MX353930B - Composiciones y metodos para alterar la especificidad tisular y mejorar la transferencia de genes mediada por vector viral adeno-asociado 9. - Google Patents
Composiciones y metodos para alterar la especificidad tisular y mejorar la transferencia de genes mediada por vector viral adeno-asociado 9.Info
- Publication number
- MX353930B MX353930B MX2013009538A MX2013009538A MX353930B MX 353930 B MX353930 B MX 353930B MX 2013009538 A MX2013009538 A MX 2013009538A MX 2013009538 A MX2013009538 A MX 2013009538A MX 353930 B MX353930 B MX 353930B
- Authority
- MX
- Mexico
- Prior art keywords
- aav9
- galactose
- vector
- cells
- cell surface
- Prior art date
Links
- 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
- 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
- 210000000234 capsid Anatomy 0.000 abstract 1
- 230000015861 cell surface binding Effects 0.000 abstract 1
- 230000001413 cellular effect Effects 0.000 abstract 1
- 230000004700 cellular uptake Effects 0.000 abstract 1
- 150000004676 glycans Chemical class 0.000 abstract 1
- 102000006240 membrane receptors Human genes 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
Classifications
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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
-
- 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
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- 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
-
- 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
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N2750/14045—Special targeting system for viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161443879P | 2011-02-17 | 2011-02-17 | |
| PCT/US2012/025550 WO2012112832A1 (en) | 2011-02-17 | 2012-02-17 | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| MX2013009538A MX2013009538A (es) | 2013-09-26 |
| MX353930B true MX353930B (es) | 2018-02-02 |
Family
ID=45757234
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2013009538A MX353930B (es) | 2011-02-17 | 2012-02-17 | Composiciones y metodos para alterar la especificidad tisular y mejorar la transferencia de genes mediada por vector viral adeno-asociado 9. |
Country Status (16)
| Country | Link |
|---|---|
| US (5) | US9884071B2 (enExample) |
| EP (1) | EP2675902B1 (enExample) |
| JP (1) | JP6224459B2 (enExample) |
| KR (1) | KR101956885B1 (enExample) |
| CN (1) | CN103502458B (enExample) |
| AU (1) | AU2012219380B2 (enExample) |
| BR (1) | BR112013020734A2 (enExample) |
| CA (1) | CA2826316C (enExample) |
| DK (1) | DK2675902T3 (enExample) |
| ES (1) | ES2724800T3 (enExample) |
| HU (1) | HUE043921T2 (enExample) |
| MX (1) | MX353930B (enExample) |
| PL (1) | PL2675902T3 (enExample) |
| SG (3) | SG10202007803QA (enExample) |
| TR (1) | TR201908020T4 (enExample) |
| WO (1) | WO2012112832A1 (enExample) |
Families Citing this family (121)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3072423A1 (en) * | 2003-09-30 | 2005-04-14 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| WO2011133901A2 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Aav-based treatment of cholesterol-related disorders |
| DK2561073T3 (en) | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system and methods of use thereof |
| JP6042825B2 (ja) * | 2011-02-10 | 2016-12-14 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 改変された形質導入プロファイルを有するウイルスベクターならびにその製造および使用の方法 |
| BR112013020734A2 (pt) | 2011-02-17 | 2017-06-13 | Univ Pennsylvania | composições e métodos para alterar a especificidade do tecido e aprimorar a transferência gênica mediada por aav9 |
| ES2661680T3 (es) | 2011-04-21 | 2018-04-03 | University Of Massachusetts | Composiciones basadas en VAAr y métodos para tratar deficiencias de alfa-1 anti-tripsina |
| JP6385920B2 (ja) | 2012-05-09 | 2018-09-05 | オレゴン ヘルス アンド サイエンス ユニバーシティー | アデノ随伴ウイルスプラスミド及びベクター |
| EP2749569A1 (en) | 2012-12-28 | 2014-07-02 | Annibale Alessandro Puca | Variant of BPIFB4 protein |
| US20140219964A1 (en) * | 2013-02-07 | 2014-08-07 | Children's Medical Center Corporation | Methods for inducing cardiomyocyte proliferation |
