IL261014B2 - Aav vectors for treatment of dominant retinitis pigmentosa - Google Patents
Aav vectors for treatment of dominant retinitis pigmentosaInfo
- Publication number
- IL261014B2 IL261014B2 IL261014A IL26101418A IL261014B2 IL 261014 B2 IL261014 B2 IL 261014B2 IL 261014 A IL261014 A IL 261014A IL 26101418 A IL26101418 A IL 26101418A IL 261014 B2 IL261014 B2 IL 261014B2
- Authority
- IL
- Israel
- Prior art keywords
- rna
- rho
- vector
- sequence
- seq
- Prior art date
Links
Classifications
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- 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
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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- 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
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- 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
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- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
- C12N2310/122—Hairpin
-
- 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]
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- 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
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted 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
- 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)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Virology (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Ophthalmology & Optometry (AREA)
- General Chemical & Material Sciences (AREA)
- Immunology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Pigments, Carbon Blacks, Or Wood Stains (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Inorganic Compounds Of Heavy Metals (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Paper (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662302122P | 2016-03-01 | 2016-03-01 | |
| US201662398451P | 2016-09-22 | 2016-09-22 | |
| PCT/US2017/020289 WO2017151823A1 (en) | 2016-03-01 | 2017-03-01 | Aav vectors for treatment of dominant retinitis pigmentosa |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| IL261014A IL261014A (en) | 2018-10-31 |
| IL261014B1 IL261014B1 (en) | 2023-06-01 |
| IL261014B2 true IL261014B2 (en) | 2023-10-01 |
Family
ID=59743213
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL261014A IL261014B2 (en) | 2016-03-01 | 2017-03-01 | Aav vectors for treatment of dominant retinitis pigmentosa |
| IL302455A IL302455A (en) | 2016-03-01 | 2017-03-01 | Adeno-associated virus vectors for the treatment of dominant retinitis pigmentosa |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL302455A IL302455A (en) | 2016-03-01 | 2017-03-01 | Adeno-associated virus vectors for the treatment of dominant retinitis pigmentosa |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US11118185B2 (cg-RX-API-DMAC7.html) |
| EP (2) | EP3423582B1 (cg-RX-API-DMAC7.html) |
| JP (3) | JP7272795B2 (cg-RX-API-DMAC7.html) |
| CN (2) | CN109154002B (cg-RX-API-DMAC7.html) |
| AU (2) | AU2017227776C1 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3014671A1 (cg-RX-API-DMAC7.html) |
| ES (1) | ES2923877T3 (cg-RX-API-DMAC7.html) |
| IL (2) | IL261014B2 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2017151823A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2017227776C1 (en) | 2016-03-01 | 2024-01-25 | The Trustees Of The University Of Pennsylvania | AAV vectors for treatment of dominant retinitis pigmentosa |
| AU2018366034B2 (en) * | 2017-11-07 | 2024-11-21 | The University Of North Carolina At Chapel Hill | Methods and compositions for circular RNA molecules |
