MX2020002148A - Vectores adenoasociados recombinantes. - Google Patents
Vectores adenoasociados recombinantes.Info
- Publication number
- MX2020002148A MX2020002148A MX2020002148A MX2020002148A MX2020002148A MX 2020002148 A MX2020002148 A MX 2020002148A MX 2020002148 A MX2020002148 A MX 2020002148A MX 2020002148 A MX2020002148 A MX 2020002148A MX 2020002148 A MX2020002148 A MX 2020002148A
- Authority
- MX
- Mexico
- Prior art keywords
- vectors
- recombinant adeno
- aav
- associated vectors
- adeno
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
-
- 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
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Peptides Or Proteins (AREA)
Abstract
Se proporcionan vectores de virus adenoasociados (AAV) y usos de los mismos. Más específicamente, se proporcionan vectores AAV que muestran tropismo específico para ciertos tejidos blanco, tales como sistema nervioso central (SNC) y tejido adiposo y que se puede usar para transducir las células para introducción de genes de interés en los tejidos blanco. Asimismo se proporcionan composiciones farmacéuticas que incluyen vectores AAV y un excipiente, diluyente o vehículo farmacéuticamente aceptable.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762550458P | 2017-08-25 | 2017-08-25 | |
PCT/US2018/047466 WO2019040586A1 (en) | 2017-08-25 | 2018-08-22 | RECOMBINANT ADENO-ASSOCIATE VECTORS |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2020002148A true MX2020002148A (es) | 2020-07-20 |
Family
ID=65439655
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2020002148A MX2020002148A (es) | 2017-08-25 | 2018-08-22 | Vectores adenoasociados recombinantes. |
Country Status (10)
Country | Link |
---|---|
US (2) | US20190085358A1 (es) |
EP (1) | EP3655041A4 (es) |
JP (1) | JP2020533968A (es) |
KR (1) | KR20200042935A (es) |
CN (1) | CN111163811A (es) |
AU (1) | AU2018320849A1 (es) |
CA (1) | CA3073937A1 (es) |
IL (1) | IL272655A (es) |
MX (1) | MX2020002148A (es) |
WO (1) | WO2019040586A1 (es) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2020106927A1 (en) | 2018-11-21 | 2020-05-28 | Certego Therapeutics | Gaboxadol for reducing risk of suicide and rapid relief of depression |
IL298334A (en) | 2020-05-20 | 2023-01-01 | Certego Therapeutics Inc | A canceled gaboxadol ring and its use for the treatment of psychiatric disorders |
JP2023540756A (ja) * | 2020-09-04 | 2023-09-26 | オハイオ・ステイト・イノベーション・ファウンデーション | 向上した形質導入効率及び脳内における広範な分布を有する組換えアデノ随伴ウイルスベクターの新規の操作されたキャプシド血清型 |
Family Cites Families (31)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
ATE402254T1 (de) * | 1998-05-28 | 2008-08-15 | Us Gov Health & Human Serv | Aav5 vektoren und deren verwendung |
WO2000075365A2 (en) * | 1999-06-08 | 2000-12-14 | University Of Iowa Research Foundation | Compounds and methods to enhance raav transduction |
WO2004014222A2 (en) * | 2002-08-12 | 2004-02-19 | The Regents Of The University Of Michigan | Diagnosis and treatment of tuberous sclerosis |
US7427396B2 (en) * | 2004-06-03 | 2008-09-23 | Genzyme Corporation | AAV vectors for gene delivery to the lung |
US7838657B2 (en) * | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
US8999678B2 (en) * | 2005-04-07 | 2015-04-07 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an AAV vector |
US9415121B2 (en) * | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
EP2692868A1 (en) * | 2012-08-02 | 2014-02-05 | Universitat Autònoma De Barcelona | Adeno-associated viral (AAV) vectors useful for transducing adipose tissue |
WO2014178863A1 (en) * | 2013-05-01 | 2014-11-06 | Genzyme Corporation | Compositions and methods for treating spinal muscular atrophy |
EP3024498B1 (en) * | 2013-07-22 | 2019-12-04 | The Children's Hospital of Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
GB201403684D0 (en) * | 2014-03-03 | 2014-04-16 | King S College London | Vector |
WO2015171547A1 (en) * | 2014-05-05 | 2015-11-12 | Ovid Therapeutics Inc. | Methods of treating cognitive impairment associated with neurodegenerative disorders |
JP2017518372A (ja) * | 2014-05-30 | 2017-07-06 | ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー | 潜伏性ウイルス感染用の処置剤を送達するための組成物および方法 |
US10577627B2 (en) * | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
US10711270B2 (en) * | 2014-10-03 | 2020-07-14 | University Of Massachusetts | High efficiency library-identified AAV vectors |
WO2017100671A1 (en) * | 2015-12-11 | 2017-06-15 | California Institute Of Technology | TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs) |
KR20180086266A (ko) * | 2015-12-14 | 2018-07-30 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 척수성 근위축증의 치료에 유용한 아데노-관련 바이러스 벡터 |
FI3445773T3 (fi) * | 2016-05-13 | 2023-03-30 | 4D Molecular Therapeutics Inc | Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä |
EA038695B1 (ru) * | 2017-02-15 | 2021-10-06 | Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл | Способы и композиции для переноса генов по сосудистой сети |
CN110621318A (zh) * | 2017-03-15 | 2019-12-27 | 奥维德医疗公司 | 排他地由设计者药物激活的设计者受体在治疗癫痫发作紊乱中的用途 |