| EP2970946A4 (en) * | 2013-03-13 | 2016-09-07 | Philadelphia Children Hospital | ADENOASSOZED VIRUS VECTORS AND METHOD FOR USE THEREOF |
| IL298049B2 (en) * | 2013-03-15 | 2023-10-01 | Univ North Carolina Chapel Hill | Methods and compounds for ligation of paired AAV glycan vectors |
| SG11201507507PA (en) | 2013-03-15 | 2015-10-29 | Univ Pennsylvania | Compositions and methods for treating mpsi |
| SG10201808218YA (en) | 2014-03-21 | 2018-10-30 | Genzyme Corp | Gene therapy for retinitis pigmentosa |
| US10746742B2 (en) | 2014-04-25 | 2020-08-18 | Oregon Health & Science University | Methods of viral neutralizing antibody epitope mapping |
| WO2015187825A2 (en) | 2014-06-03 | 2015-12-10 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| EP4012035B1 (en) | 2014-09-16 | 2024-11-06 | Genzyme Corporation | Adeno-associated viral vectors for treating myocilin (myoc) glaucoma |
| PL4012035T3 (pl) | 2014-09-16 | 2025-03-24 | Genzyme Corporation | Wektory wirusowe związane z adenowirusami do leczenia jaskry miocylinowej (myoc) |
| JP6842410B2 (ja) | 2014-10-03 | 2021-03-17 | ユニバーシティ オブ マサチューセッツ | 新規の高効率ライブラリーにより同定されるａａｖベクター |
| WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
| JP7023108B2 (ja) | 2014-10-21 | 2022-02-21 | ユニバーシティ オブ マサチューセッツ | 組み換えａａｖバリアントおよびその使用 |
| BR112017009497A2 (pt) | 2014-11-05 | 2018-02-06 | Voyager Therapeutics, Inc. | polinucleotídeos de aadc para o tratamento da doença de parkinson |
| US10570395B2 (en) | 2014-11-14 | 2020-02-25 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| CA2967367C (en) | 2014-11-14 | 2023-05-16 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| HK1245326A1 (zh) | 2014-12-12 | 2018-08-24 | Voyager Therapeutics, Inc. | 用於生产scaav的组合物和方法 |
| JP2018505695A (ja) | 2015-01-20 | 2018-03-01 | ジェンザイム・コーポレーション | 組換えウイルス粒子の特徴付けのための分析超遠心分離法 |
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| EP3256588A2 (en) | 2015-02-10 | 2017-12-20 | Genzyme Corporation | VARIANT RNAi |
| WO2016131009A1 (en) | 2015-02-13 | 2016-08-18 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
| TWI707951B (zh) | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| US11046955B2 (en) | 2015-04-24 | 2021-06-29 | University Of Massachusetts | Modified AAV constructs and uses thereof |
| CN114657180A (zh) | 2015-05-16 | 2022-06-24 | 建新公司 | 深内含子突变的基因编辑 |
| CA2988471A1 (en) * | 2015-07-28 | 2017-02-02 | University Of Massachusetts | Transgenic expression of dnase i in vivo delivered by an adeno-associated virus vector |
| EP3364996B1 (en) | 2015-10-22 | 2021-08-25 | University of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| EP3364997B1 (en) | 2015-10-22 | 2024-01-17 | University of Massachusetts | Aspartoacylase gene therapy in the treatment of canavan disease |
| AU2016362477A1 (en) | 2015-12-02 | 2018-06-14 | Voyager Therapeutics, Inc. | Assays for the detection of AAV neutralizing antibodies |
| EP4215605A1 (en) | 2015-12-11 | 2023-07-26 | The Trustees of The University of Pennsylvania | Scalable purification method for aav8 |
| EP3992283A1 (en) | 2015-12-11 | 2022-05-04 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aavrh10 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| WO2017160360A2 (en) | 2015-12-11 | 2017-09-21 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| KR102833376B1 (ko) | 2015-12-15 | 2025-07-11 | 젠자임 코포레이션 | 뮤코지질증 ii형을 치료하기 위한 아데노-관련 바이러스 벡터 |
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| CA3012344A1 (en) | 2016-02-12 | 2017-08-17 | University Of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
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| AU2017250298B2 (en) | 2016-04-15 | 2024-10-03 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
| WO2017181105A1 (en) | 2016-04-15 | 2017-10-19 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
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| US11298041B2 (en) | 2016-08-30 | 2022-04-12 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
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