| WO2019222354A1 (en) * | 2018-05-15 | 2019-11-21 | University Of Washington | Compositions and methods for reducing spliceopathy and treating rna dominance disorders |
| US12203074B2 (en) | 2018-06-01 | 2025-01-21 | University Of Florida Research Foundation, Incorporated | Compositions and methods for treatment of dominant retinitis pigmentosa |
| GB201817469D0 (en) * | 2018-10-26 | 2018-12-12 | Univ Oxford Innovation Ltd | Gene therapy for retinal disease |
| CN111518813B (zh) * | 2019-02-03 | 2023-04-28 | 武汉纽福斯生物科技有限公司 | 视紫红质的编码序列、其表达载体构建及其应用 |
| CN114555084A (zh) * | 2019-08-15 | 2022-05-27 | 费城儿童医院 | 用于治疗SCA1的转基因和内含子衍生miRNA联合疗法 |
| CN113038971B (zh) * | 2020-04-21 | 2022-06-24 | 北京中因科技有限公司 | RHO-adRP基于基因编辑的方法和组合物 |
| CN111926044B (zh) * | 2020-10-12 | 2021-01-22 | 北京大学第三医院（北京大学第三临床医学院） | 结合突变rho基因的核酸分子和试剂盒 |
| MX2023008826A (es) | 2021-02-01 | 2023-09-15 | Regenxbio Inc | Terapia génica para lipofuscinosis neuronal ceroidea. |
| WO2025231487A1 (en) * | 2024-05-03 | 2025-11-06 | The Trustees Of The University Of Pennsylvania | Photoreceptor specific promotors for use in gene therapies for treatment of retinal degeneration |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2010127209A2 (en) * | 2009-04-30 | 2010-11-04 | The Research Foundation Of State University Of New York | Compositions and methods for therapy of macular degeneration |
| WO2015143418A2 (en) * | 2014-03-21 | 2015-09-24 | Genzyme Corporation | Gene therapy for retinitis pigmentosa |
Family Cites Families (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5795587A (en) | 1995-01-23 | 1998-08-18 | University Of Pittsburgh | Stable lipid-comprising drug delivery complexes and methods for their production |
| US8551970B2 (en) | 1996-04-02 | 2013-10-08 | Optigen Patents Limited | Genetic suppression and replacement |
| GB9606961D0 (en) | 1996-04-02 | 1996-06-05 | Farrar Gwyneth J | Genetic strategy III |
| US20040234999A1 (en) | 1996-04-02 | 2004-11-25 | Farrar Gwenyth Jane | Genetic suppression and replacement |
| WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
| AU2004233043A1 (en) | 2003-04-18 | 2004-11-04 | The Trustees Of The University Of Pennsylvania | Compositions and methods for siRNA inhibition of angiopoietin 1 and 2 and their receptor Tie2 |
| US20070009899A1 (en) | 2003-10-02 | 2007-01-11 | Mounts William M | Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases |
| CN1977044A (zh) | 2004-03-24 | 2007-06-06 | 肿瘤疗法科学股份有限公司 | 治疗胰腺癌的组合物和方法 |
| US20080221057A1 (en) | 2007-02-16 | 2008-09-11 | Wyeth | Secreted protein ccdc80 regulates adipocyte differentiation |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| US8257969B2 (en) * | 2007-04-12 | 2012-09-04 | The Provost Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth | Genetic suppression and replacement |
| CA2702701A1 (en) | 2007-08-03 | 2009-03-19 | Melba Ketchum | Compositions, methods and systems for the simultaneous determination of parentage, identity, sex, genotype and/or phenotype, and breed determination in animals |
| CN107648604A (zh) | 2010-07-30 | 2018-02-02 | 库瑞瓦格股份公司 | 聚合物载体运载物复合物及聚合物载体的用途 |
| CN102061308B (zh) | 2010-10-29 | 2013-05-08 | 北京未名凯拓作物设计中心有限公司 | 一种用百草枯筛选转基因植物的方法 |
| CN102061305B (zh) * | 2010-12-15 | 2012-07-18 | 深圳市百恩维生物科技有限公司 | 一种多ShRNA高效协同沉默基因方法及载体 |
| US20130064815A1 (en) * | 2011-09-12 | 2013-03-14 | The Trustees Of Princeton University | Inducing apoptosis in quiescent cells |
| WO2013175446A1 (en) | 2012-05-25 | 2013-11-28 | Commissariat A L'energie Atomique Et Aux Energies Alternatives | Vector for the selective silencing of a gene in astrocytes |
