JP2018515102A5 - - Google Patents
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- Publication number
- JP2018515102A5 JP2018515102A5 JP2017559315A JP2017559315A JP2018515102A5 JP 2018515102 A5 JP2018515102 A5 JP 2018515102A5 JP 2017559315 A JP2017559315 A JP 2017559315A JP 2017559315 A JP2017559315 A JP 2017559315A JP 2018515102 A5 JP2018515102 A5 JP 2018515102A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- acid molecule
- itr2
- itr1
- hpm
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
Links
- 150000007523 nucleic acids Chemical class 0.000 claims 42
- 108020004707 nucleic acids Proteins 0.000 claims 40
- 102000039446 nucleic acids Human genes 0.000 claims 40
- 239000002245 particle Substances 0.000 claims 19
- 230000003612 virological effect Effects 0.000 claims 14
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 4
- 241000700605 Viruses Species 0.000 claims 4
- 125000006850 spacer group Chemical group 0.000 claims 4
- 108091027967 Small hairpin RNA Proteins 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 239000004055 small Interfering RNA Substances 0.000 claims 3
- 108700026244 Open Reading Frames Proteins 0.000 claims 2
- 241000125945 Protoparvovirus Species 0.000 claims 2
- 150000001413 amino acids Chemical class 0.000 claims 2
- 239000001963 growth medium Substances 0.000 claims 2
- 230000002401 inhibitory effect Effects 0.000 claims 2
- 239000003550 marker Substances 0.000 claims 2
- 108091070501 miRNA Proteins 0.000 claims 2
- 239000002679 microRNA Substances 0.000 claims 2
- 108020005544 Antisense RNA Proteins 0.000 claims 1
- 108090000994 Catalytic RNA Proteins 0.000 claims 1
- 102000053642 Catalytic RNA Human genes 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 102000040945 Transcription factor Human genes 0.000 claims 1
- 108091023040 Transcription factor Proteins 0.000 claims 1
- 239000003242 anti bacterial agent Substances 0.000 claims 1
- 230000003115 biocidal effect Effects 0.000 claims 1
- 238000010367 cloning Methods 0.000 claims 1
- 239000003184 complementary RNA Substances 0.000 claims 1
- 239000000470 constituent Substances 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 125000004122 cyclic group Chemical group 0.000 claims 1
- 230000002452 interceptive effect Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 230000008488 polyadenylation Effects 0.000 claims 1
- 108091033319 polynucleotide Proteins 0.000 claims 1
- 102000040430 polynucleotide Human genes 0.000 claims 1
- 239000002157 polynucleotide Substances 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 230000010076 replication Effects 0.000 claims 1
- 108091092562 ribozyme Proteins 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021130440A JP7316326B2 (ja) | 2015-05-14 | 2021-08-10 | スペーサーを含む核酸分子およびその使用の方法 |
| JP2023115773A JP2023138527A (ja) | 2015-05-14 | 2023-07-14 | スペーサーを含む核酸分子およびその使用の方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562161505P | 2015-05-14 | 2015-05-14 | |
| US62/161,505 | 2015-05-14 | ||
| PCT/US2016/032321 WO2016183422A1 (en) | 2015-05-14 | 2016-05-13 | Nucleic acid molecules containing spacers and methods of use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021130440A Division JP7316326B2 (ja) | 2015-05-14 | 2021-08-10 | スペーサーを含む核酸分子およびその使用の方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018515102A JP2018515102A (ja) | 2018-06-14 |
| JP2018515102A5 true JP2018515102A5 (enExample) | 2019-06-13 |
| JP6929230B2 JP6929230B2 (ja) | 2021-09-01 |
Family
ID=57249066
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017559315A Active JP6929230B2 (ja) | 2015-05-14 | 2016-05-13 | スペーサーを含む核酸分子およびその使用の方法 |
| JP2021130440A Active JP7316326B2 (ja) | 2015-05-14 | 2021-08-10 | スペーサーを含む核酸分子およびその使用の方法 |
| JP2023115773A Pending JP2023138527A (ja) | 2015-05-14 | 2023-07-14 | スペーサーを含む核酸分子およびその使用の方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021130440A Active JP7316326B2 (ja) | 2015-05-14 | 2021-08-10 | スペーサーを含む核酸分子およびその使用の方法 |
| JP2023115773A Pending JP2023138527A (ja) | 2015-05-14 | 2023-07-14 | スペーサーを含む核酸分子およびその使用の方法 |
Country Status (5)
| Country | Link |
|---|---|
| US (2) | US11103597B2 (enExample) |
| EP (1) | EP3294309A4 (enExample) |
| JP (3) | JP6929230B2 (enExample) |
| AU (3) | AU2016260401B2 (enExample) |
