CL2019003582A1 - Vectores aav autoreguladores para la correcta expresión de mecp2 en el sindrome de rett. - Google Patents
Vectores aav autoreguladores para la correcta expresión de mecp2 en el sindrome de rett.Info
- Publication number
- CL2019003582A1 CL2019003582A1 CL2019003582A CL2019003582A CL2019003582A1 CL 2019003582 A1 CL2019003582 A1 CL 2019003582A1 CL 2019003582 A CL2019003582 A CL 2019003582A CL 2019003582 A CL2019003582 A CL 2019003582A CL 2019003582 A1 CL2019003582 A1 CL 2019003582A1
- Authority
- CL
- Chile
- Prior art keywords
- mecp2
- rett syndrome
- autoregulatory
- aav vectors
- correct expression
- 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
- C12N15/63—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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
- C07K2319/41—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a Myc-tag
-
- 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
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
- C12N2330/00—Production
- C12N2330/10—Production naturally occurring
-
- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific
-
- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Gastroenterology & Hepatology (AREA)
- Physics & Mathematics (AREA)
- Toxicology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762516060P | 2017-06-06 | 2017-06-06 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2019003582A1 true CL2019003582A1 (es) | 2020-09-25 |
Family
ID=64567243
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2019003582A CL2019003582A1 (es) | 2017-06-06 | 2019-12-06 | Vectores aav autoreguladores para la correcta expresión de mecp2 en el sindrome de rett. |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US11680275B2 (enExample) |
| EP (1) | EP3635122A4 (enExample) |
| JP (2) | JP2020522269A (enExample) |
| KR (1) | KR20200015701A (enExample) |
| CN (1) | CN111065742A (enExample) |
| AU (1) | AU2018281145A1 (enExample) |
| BR (1) | BR112019025732A2 (enExample) |
| CA (1) | CA3066623A1 (enExample) |
| CL (1) | CL2019003582A1 (enExample) |
| CO (1) | CO2019014840A2 (enExample) |
| EA (1) | EA201992882A1 (enExample) |
| IL (1) | IL271170A (enExample) |
| MX (1) | MX2019014760A (enExample) |
| SG (1) | SG11201911737PA (enExample) |
| WO (1) | WO2018226785A1 (enExample) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
| EP3256170B1 (en) | 2015-02-13 | 2020-09-23 | University of Massachusetts | Compositions and methods for transient delivery of nucleases |
| IL322146A (en) | 2016-11-17 | 2025-09-01 | Nationwide Childrens Hospital Inc | Intrathecal administration of adenovirus encoding methyl-CpG-binding protein 2 |
| FI3574091T3 (fi) | 2017-01-30 | 2023-05-03 | Brainvectis | Ekspressiovektori kolesterolin 24-hydrolaasia varten polyglutamiinin toiston spinoserebellaariataksioiden hoidossa |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| CA3095179A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
| CN113166208B (zh) | 2018-10-02 | 2024-11-22 | 沃雅戈治疗公司 | 重新定向aav衣壳的嗜性 |
| CN113727992A (zh) | 2019-03-21 | 2021-11-30 | 斯特里迪比奥公司 | 重组腺相关病毒载体 |
| WO2020219766A1 (en) * | 2019-04-24 | 2020-10-29 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
| EP3972631B1 (en) * | 2019-05-21 | 2024-07-10 | Institut National de la Santé et de la Recherche Médicale (INSERM) | Expression vector for cholesterol 24-hydrolase in therapy of rett syndrome |
| MX2022004353A (es) | 2019-10-17 | 2022-07-19 | Casete de transferencia de aav. | |
| CN114787180A (zh) | 2019-10-17 | 2022-07-22 | 斯特里迪比奥公司 | 用于治疗c型尼曼-匹克病的腺相关病毒载体 |
| KR20220107243A (ko) | 2019-11-25 | 2022-08-02 | 코넬 유니버시티 | Apoe 유전자 요법 |
| US11891616B2 (en) | 2019-12-05 | 2024-02-06 | Board Of Regents Of The University Of Texas System | Transgene cassettes designed to express a human MECP2 gene |
| WO2021183433A1 (en) * | 2020-03-09 | 2021-09-16 | University Of Massachusetts | Gene replacement therapy for foxg1 syndrome |
