JP2021184734A5 - - Google Patents
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- Publication number
- JP2021184734A5 JP2021184734A5 JP2021130440A JP2021130440A JP2021184734A5 JP 2021184734 A5 JP2021184734 A5 JP 2021184734A5 JP 2021130440 A JP2021130440 A JP 2021130440A JP 2021130440 A JP2021130440 A JP 2021130440A JP 2021184734 A5 JP2021184734 A5 JP 2021184734A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- acid molecule
- cell
- itr2
- itr1
- 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
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- 150000007523 nucleic acids Chemical class 0.000 claims 28
- 108020004707 nucleic acids Proteins 0.000 claims 26
- 102000039446 nucleic acids Human genes 0.000 claims 26
- 210000004027 cell Anatomy 0.000 claims 13
- 239000003623 enhancer Substances 0.000 claims 11
- 230000008488 polyadenylation Effects 0.000 claims 5
- 210000001519 tissue Anatomy 0.000 claims 5
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 4
- 125000006850 spacer group Chemical group 0.000 claims 4
- 108091027967 Small hairpin RNA Proteins 0.000 claims 3
- 210000004185 liver Anatomy 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 239000004055 small Interfering RNA Substances 0.000 claims 3
- 108700011259 MicroRNAs Proteins 0.000 claims 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- 230000001580 bacterial effect Effects 0.000 claims 2
- 210000003527 eukaryotic cell Anatomy 0.000 claims 2
- 230000002401 inhibitory effect Effects 0.000 claims 2
- 210000004962 mammalian cell Anatomy 0.000 claims 2
- 239000003550 marker Substances 0.000 claims 2
- 239000002679 microRNA Substances 0.000 claims 2
- 210000003205 muscle Anatomy 0.000 claims 2
- 210000005036 nerve Anatomy 0.000 claims 2
- 210000001236 prokaryotic cell Anatomy 0.000 claims 2
- 101150037123 APOE gene Proteins 0.000 claims 1
- 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
- 241000701022 Cytomegalovirus Species 0.000 claims 1
- 101100216294 Danio rerio apoeb gene Proteins 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 241000588724 Escherichia coli Species 0.000 claims 1
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 1
- 241000238631 Hexapoda Species 0.000 claims 1
- 101000899111 Homo sapiens Hemoglobin subunit beta Proteins 0.000 claims 1
- 239000003242 anti bacterial agent Substances 0.000 claims 1
- 230000003115 biocidal effect Effects 0.000 claims 1
- 108010006025 bovine growth hormone Proteins 0.000 claims 1
- 238000010367 cloning Methods 0.000 claims 1
- 239000003184 complementary RNA Substances 0.000 claims 1
- 230000002452 interceptive effect 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 (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| 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 | ||
| JP2017559315A 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 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017559315A Division 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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023115773A Division 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021184734A JP2021184734A (ja) | 2021-12-09 |
| JP2021184734A5 true JP2021184734A5 (enExample) | 2022-01-18 |
| JP7316326B2 JP7316326B2 (ja) | 2023-07-27 |
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 Before (1)
| 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
|---|---|---|---|---|
| AU2016260401B2 (en) | 2015-05-14 | 2022-05-19 | St. Jude Children's Research Hospital, Inc. | Nucleic acid molecules containing spacers and methods of use thereof |
| EP3361869B1 (en) * | 2015-10-14 | 2025-12-03 | 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 |
| KR102780287B1 (ko) | 2017-08-09 | 2025-03-14 | 바이오버라티브 테라퓨틱스 인크. | 핵산 분자 및 이의 용도 |
| CA3092088A1 (en) * | 2018-03-15 | 2019-09-19 | Igtx, Llc | Synthetic dna vectors and methods of use |
| BR112021002017A2 (pt) | 2018-08-09 | 2021-05-11 | Bioverativ Therapeutics Inc. | moléculas de ácido nucleico e usos das mesmas para terapia genética não viral |
| CA3112883A1 (en) | 2018-10-25 | 2020-04-30 | Baxalta Incorporated | Aav triple-plasmid system |
| CA3151464A1 (en) | 2019-09-18 | 2021-03-25 | Bruce C. SCHNEPP | Synthetic dna vectors and methods of use |
| 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 |
| JP2001500014A (ja) * | 1996-09-06 | 2001-01-09 | トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組換えアデノ随伴ウイルスの生産のためにｃｒｅ―ｌｏｘを用いる方法 |
| 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) |
| WO2002097056A2 (en) | 2001-05-31 | 2002-12-05 | The Rockefeller University | Method for generating replication defective viral vectors that are helper 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 |
| CA2500397A1 (en) | 2002-09-26 | 2004-04-08 | Greenville Hospital System | Aav itr-mediated modulation |
| EP1804839B1 (en) * | 2004-09-22 | 2012-03-14 | St. Jude Children's Research Hospital | Improved expression of factor ix in gene therapy vectors |
| 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 |
| EP2911687B1 (en) | 2012-10-26 | 2019-02-13 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| RU2015139732A (ru) | 2013-03-15 | 2017-04-24 | ЛУМЕНА ФАРМАСЬЮТИКАЛС ЭлЭлСи | Ингибиторы рециркуляции желчных кислот для лечения пищевода барретта и гастроэзофагеальной рефлюксной болезни |
| CA2904156C (en) * | 2013-03-15 | 2023-01-10 | The Children's Hospital Of Philadelphia | Vectors comprising stuffer/filler polynucleotide sequences and methods of use |
| CA2919103C (en) * | 2013-07-22 | 2023-10-10 | The Children's Hospital Of Philadelphia | Variant aav and compositions, methods and uses for gene transfer 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 |
| AU2016260401B2 (en) | 2015-05-14 | 2022-05-19 | St. Jude Children's Research Hospital, Inc. | Nucleic acid molecules containing spacers and methods of use thereof |
-
2016
- 2016-05-13 AU AU2016260401A patent/AU2016260401B2/en active Active
- 2016-05-13 US US15/573,684 patent/US11103597B2/en active Active
- 2016-05-13 WO PCT/US2016/032321 patent/WO2016183422A1/en not_active Ceased
- 2016-05-13 EP EP16793594.9A patent/EP3294309A4/en active Pending
- 2016-05-13 JP JP2017559315A patent/JP6929230B2/ja active Active
-
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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