BR112023015769A2 - Vetor de vírus adenoassociado de rna (raav) e usos do mesmo - Google Patents
Vetor de vírus adenoassociado de rna (raav) e usos do mesmoInfo
- Publication number
- BR112023015769A2 BR112023015769A2 BR112023015769A BR112023015769A BR112023015769A2 BR 112023015769 A2 BR112023015769 A2 BR 112023015769A2 BR 112023015769 A BR112023015769 A BR 112023015769A BR 112023015769 A BR112023015769 A BR 112023015769A BR 112023015769 A2 BR112023015769 A2 BR 112023015769A2
- Authority
- BR
- Brazil
- Prior art keywords
- raav
- adeno
- vector
- rna
- rna virus
- Prior art date
Links
Classifications
-
- 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
- C12N15/87—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
-
- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14123—Virus like particles [VLP]
-
- 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
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Mycology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN2021075874 | 2021-02-07 | ||
| PCT/CN2022/075366 WO2022166954A1 (en) | 2021-02-07 | 2022-02-07 | Rna adeno-associated virus (raav) vector and uses thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112023015769A2 true BR112023015769A2 (pt) | 2023-12-12 |
Family
ID=74946911
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112023015769A BR112023015769A2 (pt) | 2021-02-07 | 2022-02-07 | Vetor de vírus adenoassociado de rna (raav) e usos do mesmo |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US12031148B2 (https=) |
| EP (1) | EP4288112A4 (https=) |
| JP (1) | JP2024508696A (https=) |
| KR (1) | KR20230142776A (https=) |
| CN (1) | CN115209924A (https=) |
| AU (1) | AU2022217349A1 (https=) |
| BR (1) | BR112023015769A2 (https=) |
| CA (1) | CA3207601A1 (https=) |
| IL (1) | IL305002A (https=) |
| WO (1) | WO2022166954A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN116218904B (zh) * | 2022-11-03 | 2025-04-15 | 国药中生生物技术研究院有限公司 | 一种可提高工程化细胞外泌体中特定核酸分子载量的方法及其应用 |
| CN120659629A (zh) * | 2022-12-08 | 2025-09-16 | 辉大基因治疗(新加坡)私人有限公司 | 用于产生rna包装的aav颗粒的改善的系统 |
| CN117778431B (zh) * | 2023-12-04 | 2024-10-25 | 广州派真生物技术有限公司 | 一种用于重组腺相关病毒包装的质粒系统及其应用 |
| WO2025124549A1 (en) * | 2023-12-13 | 2025-06-19 | Huidagene Therapeutics (Singapore) Pte. Ltd. | Improved system and method for producing recombinant dna-packaged aav particles |
| CN117701638B (zh) * | 2023-12-18 | 2024-10-18 | 苏州新芽基因生物技术有限公司 | 一种抑制残留aav载体质粒和质粒骨架转录活性的方法 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6346415B1 (en) * | 1997-10-21 | 2002-02-12 | Targeted Genetics Corporation | Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors |
| US9790490B2 (en) * | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| JP7044373B2 (ja) * | 2015-07-15 | 2022-03-30 | ラトガース，ザ ステート ユニバーシティ オブ ニュージャージー | ヌクレアーゼ非依存的な標的化遺伝子編集プラットフォームおよびその用途 |
| WO2018035387A1 (en) * | 2016-08-17 | 2018-02-22 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
| EP4485466A3 (en) * | 2016-08-17 | 2025-04-02 | The Broad Institute Inc. | Novel crispr enzymes and systems |
| EP3565608A4 (en) * | 2017-01-05 | 2020-12-16 | Rutgers, The State University of New Jersey | TARGETED GENE EDITING PLATFORM INDEPENDENT OF BICATENARY DNA BREAKAGE AND ITS USES |
| JP7215716B2 (ja) * | 2017-01-13 | 2023-01-31 | 学校法人自治医科大学 | 肝臓ゲノム上の凝固関連因子遺伝子を破壊するためのａａｖベクター |
| BR112019028146A2 (pt) * | 2017-07-31 | 2020-07-07 | Sigma-Aldrich Co. Llc | rna guia sintético para sistemas ativadores de crispr/cas |
| CN109055375B (zh) * | 2018-07-26 | 2021-11-12 | 东南大学 | 一种crispr辅助反式增强子激活基因表达的方法及其应用 |
| CN111349148A (zh) * | 2020-03-13 | 2020-06-30 | 辉大(上海)生物科技有限公司 | 一种腺相关病毒载体及其用途 |
| JP2023537296A (ja) * | 2020-07-29 | 2023-08-31 | ザ・キャソリック・ユニバーシティ・オブ・アメリカ | ヒトゲノムリモデリングのためのバクテリオファージベースの人工ウイルスの設計 |
-
2022
- 2022-02-07 CN CN202280002654.0A patent/CN115209924A/zh active Pending
- 2022-02-07 CA CA3207601A patent/CA3207601A1/en active Pending
- 2022-02-07 AU AU2022217349A patent/AU2022217349A1/en active Pending
- 2022-02-07 KR KR1020237030365A patent/KR20230142776A/ko active Pending
- 2022-02-07 IL IL305002A patent/IL305002A/en unknown