| IL298138A (en) * | 2020-05-12 | 2023-01-01 | Univ Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| US12630842B2 (en) * | 2020-05-13 | 2026-05-19 | Voyager Therapeutics, Inc. | Redirection of tropism of AAV capsids |
| MX2023000124A (es) * | 2020-06-30 | 2023-03-08 | Univ Court Univ Of Edinburgh | Sistema de expresion de transgenes. |
| JP2023542614A (ja) * | 2020-08-19 | 2023-10-11 | サレプタ セラピューティクス， インコーポレイテッド | レット症候群の治療のためのアデノ随伴ウイルスベクター |
| CN112194706B (zh) * | 2020-09-30 | 2022-03-08 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2022094078A1 (en) * | 2020-10-28 | 2022-05-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
| EP4426716A1 (en) | 2021-11-02 | 2024-09-11 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| GB202201242D0 (en) * | 2022-01-31 | 2022-03-16 | Univ Edinburgh | Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders |
| KR20260016905A (ko) * | 2023-03-22 | 2026-02-04 | 오스페달레 산 라파엘 에스.알.엘. | 유전자 치료 |
| CN121175427A (zh) * | 2023-05-15 | 2025-12-19 | 上海金珂博生物技术有限公司 | 一种Rett综合症的基因治疗 |
| AU2024285035A1 (en) * | 2023-06-09 | 2026-01-15 | Rinuagene Biotechnology Co., Ltd. | Expression-adjustable engineered rna molecule |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5399363A (en) | 1991-01-25 | 1995-03-21 | Eastman Kodak Company | Surface modified anticancer nanoparticles |
| US5543158A (en) | 1993-07-23 | 1996-08-06 | Massachusetts Institute Of Technology | Biodegradable injectable nanoparticles |
| IE80468B1 (en) | 1995-04-04 | 1998-07-29 | Elan Corp Plc | Controlled release biodegradable nanoparticles containing insulin |
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| US20040106566A1 (en) | 2002-05-17 | 2004-06-03 | Shi-Lung Lin | RNA-splicing and processing-directed gene silencing and the relative applications thereof |
| KR20100089851A (ko) | 2007-10-23 | 2010-08-12 | 노파르티스 아게 | 호흡기 질환의 치료를 위한 ｔｒｋｂ 항체의 용도 |
| JP5420568B2 (ja) * | 2008-01-17 | 2014-02-19 | アイアールエム・リミテッド・ライアビリティ・カンパニー | 改良された抗ＴｒｋＢ抗体 |
| WO2010105096A2 (en) | 2009-03-11 | 2010-09-16 | University Of Massachusetts | Modulation of human cytomegalovirus replication by micro-rna 132 (mir132), micro-rna 145 (mir145) and micro-rna 212 (mir212) |
| NZ609640A (en) | 2010-10-25 | 2015-06-26 | Univ D’Aix Marseille | Treatment of mecp2-associated disorders |
| US20160000794A1 (en) * | 2012-09-25 | 2016-01-07 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Serv | Methods for the diagnosis and treatment of sjogren's syndrome |
| EP3564379A1 (en) | 2013-09-13 | 2019-11-06 | California Institute of Technology | Selective recovery |
| WO2015127128A2 (en) | 2014-02-19 | 2015-08-27 | University Of Massachusetts | Recombinant aavs having useful transcytosis properties |
| WO2015164786A1 (en) * | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| WO2016054557A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Novel high efficiency library-identified aav vectors |
| US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
| WO2016065001A1 (en) | 2014-10-21 | 2016-04-28 | University Of Massachusetts | Recombinant aav variants and uses thereof |
| US11046955B2 (en) | 2015-04-24 | 2021-06-29 | University Of Massachusetts | Modified AAV constructs and uses thereof |
| EP3121284A1 (en) * | 2015-07-22 | 2017-01-25 | Ecole Polytechnique Fédérale de Lausanne (EPFL) | Bicistronic aav vector for rna interference in als |
-
2018
- 2018-06-06 EP EP18812887.0A patent/EP3635122A4/en active Pending
- 2018-06-06 WO PCT/US2018/036200 patent/WO2018226785A1/en not_active Ceased
- 2018-06-06 MX MX2019014760A patent/MX2019014760A/es unknown
- 2018-06-06 CN CN201880051148.4A patent/CN111065742A/zh active Pending
- 2018-06-06 BR BR112019025732-2A patent/BR112019025732A2/pt not_active IP Right Cessation
- 2018-06-06 KR KR1020207000120A patent/KR20200015701A/ko not_active Ceased
- 2018-06-06 AU AU2018281145A patent/AU2018281145A1/en not_active Abandoned
- 2018-06-06 US US16/619,733 patent/US11680275B2/en active Active
