AR118696A1 - Administración mejorada de vectores de tratamiento génico a células retinianas mediante el uso de una enzima glicósido hidrolasa - Google Patents
Administración mejorada de vectores de tratamiento génico a células retinianas mediante el uso de una enzima glicósido hidrolasaInfo
- Publication number
- AR118696A1 AR118696A1 ARP200101063A ARP200101063A AR118696A1 AR 118696 A1 AR118696 A1 AR 118696A1 AR P200101063 A ARP200101063 A AR P200101063A AR P200101063 A ARP200101063 A AR P200101063A AR 118696 A1 AR118696 A1 AR 118696A1
- Authority
- AR
- Argentina
- Prior art keywords
- cells
- retinian
- hydrolase
- gene treatment
- treatment vectors
- Prior art date
Links
- 239000013598 vector Substances 0.000 title abstract 3
- 108090000623 proteins and genes Proteins 0.000 title abstract 2
- 108090000790 Enzymes Proteins 0.000 title 1
- 102000004190 Enzymes Human genes 0.000 title 1
- 229930182470 glycoside Natural products 0.000 title 1
- 150000002338 glycosides Chemical class 0.000 title 1
- 102000005744 Glycoside Hydrolases Human genes 0.000 abstract 2
- 108010031186 Glycoside Hydrolases Proteins 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 102000005348 Neuraminidase Human genes 0.000 abstract 1
- 108010006232 Neuraminidase Proteins 0.000 abstract 1
- 201000007737 Retinal degeneration Diseases 0.000 abstract 1
- 206010047571 Visual impairment Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 230000004438 eyesight Effects 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 210000001525 retina Anatomy 0.000 abstract 1
- 230000004258 retinal degeneration Effects 0.000 abstract 1
- 230000002207 retinal effect Effects 0.000 abstract 1
- 230000008685 targeting Effects 0.000 abstract 1
- 208000029257 vision disease Diseases 0.000 abstract 1
- 230000004393 visual impairment Effects 0.000 abstract 1
Classifications
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0048—Eye, e.g. artificial tears
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01018—Exo-alpha-sialidase (3.2.1.18), i.e. trans-sialidase
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01129—Endo-alpha-sialidase (3.2.1.129)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
-
- 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
- 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
- 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
- C12N2750/14171—Demonstrated in vivo effect
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Ophthalmology & Optometry (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962849794P | 2019-05-17 | 2019-05-17 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR118696A1 true AR118696A1 (es) | 2021-10-27 |
Family
ID=70482910
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP200101063A AR118696A1 (es) | 2019-05-17 | 2020-04-15 | Administración mejorada de vectores de tratamiento génico a células retinianas mediante el uso de una enzima glicósido hidrolasa |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US20220233655A1 (https=) |
| EP (2) | EP3969060A1 (https=) |
| JP (2) | JP2022533645A (https=) |
| KR (1) | KR20220009427A (https=) |
| CN (1) | CN114126667A (https=) |
| AR (1) | AR118696A1 (https=) |
| AU (2) | AU2020278960A1 (https=) |
| CA (2) | CA3141020A1 (https=) |
| TW (1) | TW202110486A (https=) |
| WO (2) | WO2020236351A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL294102A (en) * | 2019-12-25 | 2022-08-01 | Lumus Ltd | Optical systems and methods for tracking eyes based on directing light from the eye through an optical arrangement associated with a light-directing optical element |
| CN114516901B (zh) * | 2022-03-11 | 2023-01-17 | 上海勉亦生物科技有限公司 | 一种神经系统高亲和性的aav载体及其应用 |
| CN115323001B (zh) * | 2022-07-28 | 2024-11-26 | 深圳先进技术研究院 | 一种靶向视网膜的基因递送系统及其应用 |
Family Cites Families (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| AU707866B2 (en) | 1994-12-06 | 1999-07-22 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant AAV vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| AU722196B2 (en) | 1995-08-30 | 2000-07-27 | Genzyme Corporation | Chromatographic purification of adenovirus and AAV |
