용하는 유전자 발현의 조절 |
KR102455249B1 (ko) | 2016-08-24 | 2022-10-17 | 상가모 테라퓨틱스, 인코포레이티드 | 가공된 표적 특이적 뉴클레아제 |
MX2019002699A (es) * | 2016-09-08 | 2019-12-16 | Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P | Terapia genica para pacientes con anemia de fanconi. |
JP2019530464A (ja) | 2016-10-14 | 2019-10-24 | 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 | 核酸塩基エディターのaav送達 |
EP4360567A3 (en) * | 2016-10-24 | 2024-07-31 | Yale University | Aspiration device |
US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
EP3568474A1 (en) | 2017-01-10 | 2019-11-20 | Intrexon Corporation | Modulating expression of polypeptides via new gene switch expression systems |
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 |
IL306092A (en) | 2017-03-23 | 2023-11-01 | Harvard College | Nucleic base editors that include nucleic acid programmable DNA binding proteins |
WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
SG11201911729SA (en) * | 2017-06-16 | 2020-01-30 | Sangamo Therapeutics Inc | Targeted disruption of t cell and/or hla receptors |
US11512287B2 (en) * | 2017-06-16 | 2022-11-29 | Sangamo Therapeutics, Inc. | Targeted disruption of T cell and/or HLA receptors |
WO2019005853A2 (en) * | 2017-06-26 | 2019-01-03 | Arizona Board Of Regents On Behalf Of Arizona State University | CRISPR SYNTHETIC GENE CIRCUITS AS GENERIC THERAPY OF THE NEW GENERATION INTERNAL EAR |
US11866726B2 (en) | 2017-07-14 | 2024-01-09 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
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) |
CN107881160A (zh) * | 2017-08-11 | 2018-04-06 | 百奥泰生物科技(广州)有限公司 | 一种由基因组被编辑的cho宿主细胞产生的具有独特糖谱的重组抗体及其制备方法 |
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WO2019148029A1 (en) * | 2018-01-25 | 2019-08-01 | Pai Life Sciences, Inc. | Conjugatable desmoglein 2 (dsg2) binding proteins and uses therefor |
MA51787A (fr) | 2018-02-05 | 2020-12-16 | Vertex Pharma | Substances et méthodes de traitement d'hémoglobinopathies |
DE112020001306T5 (de) | 2019-03-19 | 2022-01-27 | Massachusetts Institute Of Technology | Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen |
JP2022539248A (ja) * | 2019-07-02 | 2022-09-07 | フレッド ハッチンソン キャンサー リサーチ センター | 組換えad35ベクター及び関連遺伝子治療改善 |
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KR20230002681A (ko) * | 2020-04-13 | 2023-01-05 | 프레드 허친슨 캔서 센터 | 대형 아데노바이러스 페이로드의 통합 |
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US20060062764A1 (en) * | 2004-08-25 | 2006-03-23 | Seshidar-Reddy Police | Fiber-modified adenoviral vectors for enhanced transduction of tumor cells |
US8110379B2 (en) * | 2007-04-26 | 2012-02-07 | Sangamo Biosciences, Inc. | Targeted integration into the PPP1R12C locus |
ES2543166T3 (es) * | 2009-03-31 | 2015-08-17 | University Of Washington | Composiciones y métodos para modular la actividad de proteínas reguladoras del complemento sobre células diana |
US8927514B2 (en) * | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
US9233127B2 (en) * | 2012-02-29 | 2016-01-12 | University Of Virginia Patent Foundation | Compositions and methods for CXCR4 signaling and umbilical cord blood stem cell engraftment |
EP2872154B1 (en) * | 2012-07-11 | 2017-05-31 | Sangamo BioSciences, Inc. | Methods and compositions for delivery of biologics |
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- 2015-05-01 US US15/305,300 patent/US20170037431A1/en not_active Abandoned
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- 2015-05-01 EP EP15785973.7A patent/EP3137120A4/en not_active Withdrawn
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US20170037431A1 (en) | 2017-02-09 |
JP2017514476A (ja) | 2017-06-08 |
WO2015168547A3 (en) | 2016-01-07 |
WO2015168547A2 (en) | 2015-11-05 |
EP3137120A2 (en) | 2017-03-08 |
CA2947466A1 (en) | 2015-11-05 |
CN107405411A (zh) | 2017-11-28 |
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