TR201908020T4 - Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler. - Google Patents
Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler. Download PDFInfo
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- TR201908020T4 TR201908020T4 TR2019/08020T TR201908020T TR201908020T4 TR 201908020 T4 TR201908020 T4 TR 201908020T4 TR 2019/08020 T TR2019/08020 T TR 2019/08020T TR 201908020 T TR201908020 T TR 201908020T TR 201908020 T4 TR201908020 T4 TR 201908020T4
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- Prior art keywords
- aav9
- galactose
- vector
- cell surface
- binding domain
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- 238000000034 method Methods 0.000 title abstract 4
- 239000000203 mixture Substances 0.000 title abstract 2
- 230000001404 mediated effect Effects 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 title 1
- 210000004027 cell Anatomy 0.000 abstract 4
- 239000013598 vector Substances 0.000 abstract 4
- 230000027455 binding Effects 0.000 abstract 3
- 102000005348 Neuraminidase Human genes 0.000 abstract 2
- 108010006232 Neuraminidase Proteins 0.000 abstract 2
- WQZGKKKJIJFFOK-FPRJBGLDSA-N beta-D-galactose Chemical group OC[C@H]1O[C@@H](O)[C@H](O)[C@@H](O)[C@H]1O WQZGKKKJIJFFOK-FPRJBGLDSA-N 0.000 abstract 2
- 230000004700 cellular uptake Effects 0.000 abstract 2
- 229930182830 galactose Natural products 0.000 abstract 2
- 230000004048 modification Effects 0.000 abstract 2
- 238000012986 modification Methods 0.000 abstract 2
- 230000008685 targeting Effects 0.000 abstract 2
- 108010001857 Cell Surface Receptors Proteins 0.000 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 239000002775 capsule Substances 0.000 abstract 1
- 230000015861 cell surface binding Effects 0.000 abstract 1
- 238000003776 cleavage reaction Methods 0.000 abstract 1
- 230000002708 enhancing effect Effects 0.000 abstract 1
- 150000004676 glycans Chemical class 0.000 abstract 1
- 102000006240 membrane receptors Human genes 0.000 abstract 1
- 230000007017 scission Effects 0.000 abstract 1
- 125000005629 sialic acid group Chemical group 0.000 abstract 1
- 239000007787 solid Substances 0.000 abstract 1
- 239000013603 viral vector Substances 0.000 abstract 1
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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
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- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/66—Phosphorus compounds
- A61K31/683—Diesters of a phosphorus acid with two hydroxy compounds, e.g. phosphatidylinositols
- A61K31/685—Diesters of a phosphorus acid with two hydroxy compounds, e.g. phosphatidylinositols one of the hydroxy compounds having nitrogen atoms, e.g. phosphatidylserine, lecithin
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
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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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- 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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- 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
- C12N15/861—Adenoviral vectors
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14041—Use of virus, viral particle or viral elements as a vector
- C12N2750/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14041—Use of virus, viral particle or viral elements as a vector
- C12N2750/14045—Special targeting system for viral vectors
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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
- C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
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- Biochemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
Abstract
Bir AAV9 hücre yüzeyi bağlanma alanını içeren bir kapside sahip bir adeno-ilişkili virüs (AAV) virütik vektörün hedeflenmesini ve/veya hücresel alım verimliliğini değiştirmenin bir yöntemi tanımlanır. Yöntem bir terminal siyalik asit kalıntısına ve bir sondan bir önceki ß-galaktoz kalıntısına sahip olan bir glikan içeren bir aynı atadan gelen organizma F hücre yüzeyi reseptörünün modifiye edilmesini içerir. Modifikasyon hücrelerin bir alt-setinde AAV9 bağlanmasının geçici şekilde fonksiyonel olarak kesilip çıkarılması ve böylece, vektörün hücrelerin bir başka alt-setine yeniden-yönlendirilmesi aracılığıyla vektörü yeniden-hedeflemeyi içerebilir. Alternatif olarak, modifikasyon hücre yüzeyi ß-galaktozu açıkta bırakmak üzere bir nöraminidaz ile hücrelerin muamele edilmesi aracılığıyla hücresel alım verimliliğinin artırılmasını içerebilir. Aynı zamanda, AAV9 vektörünü ve nöraminidazı içeren bileşimler sağlanırlar. Aynı zamanda, katı desteğe bağlantılandırılmış ß-galaktoz kullanılarak AAV9?u saflaştırmak için bir yöntem sağlanır. Aynı zamanda, içinde galaktoz bağlanma alanının mutasyona uğratıldığı mutant AAV9 ve içinde bir AAV9 galaktoz bağlanma alanının mühendisliğe tabi tutulduğu AAV içeren, onların hedefleme özgüllüğünü değiştirmek üzere modifiye edilmiş bulunan mutant vektörler sağlanırlar.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201161443879P | 2011-02-17 | 2011-02-17 |
Publications (1)
Publication Number | Publication Date |
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TR201908020T4 true TR201908020T4 (tr) | 2019-06-21 |
Family
ID=45757234
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
TR2019/08020T TR201908020T4 (tr) | 2011-02-17 | 2012-02-17 | Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler. |
Country Status (16)
Country | Link |
---|---|
US (5) | US9884071B2 (tr) |
EP (1) | EP2675902B1 (tr) |
JP (1) | JP6224459B2 (tr) |
KR (1) | KR101956885B1 (tr) |
CN (1) | CN103502458B (tr) |
AU (1) | AU2012219380B2 (tr) |
BR (1) | BR112013020734A2 (tr) |
CA (1) | CA2826316C (tr) |
DK (1) | DK2675902T3 (tr) |
ES (1) | ES2724800T3 (tr) |
HU (1) | HUE043921T2 (tr) |
MX (1) | MX353930B (tr) |
PL (1) | PL2675902T3 (tr) |
SG (3) | SG10202007803QA (tr) |
TR (1) | TR201908020T4 (tr) |
WO (1) | WO2012112832A1 (tr) |
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SG192268A1 (en) | 2013-08-30 |
DK2675902T3 (da) | 2019-06-03 |
KR20140046407A (ko) | 2014-04-18 |
CA2826316A1 (en) | 2012-08-23 |
US20200101099A1 (en) | 2020-04-02 |
MX353930B (es) | 2018-02-02 |
US10406173B2 (en) | 2019-09-10 |
CN103502458B (zh) | 2016-11-16 |
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HUE043921T2 (hu) | 2019-09-30 |
MX2013009538A (es) | 2013-09-26 |
BR112013020734A2 (pt) | 2017-06-13 |
US20180185405A1 (en) | 2018-07-05 |
WO2012112832A1 (en) | 2012-08-23 |
PL2675902T3 (pl) | 2019-08-30 |
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