| EP1983057A3 (en) | 1995-09-08 | 2009-01-07 | Genzyme Corporation | Improved AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| EP0932418B1 (en) | 1996-09-06 | 2007-12-05 | The Trustees Of The University Of Pennsylvania | Method for recombinant adeno-associated virus-directed gene therapy |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| JP2001514845A (ja) | 1997-09-05 | 2001-09-18 | ターゲティッド ジェネティクス コーポレイション | 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法 |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| WO2012109214A1 (en) * | 2011-02-07 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Targeted transduction of aav vectors |
| DK2675902T3 (da) * | 2011-02-17 | 2019-06-03 | Univ Pennsylvania | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel |
| US9434928B2 (en) | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| AU2013296425B2 (en) * | 2012-08-01 | 2018-06-07 | Nationwide Children's Hospital | Intrathecal delivery of recombinant adeno-associated virus 9 |
| US9730888B2 (en) * | 2013-03-14 | 2017-08-15 | Wayne State University | Method of enhancing delivery of therapeutic compounds to the eye |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| WO2015126972A1 (en) * | 2014-02-21 | 2015-08-27 | University Of Florida Research Foundation, Inc. | Methods and compositions for gene delivery to on bipolar cells |
| GB201403260D0 (en) * | 2014-02-25 | 2014-04-09 | Univ Manchester | Treatment of retinal degeneration using gene therapy |
| JP6202701B2 (ja) | 2014-03-21 | 2017-09-27 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及びプログラム |
| IL248102B (en) * | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| CN107249646B (zh) * | 2014-12-16 | 2021-06-29 | 内布拉斯加大学董事会 | 用于青少年巴滕病的基因疗法 |
| JP6684343B2 (ja) * | 2015-05-21 | 2020-04-22 | アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル | ニューロン生存因子の相乗組み合わせ及びその使用 |
| CA2994160C (en) * | 2015-07-30 | 2021-08-10 | Massachusetts Eye And Ear Infirmary | Ancestral virus sequences and uses thereof |
| US20180236105A1 (en) * | 2015-10-23 | 2018-08-23 | University Of Iowa Research Foundation | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| RU2764919C2 (ru) * | 2016-06-13 | 2022-01-24 | Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл | Оптимизированные гены и экспрессионные кассеты cln1, и их применение |
| JP7048585B2 (ja) * | 2016-09-22 | 2022-04-05 | ソルボンヌ・ユニヴェルシテ | 網膜変性疾患の処置に使用するための光遺伝学的に形質転換した光受容体前駆細胞 |
| AU2018350990A1 (en) * | 2017-10-18 | 2020-05-21 | Regenxbio Inc. | Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified VEGF-Trap |
-
2020
- 2020-04-15 JP JP2021568543A patent/JP2022533645A/ja active Pending
- 2020-04-15 KR KR1020217040926A patent/KR20220009427A/ko not_active Withdrawn
- 2020-04-15 JP JP2021568813A patent/JP7707081B2/ja active Active
- 2020-04-15 WO PCT/US2020/028207 patent/WO2020236351A1/en not_active Ceased
- 2020-04-15 CA CA3141020A patent/CA3141020A1/en active Pending
- 2020-04-15 WO PCT/US2020/028352 patent/WO2020236352A1/en not_active Ceased
- 2020-04-15 CA CA3141017A patent/CA3141017A1/en active Pending
- 2020-04-15 AU AU2020278960A patent/AU2020278960A1/en active Pending
- 2020-04-15 AU AU2020278499A patent/AU2020278499A1/en active Pending
- 2020-04-15 AR ARP200101063A patent/AR118696A1/es unknown
- 2020-04-15 EP EP20727414.3A patent/EP3969060A1/en active Pending
- 2020-04-15 US US17/611,972 patent/US20220233655A1/en not_active Abandoned
- 2020-04-15 EP EP20723738.9A patent/EP3969059A1/en active Pending
- 2020-04-15 CN CN202080051735.0A patent/CN114126667A/zh active Pending
- 2020-04-15 US US17/611,982 patent/US20220226507A1/en not_active Abandoned
- 2020-04-15 TW TW109112715A patent/TW202110486A/zh unknown
Also Published As
| Publication number | Publication date |
|---|---|
| AU2020278499A1 (en) | 2022-01-06 |
| KR20220009427A (ko) | 2022-01-24 |
| JP7707081B2 (ja) | 2025-07-14 |
| JP2022533645A (ja) | 2022-07-25 |
| CA3141020A1 (en) | 2020-11-26 |
| WO2020236351A1 (en) | 2020-11-26 |
| EP3969060A1 (en) | 2022-03-23 |
| CA3141017A1 (en) | 2020-11-26 |
| EP3969059A1 (en) | 2022-03-23 |
| TW202110486A (zh) | 2021-03-16 |
| WO2020236352A1 (en) | 2020-11-26 |
| US20220226507A1 (en) | 2022-07-21 |
| CN114126667A (zh) | 2022-03-01 |
| US20220233655A1 (en) | 2022-07-28 |
| JP2022533983A (ja) | 2022-07-27 |
| AU2020278960A1 (en) | 2021-12-23 |
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