SA518391113B1 - طرق ومواد للعلاج بجين galgt2 - Google Patents
طرق ومواد للعلاج بجين galgt2 Download PDFInfo
- Publication number
- SA518391113B1 SA518391113B1 SA518391113A SA518391113A SA518391113B1 SA 518391113 B1 SA518391113 B1 SA 518391113B1 SA 518391113 A SA518391113 A SA 518391113A SA 518391113 A SA518391113 A SA 518391113A SA 518391113 B1 SA518391113 B1 SA 518391113B1
- Authority
- SA
- Saudi Arabia
- Prior art keywords
- sequence
- virus
- muscular dystrophy
- nucleotides
- muscle
- Prior art date
Links
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
- A61K48/005—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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
- A61K38/45—Transferases (2)
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
-
- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
-
- 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
- C12N9/10—Transferases (2.)
- C12N9/1048—Glycosyltransferases (2.4)
-
- 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
- C12N9/10—Transferases (2.)
- C12N9/1048—Glycosyltransferases (2.4)
- C12N9/1051—Hexosyltransferases (2.4.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y204/00—Glycosyltransferases (2.4)
- C12Y204/01—Hexosyltransferases (2.4.1)
- C12Y204/01165—N-Acetylneuraminylgalactosylglucosylceramide beta-1,4-N-acetylgalactosaminyltransferase (2.4.1.165)
-
- 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
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Analytical Chemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562220107P | 2015-09-17 | 2015-09-17 | |
| US201562221068P | 2015-09-20 | 2015-09-20 | |
| US201662301260P | 2016-02-29 | 2016-02-29 | |
| PCT/US2016/052051 WO2017049031A1 (en) | 2015-09-17 | 2016-09-16 | Methods and materials for galgt2 gene therapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SA518391113B1 true SA518391113B1 (ar) | 2022-10-25 |
Family
ID=58289891
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SA518391113A SA518391113B1 (ar) | 2015-09-17 | 2018-03-13 | طرق ومواد للعلاج بجين galgt2 |
Country Status (14)
| Country | Link |
|---|---|
| US (3) | US10980897B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3350331A4 (cg-RX-API-DMAC7.html) |
| JP (3) | JP7338970B2 (cg-RX-API-DMAC7.html) |
| KR (1) | KR102799163B1 (cg-RX-API-DMAC7.html) |
| CN (1) | CN108136049A (cg-RX-API-DMAC7.html) |
| AU (3) | AU2016323575B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2998636A1 (cg-RX-API-DMAC7.html) |
| CO (1) | CO2018003960A2 (cg-RX-API-DMAC7.html) |
| EA (1) | EA201890543A1 (cg-RX-API-DMAC7.html) |
| HK (1) | HK1257519A1 (cg-RX-API-DMAC7.html) |
| IL (2) | IL314884A (cg-RX-API-DMAC7.html) |
| MX (2) | MX2018003234A (cg-RX-API-DMAC7.html) |
| SA (1) | SA518391113B1 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2017049031A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3350331A4 (en) | 2015-09-17 | 2019-01-23 | Research Institute at Nationwide Children's Hospital | METHOD AND MATERIALS FOR GALGT2 GENE THERAPY |
| MA45477A (fr) | 2016-04-15 | 2019-02-20 | Res Inst Nationwide Childrens Hospital | Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire |
| JP7162021B2 (ja) | 2017-03-17 | 2022-10-27 | ニューカッスル ユニバーシティ | 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達 |
| UY37679A (es) * | 2017-04-14 | 2018-11-30 | Regenxbio Inc | Tratamiento de mucopolisacaridosis ii con iduronato-2-sulfatasa (ids) humana recombinante producida por células neurales o gliales humanas |
| MA50836A (fr) | 2017-10-18 | 2020-08-26 | Res Inst Nationwide Childrens Hospital | Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique de muscles pour traiter la dystrophie musculaire |
| MY207563A (en) | 2017-10-20 | 2025-03-04 | Res Inst Nationwide Childrens Hospital | Methods and materials for nt-3 gene therapy |
| PT3807413T (pt) | 2018-06-18 | 2025-08-19 | Res Inst Nationwide Childrens Hospital | Administração por vetor de vírus adenoassociado de microdistrofina específica do músculo para tratar a distrofia muscular |
| CA3104471A1 (en) * | 2018-06-18 | 2019-12-26 | Research Institute At Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies |
| CN114828858A (zh) * | 2019-10-25 | 2022-07-29 | 奥登茨治疗公司 | 用于治疗糖原贮积症的组合物和方法 |
| EP4051324A4 (en) * | 2019-10-28 | 2023-11-29 | University Of Florida Research Foundation, Incorporated | Gene therapy vectors |
| MX2023001411A (es) * | 2020-08-07 | 2023-05-15 | Janssen Biotech Inc | Formulaciones para particulas virales altamente purificadas. |
| EP4230196A1 (en) | 2022-02-21 | 2023-08-23 | Som Innovation Biotech, S.A. | Compounds for use in the treatment of dystrophinopathies |