| WO2014138792A1 (en) | 2013-03-14 | 2014-09-18 | Commonwealth Scientific And Industrial Research Organisation | Double-stranded rna |
| CA2977965C (en) | 2015-02-26 | 2021-12-21 | Ionis Pharmaceuticals, Inc. | Allele specific modulators of p23h rhodopsin |
| EP4008780A1 (en) | 2015-04-30 | 2022-06-08 | The Trustees of Columbia University in the City of New York | Gene therapy for autosomal dominant diseases |
| WO2017137493A1 (en) | 2016-02-09 | 2017-08-17 | Fondazione Telethon | Synthetic promoters and uses thereof |
| US20170348387A1 (en) | 2016-02-29 | 2017-12-07 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for nphp5 lca-ciliopathy |
| AU2017227776C1 (en) | 2016-03-01 | 2024-01-25 | The Trustees Of The University Of Pennsylvania | AAV vectors for treatment of dominant retinitis pigmentosa |
-
2017
- 2017-03-01 AU AU2017227776A patent/AU2017227776C1/en active Active
- 2017-03-01 IL IL261014A patent/IL261014B2/en unknown
- 2017-03-01 EP EP17760752.0A patent/EP3423582B1/en active Active
- 2017-03-01 CA CA3014671A patent/CA3014671A1/en active Pending
- 2017-03-01 JP JP2018545818A patent/JP7272795B2/ja active Active
- 2017-03-01 US US16/081,307 patent/US11118185B2/en active Active
- 2017-03-01 ES ES17760752T patent/ES2923877T3/es active Active
- 2017-03-01 CN CN201780014309.8A patent/CN109154002B/zh active Active
- 2017-03-01 CN CN202210934990.0A patent/CN115896106A/zh active Pending
- 2017-03-01 EP EP22170988.4A patent/EP4119668A1/en active Pending
- 2017-03-01 WO PCT/US2017/020289 patent/WO2017151823A1/en not_active Ceased
- 2017-03-01 IL IL302455A patent/IL302455A/en unknown
-
2021
- 2021-05-21 US US17/327,609 patent/US20210324387A1/en active Pending
-
2022
- 2022-09-29 JP JP2022156496A patent/JP7642211B2/ja active Active
-
2023
- 2023-09-27 AU AU2023237106A patent/AU2023237106B2/en active Active
-
2024
- 2024-09-27 JP JP2024168632A patent/JP2025016451A/ja active Pending
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2010127209A2 (en) * | 2009-04-30 | 2010-11-04 | The Research Foundation Of State University Of New York | Compositions and methods for therapy of macular degeneration |
| WO2015143418A2 (en) * | 2014-03-21 | 2015-09-24 | Genzyme Corporation | Gene therapy for retinitis pigmentosa |
Non-Patent Citations (1)
| Title |
|---|
| BEHRMAN, SHANNON, DIEGO ACOSTA-ALVEAR, AND PETER WALTER., A CHOP-REGULATED MICRORNA CONTROLS RHODOPSIN EXPRESSION., 14 March 2011 (2011-03-14) * |
Also Published As
| Publication number | Publication date |
|---|---|
| EP4119668A1 (en) | 2023-01-18 |
| AU2023237106B2 (en) | 2025-08-28 |
| JP7642211B2 (ja) | 2025-03-10 |
| US20210324387A1 (en) | 2021-10-21 |
| CN109154002B (zh) | 2022-08-30 |
| IL261014B1 (en) | 2023-06-01 |
| AU2017227776B2 (en) | 2023-07-06 |
| CA3014671A1 (en) | 2017-09-08 |
| IL261014A (en) | 2018-10-31 |
| AU2017227776A1 (en) | 2018-08-23 |
| JP2019511216A (ja) | 2019-04-25 |
| WO2017151823A1 (en) | 2017-09-08 |
| AU2017227776C1 (en) | 2024-01-25 |
| EP3423582A4 (en) | 2019-11-13 |
| EP3423582A1 (en) | 2019-01-09 |
| JP2025016451A (ja) | 2025-02-04 |
| US20190093111A1 (en) | 2019-03-28 |
| EP3423582B1 (en) | 2022-05-04 |
| CN109154002A (zh) | 2019-01-04 |
| ES2923877T3 (es) | 2022-10-03 |
| JP2022185052A (ja) | 2022-12-13 |
| CN115896106A (zh) | 2023-04-04 |
| IL302455A (en) | 2023-06-01 |
| JP7272795B2 (ja) | 2023-05-12 |
| AU2023237106A1 (en) | 2023-10-19 |
| US11118185B2 (en) | 2021-09-14 |
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