| WO (1) | WO2016183422A1 (enExample) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11103597B2 (en) | 2015-05-14 | 2021-08-31 | St. Jude Children's Research Hospital, Inc. | Nucleic acid molecules containing spacers outside ITR |
| WO2017066579A1 (en) | 2015-10-14 | 2017-04-20 | Audentes Therapeutics, Inc. | Nucleic acid molecules containing spacers and methods of use thereof |
| WO2018191622A1 (en) * | 2017-04-14 | 2018-10-18 | Rhode Island Hospital | Vegf gene therapy for tendon and ligament injuries |
| WO2019032898A1 (en) | 2017-08-09 | 2019-02-14 | Bioverativ Therapeutics Inc. | NUCLEIC ACID MOLECULES AND USES THEREOF |
| CA3092088A1 (en) * | 2018-03-15 | 2019-09-19 | Igtx, Llc | Synthetic dna vectors and methods of use |
| SG11202101157VA (en) | 2018-08-09 | 2021-03-30 | Bioverativ Therapeutics Inc | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| US12383587B2 (en) | 2018-10-25 | 2025-08-12 | Takeda Pharmaceutical Company Limited | AAV triple-plasmid system |
| TW202124722A (zh) | 2019-09-18 | 2021-07-01 | 美商英特佳樂帝克醫療公司 | 合成dna載體及其使用方法 |
| CN115197967B (zh) * | 2021-04-08 | 2023-09-15 | 广州派真生物技术有限公司 | 制备重组腺相关病毒的辅助质粒及其应用 |
Family Cites Families (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| CA2264499A1 (en) | 1996-09-06 | 1998-03-12 | The Trustees Of The University Of Pennsylvania | Methods using cre-lox for production of recombinant adeno-associated viruses |
| US6346415B1 (en) * | 1997-10-21 | 2002-02-12 | Targeted Genetics Corporation | Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors |
| WO2001036623A2 (en) | 1999-11-05 | 2001-05-25 | Avigen, Inc. | Ecdysone-inducible adeno-associated virus expression vectors |
| US20020182595A1 (en) | 2001-04-27 | 2002-12-05 | Weitzman Matthew D. | Method of identifying cellular regulators of adeno-associated virus (AAV) |
| EP1402023A4 (en) | 2001-05-31 | 2005-06-15 | Univ Rockefeller | PROCESS FOR GENERATING REPLICATION-DEFICIENT VIRUS VECTORS THAT ARE HELPFUL-FREE |
| US7419817B2 (en) | 2002-05-17 | 2008-09-02 | The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. | Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography |
| WO2004029278A2 (en) | 2002-09-26 | 2004-04-08 | Greenville Hospital System | Aav itr-mediated modulation |
| DK2438931T3 (da) * | 2004-09-22 | 2013-12-02 | St Jude Childrens Res Hospital | Forbedret ekspression af faktor ix i genterapivektorer |
| ES2683695T3 (es) * | 2010-01-12 | 2018-09-27 | The University Of North Carolina At Chapel Hill | Repeticiones terminales invertidas restrictivas para vectores virales |
| US9249425B2 (en) | 2011-05-16 | 2016-02-02 | The Trustees Of The University Of Pennslyvania | Proviral plasmids and production of recombinant adeno-associated virus |
| TWI775096B (zh) * | 2012-05-15 | 2022-08-21 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| US9124527B2 (en) * | 2012-08-29 | 2015-09-01 | International Business Machines Corporation | Sliced routing table management |
| JP6454643B2 (ja) * | 2012-10-26 | 2019-01-16 | フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ | 血友病の肝臓指向性遺伝子治療のためのベクター並びにその方法及び使用 |
| HK1220488A1 (zh) | 2013-03-15 | 2017-05-05 | The Children's Hospital Of Philadelphia | 含有填充者/填充物多核苷酸序列的载体及其使用方法 |
| CN105228615A (zh) | 2013-03-15 | 2016-01-06 | 鲁美纳医药公司 | 用于治疗巴雷特食管和胃食管返流疾病的胆汁酸再循环抑制剂 |
| PH12016500162B1 (en) * | 2013-07-22 | 2024-02-21 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene trnsfer to cells, organs, and tissues |
| CN107427557B (zh) * | 2014-08-13 | 2022-01-04 | 费城儿童医院 | 用于包装和表达变体因子viii以治疗血友病的改良表达组件 |
| US11103597B2 (en) | 2015-05-14 | 2021-08-31 | St. Jude Children's Research Hospital, Inc. | Nucleic acid molecules containing spacers outside ITR |
-
2016
- 2016-05-13 US US15/573,684 patent/US11103597B2/en active Active
- 2016-05-13 AU AU2016260401A patent/AU2016260401B2/en active Active
- 2016-05-13 JP JP2017559315A patent/JP6929230B2/ja active Active
- 2016-05-13 EP EP16793594.9A patent/EP3294309A4/en active Pending
- 2016-05-13 WO PCT/US2016/032321 patent/WO2016183422A1/en not_active Ceased
-
2021
- 2021-08-10 JP JP2021130440A patent/JP7316326B2/ja active Active
- 2021-08-13 US US17/401,765 patent/US12453783B2/en active Active
-
2022
- 2022-08-12 AU AU2022215274A patent/AU2022215274A1/en not_active Abandoned
-
2023
- 2023-07-14 JP JP2023115773A patent/JP2023138527A/ja active Pending
-
2025
- 2025-05-05 AU AU2025203194A patent/AU2025203194A1/en active Pending
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