CN111867595A (zh) * | 2017-12-20 | 2020-10-30 | 奥维德医疗公司 | hM4Di在治疗癫痫发作紊乱中的用途 |
MX2020013667A (es) * | 2018-06-14 | 2021-03-02 | Ovid Therapeutics Inc | Uso de mir-92a o mir-145 en el tratamiento del sindrome de angelman. |
WO2020068990A1 (en) * | 2018-09-26 | 2020-04-02 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
US10557149B1 (en) * | 2019-07-15 | 2020-02-11 | Vigene Biosciences, Inc. | Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus |
IL293068A (en) * | 2019-11-19 | 2022-07-01 | Asklepios Biopharmaceutical Inc | A therapeutic adeno-associated virus containing liver-specific promoters for the treatment of Pompe disease and lysosomal disorders |
JP2023504735A (ja) * | 2019-12-05 | 2023-02-06 | ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム | ヒトmecp2遺伝子を発現するように設計されたトランスジーンカセット |
CA3116391A1 (en) * | 2020-09-14 | 2022-03-14 | President And Fellows Of Harvard College | Recombinant adeno associated virus encoding clarin-1 and uses thereof |
KR20230152008A (ko) * | 2021-02-03 | 2023-11-02 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 단백질 m 유사체 및 융합 단백질 및 이들의 항체 기능 억제 용도 |
WO2022235702A1 (en) * | 2021-05-04 | 2022-11-10 | California Institute Of Technology | Recombinant aavs for delivery to central nervous system and brain vasculature |
CN114259502A (zh) * | 2021-12-24 | 2022-04-01 | 南京鼓楼医院 | 一种基于脂肪组织的中枢靶向递送siRNA的方法及应用 |
CN116350801A (zh) * | 2022-11-22 | 2023-06-30 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
-
2018
- 2018-08-22 JP JP2020511199A patent/JP2020533968A/ja active Pending
- 2018-08-22 EP EP18849356.3A patent/EP3655041A4/en not_active Withdrawn
- 2018-08-22 CN CN201880064381.6A patent/CN111163811A/zh active Pending
- 2018-08-22 WO PCT/US2018/047466 patent/WO2019040586A1/en unknown
- 2018-08-22 CA CA3073937A patent/CA3073937A1/en active Pending
- 2018-08-22 AU AU2018320849A patent/AU2018320849A1/en not_active Abandoned
- 2018-08-22 MX MX2020002148A patent/MX2020002148A/es unknown
- 2018-08-22 US US16/108,393 patent/US20190085358A1/en not_active Abandoned
- 2018-08-22 KR KR1020207008689A patent/KR20200042935A/ko not_active Application Discontinuation
-
2020
- 2020-02-13 IL IL272655A patent/IL272655A/en unknown
-
2021
- 2021-08-12 US US17/400,768 patent/US20210371880A1/en not_active Abandoned
Also Published As
Publication number | Publication date |
---|---|
AU2018320849A1 (en) | 2020-03-05 |
IL272655A (en) | 2020-03-31 |
US20210371880A1 (en) | 2021-12-02 |
CN111163811A (zh) | 2020-05-15 |
US20190085358A1 (en) | 2019-03-21 |
EP3655041A1 (en) | 2020-05-27 |
WO2019040586A1 (en) | 2019-02-28 |
CA3073937A1 (en) | 2019-02-28 |
JP2020533968A (ja) | 2020-11-26 |
EP3655041A4 (en) | 2021-04-21 |
KR20200042935A (ko) | 2020-04-24 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
PH12020551744A1 (en) | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues | |
MX2017012097A (es) | Variantes de virus adenoasociado y métodos de uso de estas. | |
SA517390590B1 (ar) | Ix عامل معدل، وتركيبات وطرق واستخدامات لنقل الجين إلى الخلايا، والأعضاء والأنسجة | |
WO2017058892A3 (en) | Methods and compositions for antibody-evading virus vectors | |
PH12018500594A1 (en) | Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same | |
NZ763572A (en) | Trispecific proteins and methods of use | |
MX2018006840A (es) | Focalizacion de peptidos para dirigir virus adenoasociados. | |
MX2024009690A (es) | Anticuerpos anti-cd137 y metodos de uso de los mismos. | |
EA201792236A1 (ru) | Получение увеличенных в размере векторов на основе аденоассоциированного вируса | |
AU2017248120A1 (en) | T cell receptors | |
MY190926A (en) | Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders | |
MX2014009975A (es) | Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos. | |
MX2021004397A (es) | Vectores aav recombinantes que expresan genes osteoprotectores, que incluyen has2 y lubricina, de utilidad en el tratamiento de la osteoartritis y afecciones articulares relacionadas en mamiferos. | |
MX2020002148A (es) | Vectores adenoasociados recombinantes. | |
MX2020000221A (es) | Secuencias de aminoacidos y de acidos nucleicos de adenovirus de grandes simios no humanos, vectores que contienen las mismas y usos de las mismas. | |
MX2019008105A (es) | Virus. | |
WO2012142434A8 (en) | Aav mediated ctla-4 gene transfer to treat sjogren's syndrome | |
MX2018010483A (es) | Inmunogenos para vacunacion contra vih. | |
MX2022013963A (es) | Composiciones de virus adenoasociados compatibles entre especies y metodos de uso de las mismas. | |
MX2023013509A (es) | Composiciones farmaceuticas que comprenden raav para la terapia genica galgt2 para trastornos neuromusculares y uso de las mismas. | |
PH12020550117A1 (en) | Variant rnai | |
EA202090700A1 (ru) | Компетентные по репликации аденовирусные векторы | |
MX2021004688A (es) | Distrofinas miniaturizadas y usos de las mismas. | |
EA201890274A1 (ru) | Векторная система из аденовируса 9 птиц (fadv-9) и связанные способы | |
WO2014036468A3 (en) | Aav mediated aquaporin gene transer to treat sjogren's syndrome |