Family Cites Families (23)
| 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 |
| ES2216005T3 (es) | 1993-11-09 | 2004-10-16 | Targeted Genetics Corporation | Produccion de titulos elevados de vectores de aav recombinantes. |
| CA2176215C (en) | 1993-11-09 | 2007-06-26 | James P. Trempe | Stable cell lines capable of expressing the adeno-associated virus replication gene |
| 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 |
| EP0796339A1 (en) | 1994-12-06 | 1997-09-24 | 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 |
| CA2625279A1 (en) | 1995-08-30 | 1997-03-06 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
| AU715543B2 (en) | 1995-09-08 | 2000-02-03 | 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 |
| ATE465267T1 (de) | 1996-09-06 | 2010-05-15 | Univ Pennsylvania | Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen |
| AU758708B2 (en) | 1997-09-05 | 2003-03-27 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| 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 |
| EP1263966A1 (en) | 1999-12-13 | 2002-12-11 | Lexicon Genetics Incorporated | Novel human transferase proteins and polynucleotides encoding the same |
| WO2001083692A2 (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 |
| JP2004535801A (ja) * | 2001-05-24 | 2004-12-02 | ジェンザイム コーポレイション | 筋特異的発現ベクター |
| US20060194265A1 (en) | 2001-10-23 | 2006-08-31 | Morris David W | Novel therapeutic targets in cancer |
| WO2008048986A2 (en) * | 2006-10-17 | 2008-04-24 | Children's Hospital Medical Center | Gene array technique for predicting response in inflammatory bowel diseases |
| WO2013078316A1 (en) | 2011-11-23 | 2013-05-30 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| WO2013102904A1 (en) * | 2012-01-05 | 2013-07-11 | Hadasit Medical Research Services & Development Ltd. | Methods and compositions for gene delivery |
| AU2013221212B2 (en) | 2012-02-17 | 2018-08-09 | The Children's Hospital Of Philadelphia | AAV vector compositions and methods for gene transfer to cells, organs and tissues |
| EP3350331A4 (en) | 2015-09-17 | 2019-01-23 | Research Institute at Nationwide Children's Hospital | METHOD AND MATERIALS FOR GALGT2 GENE THERAPY |
-
2016
- 2016-09-16 EP EP16847351.0A patent/EP3350331A4/en active Pending
- 2016-09-16 US US15/759,474 patent/US10980897B2/en active Active
- 2016-09-16 AU AU2016323575A patent/AU2016323575B2/en active Active
- 2016-09-16 CN CN201680054261.9A patent/CN108136049A/zh active Pending
- 2016-09-16 WO PCT/US2016/052051 patent/WO2017049031A1/en not_active Ceased
- 2016-09-16 MX MX2018003234A patent/MX2018003234A/es unknown
- 2016-09-16 CA CA2998636A patent/CA2998636A1/en active Pending
- 2016-09-16 JP JP2018513504A patent/JP7338970B2/ja active Active
- 2016-09-16 IL IL314884A patent/IL314884A/en unknown
- 2016-09-16 HK HK18116463.4A patent/HK1257519A1/zh unknown
- 2016-09-16 IL IL258005A patent/IL258005B2/en unknown
- 2016-09-16 KR KR1020187010628A patent/KR102799163B1/ko active Active
- 2016-09-16 EA EA201890543A patent/EA201890543A1/ru unknown
-
2018
- 2018-03-13 SA SA518391113A patent/SA518391113B1/ar unknown
- 2018-03-14 MX MX2023013509A patent/MX2023013509A/es unknown
- 2018-04-13 CO CONC2018/0003960A patent/CO2018003960A2/es unknown
-
2021
- 2021-03-16 US US17/203,232 patent/US12121595B2/en active Active
- 2021-11-05 JP JP2021180893A patent/JP2022010079A/ja active Pending
-
2023
- 2023-01-31 AU AU2023200499A patent/AU2023200499B2/en active Active
-
2024
- 2024-05-30 JP JP2024087848A patent/JP2024109934A/ja active Pending
- 2024-10-03 US US18/905,388 patent/US20250025578A1/en active Pending
-
2025
- 2025-08-06 AU AU2025213610A patent/AU2025213610A1/en active Pending
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| SA518391113B1 (ar) | طرق ومواد للعلاج بجين galgt2 | |
| TWI844587B (zh) | Aav病毒載體及其用途 | |
| CA2693178C (en) | Myostatin inhibition for enhancing muscle and/or improving muscle function | |
| AU2025206429A1 (en) | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A | |
| JP2021500029A (ja) | Ｎｔ−３遺伝子治療のための方法及び材料 | |
| CN110325219B (zh) | 编码甲基-CpG结合蛋白2的重组腺相关病毒的鞘内递送 | |
| CN116887867A (zh) | 用于治疗grn相关成人发作性神经退行性病变的重组腺相关病毒 | |
| RU2796274C2 (ru) | Вирусные векторы на основе aav и пути их применения | |
| EA044183B1 (ru) | Способы и материалы для генной терапии galgt2 | |
| BR112018005177B1 (pt) | Ácido nucleico, partícula de vírus adeno-associado recombinante (raav) compreendendo o mesmo e composição | |
| AU2023299274A1 (en) | Methods of treating muscular dystrophy | |
| WO2021231443A1 (en) | Compositions useful in treatment of krabbe disease |