- 2022-02-07 EP EP22749233.7A patent/EP4288112A4/en active Pending
- 2022-02-07 JP JP2023548288A patent/JP2024508696A/ja active Pending
- 2022-02-07 BR BR112023015769A patent/BR112023015769A2/pt not_active Application Discontinuation
- 2022-02-07 WO PCT/CN2022/075366 patent/WO2022166954A1/en not_active Ceased
- 2022-06-09 US US17/836,222 patent/US12031148B2/en active Active
-
2024
- 2024-05-15 US US18/664,495 patent/US20240368629A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| KR20230142776A (ko) | 2023-10-11 |
| US12031148B2 (en) | 2024-07-09 |
| CA3207601A1 (en) | 2022-08-11 |
| EP4288112A4 (en) | 2025-08-06 |
| JP2024508696A (ja) | 2024-02-28 |
| CN115209924A (zh) | 2022-10-18 |
| US20230062529A1 (en) | 2023-03-02 |
| US20240368629A1 (en) | 2024-11-07 |
| IL305002A (en) | 2023-10-01 |
| WO2022166954A1 (en) | 2022-08-11 |
| AU2022217349A1 (en) | 2023-08-31 |
| EP4288112A1 (en) | 2023-12-13 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| BR112023015769A2 (pt) | Vetor de vírus adenoassociado de rna (raav) e usos do mesmo | |
| BR112021020054A2 (pt) | Capsídeos de vírus adeno-associado (aav) e composições contendo os mesmos | |
| BR112021018776A2 (pt) | Vetores de vírus adenoassociado recombinante | |
| MX2022014256A (es) | Redirección del tropismo de las cápsides de aav. | |
| MX388331B (es) | Variantes de virus adenoasociado y métodos de uso de estas | |
| CL2020000728A1 (es) | Variantes de cápsides de virus adenoasociados y métodos de uso de estas. | |
| CL2023000264A1 (es) | Nuevos vectores virales adeno-asociados dirigidos al hígado | |
| PH12018500594A1 (en) | Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same | |
| BR112021021792A2 (pt) | Composições úteis para tratamento de doença de pompe | |
| EP4219695A3 (en) | Adeno-associated virus variant capsids and use for inhibiting angiogenesis | |
| EA201991911A1 (ru) | Способы и композиции для переноса генов по сосудистой сети | |
| PH12018502387A1 (en) | Adeno-associated virus variant capsids and methods of use thereof | |
| DE602005026269D1 (de) | Sequenzielle freisetzung immunogener moleküle über adenoviren und adeno-assoziierte viren vermittelte abgaben | |
| BR112019002904A2 (pt) | métodos e composições para transferência gênica direcionada | |
| MX2022013963A (es) | Composiciones de virus adenoasociados compatibles entre especies y metodos de uso de las mismas. | |
| EA202091352A1 (ru) | Генотерапия мукополисахаридоза iiib | |
| EP4110792A4 (en) | POLYPEPTIDES AND CAPSIDIC VECTORS OF ADENO-ASSOCIATED VIRUSES | |
| PH12022550109A1 (en) | Modified aav capsid proteins for treatment of arthritic disease | |
| AR119869A1 (es) | Proteína vp1 modificada aislada de la cápside del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en la misma | |
| BRPI0407840A (pt) | polipeptìdeo purificado, anticorpo policlonal ou monoclonal purificado, vetor de expressão, seqüência de polinucleotìdeo purificada, uso de uma seqüência de polinucleotìdeo, vetor viral recombinante, vìrus do sarampo recombinante, composição farmacêutica, uso da mesma, célula hospedeira, método para produzir um vìrus recombinante para a produção de um vìrus recombinante, linhagem de células, ensaio de neutralização de vìrus do nilo ocidental, e, método para tratar e/ou prevenir uma infecção ou doença associada com wnv ou vìrus do dengue em um animal | |
| CO2023000444A2 (es) | Terapia génica usando constructos de ácido nucleico que comprenden secuencias promotoras del gen de la proteína 2 de unión a metil cpg (mecp2) | |
| WO2023004407A3 (en) | Adeno-associated virus compositions and methods of use thereof | |
| BR112017024283A2 (pt) | vacinas contra a dengue | |
| Cioni et al. | Comparing Effects of BK Virus Agnoprotein and Herpes Simplex‐1 ICP47 on MHC‐I and MHC‐II Expression | |
| WO2007144886A3 (en) | Virus-like particles for treatment of viral infections |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| B11A | Dismissal acc. art.33 of ipl - examination not requested within 36 months of filing | ||
| B11Y | Definitive dismissal - extension of time limit for request of examination expired [chapter 11.1.1 patent gazette] |