- 2018-06-06 JP JP2019567677A patent/JP2020522269A/ja active Pending
- 2018-06-06 EA EA201992882A patent/EA201992882A1/ru unknown
- 2018-06-06 SG SG11201911737PA patent/SG11201911737PA/en unknown
- 2018-06-06 CA CA3066623A patent/CA3066623A1/en active Pending
-
2019
- 2019-12-04 IL IL271170A patent/IL271170A/en unknown
- 2019-12-06 CL CL2019003582A patent/CL2019003582A1/es unknown
- 2019-12-27 CO CONC2019/0014840A patent/CO2019014840A2/es unknown
-
2023
- 2023-05-03 US US18/311,367 patent/US20230416779A1/en active Pending
- 2023-08-23 JP JP2023135189A patent/JP2023164447A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EA201992882A1 (ru) | 2020-05-25 |
| KR20200015701A (ko) | 2020-02-12 |
| JP2020522269A (ja) | 2020-07-30 |
| BR112019025732A2 (pt) | 2020-06-30 |
| EP3635122A1 (en) | 2020-04-15 |
| CN111065742A (zh) | 2020-04-24 |
| CA3066623A1 (en) | 2019-12-13 |
| AU2018281145A1 (en) | 2020-01-02 |
| EP3635122A4 (en) | 2021-03-31 |
| CO2019014840A2 (es) | 2020-01-17 |
| IL271170A (en) | 2020-01-30 |
| JP2023164447A (ja) | 2023-11-10 |
| US20230416779A1 (en) | 2023-12-28 |
| MX2019014760A (es) | 2020-08-03 |
| WO2018226785A1 (en) | 2018-12-13 |
| US20200181646A1 (en) | 2020-06-11 |
| SG11201911737PA (en) | 2020-01-30 |
| US11680275B2 (en) | 2023-06-20 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CO2019014840A2 (es) | Vectores aav auto reguladores para la expresion segura de mecp2 en sidrome de rett | |
| MX2024000329A (es) | Compuestos bifuncionales que comprenden una molecula que contiene una porcion dirigida a la proteina smarca (ptm) enlazada a una porcion de una molecula de union de ligasa de ubiquitina e3 (ulm), y su uso como moduladores de la ubiquitinacion dirigidos al tratamiento del cancer. | |
| CR20170174A (es) | Composiciones y métodos para el silenciamiento de la expresión cénica del virus de la hepatitis b | |
| MX2017014908A (es) | Proteinas de union triespecificas y metodos de uso. | |
| PE20181176A1 (es) | Composiciones biofarmaceuticas | |
| CL2018001107A1 (es) | Variantes del factor viii cpg reducido, composiciones y métodos y usos para el tratamiento de trastornos de la hemostasis. | |
| BR112018068189A2 (pt) | proteínas de ligação induzíveis e métodos de uso | |
| MX2019007873A (es) | Terapia génica para tratar la enfermedad de wilson. | |
| CL2019001293A1 (es) | Agentes, usos y métodos para el tratamiento de la sinucleinopatía. | |
| MX2024001616A (es) | Composiciones y metodos para la degradacion de proteinas mal plegadas. | |
| UY38003A (es) | Construcciones de arni para inhibir la expresión de pnpla3 y métodos de uso de las mismas | |
| MX2024002398A (es) | Proteinas de fusion gdf15 y usos de estas. | |
| CL2018000740A1 (es) | Vectores del factor viii del virus adeno-asociado, partículas víricas asociadas y formulaciones terapéuticas que los comprenden | |
| CO2021000914A2 (es) | Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a | |
| CY1124704T1 (el) | Παραγωγα sobetirome | |
| MX2018007237A (es) | Proteinas de cápside modificadas para un suministro mejorado de vectores de parvovirus. | |
| MX387144B (es) | Promotor novedoso y usos del mismo. | |
| MX2018003388A (es) | Agonistas del receptor farnesoide x y usos de los mismos. | |
| MX2017010117A (es) | Casetes optimizados de expresión del gen humano del factor viii de coagulación y su uso. | |
| CR20160561A (es) | Variantes del anticuerpo anti-factor d y sus usos | |
| MX2019012452A (es) | Meganucleasas diseñadas especificas para secuencias de reconocimiento en el gen pcsk9. | |
| ES2721309T3 (es) | Composición para preparar tagatosa y método para preparar tagatosa a partir de fructosa | |
| CL2018001548A1 (es) | Tratamiento de colestasis intrahepática y enfermedades del hígado relacionadas | |
| EA201691754A1 (ru) | Рекомбинантный микроорганизм, экспрессирующий аналог авермектина, и его применение | |
| MX2019008922A (es) | Mutantes de la caja tata y de la caja caat selectivas de tumor. |