JP2021184734A5 - - Google Patents
Download PDFInfo
- 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
Links
- 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 JP7796702B2 (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 JP7796702B2 (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 (https=) | 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 Active JP7796702B2 (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 Active JP7796702B2 (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 (https=) |
| EP (1) | EP3294309A4 (https=) |
| JP (3) | JP6929230B2 (https=) |
| AU (3) | AU2016260401B2 (https=) |
| WO (1) | WO2016183422A1 (https=) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3294309A4 (en) | 2015-05-14 | 2019-01-16 | St. Jude Children's Research Hospital, Inc. | NUCLEIC ACID MOLECULES WITH SPACERS AND METHOD FOR USE THEREOF |
| CA3001594A1 (en) | 2015-10-14 | 2017-04-20 | Audentes Therapeutics, Inc. | Nucleic acid molecules containing spacers and methods of use thereof |
| US20200113972A1 (en) * | 2017-04-14 | 2020-04-16 | Rhode Island Hospital | Vegf gene therapy for tendon and ligament injuries |
| NZ762100A (en) | 2017-08-09 | 2025-12-19 | Bioverativ Therapeutics Inc | Nucleic acid molecules and uses thereof |
| US20210002667A1 (en) * | 2018-03-15 | 2021-01-07 | Intergalactic Therapeutics, Inc. | Synthetic dna vectors and methods of use |
| EP3833766A1 (en) | 2018-08-09 | 2021-06-16 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| CA3112883A1 (en) | 2018-10-25 | 2020-04-30 | Baxalta Incorporated | Aav triple-plasmid system |
| KR20220128607A (ko) | 2019-09-18 | 2022-09-21 | 인터갈락틱 테라퓨틱스, 인크. | 합성 dna 벡터 및 사용 방법 |
| 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 |
| AU722375B2 (en) | 1996-09-06 | 2000-08-03 | Trustees Of The University Of Pennsylvania, The | 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 |
| AU3642601A (en) * | 1999-11-05 | 2001-05-30 | 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) |
| US8067156B2 (en) | 2001-05-31 | 2011-11-29 | 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 |
| US20040137626A1 (en) * | 2002-09-26 | 2004-07-15 | Greenville Hospital System | AAV ITR-mediated modulation |
| WO2006036502A2 (en) * | 2004-09-22 | 2006-04-06 | St. Jude Children's Research Hospital | Improved expression of factor ix in gene therapy vectors |
| EP2524037B1 (en) | 2010-01-12 | 2018-05-16 | The University Of North Carolina At Chapel Hill | Restrictive inverted terminal repeats for viral vectors |
| US9249425B2 (en) | 2011-05-16 | 2016-02-02 | The Trustees Of The University Of Pennslyvania | Proviral plasmids and production of recombinant adeno-associated virus |
| TWI698240B (zh) | 2012-05-15 | 2020-07-11 | 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 |
| AU2013336601B2 (en) | 2012-10-26 | 2018-01-25 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| 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 |
| AU2014229050A1 (en) | 2013-03-15 | 2015-10-22 | Lumena Pharmaceuticals Llc | Bile acid recycling inhibitors for treatment of Barrett's esophagus and gastroesophageal reflux disease |
| IL297919A (en) * | 2013-07-22 | 2023-01-01 | Childrens Hospital 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 |
| EP3294309A4 (en) | 2015-05-14 | 2019-01-16 | St. Jude Children's Research Hospital, Inc. | NUCLEIC ACID MOLECULES WITH SPACERS AND METHOD FOR USE THEREOF |
-
2016
- 2016-05-13 EP EP16793594.9A patent/EP3294309A4/en active Pending
- 2016-05-13 AU AU2016260401A patent/AU2016260401B2/en active Active
- 2016-05-13 JP JP2017559315A patent/JP6929230B2/ja 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
-
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/JP7796702B2/ja active Active
-
2025
- 2025-05-05 AU AU2025203194A patent/AU2025203194A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2021184734A5 (https=) | ||
| Li et al. | Synthetic muscle promoters: activities exceeding naturally occurring regulatory sequences | |
| Uno et al. | Arabidopsis basic leucine zipper transcription factors involved in an abscisic acid-dependent signal transduction pathway under drought and high-salinity conditions | |
| Whitehouse et al. | Antagonistic forces that position nucleosomes in vivo | |
| JP2018515102A5 (https=) | ||
| HRP20241180T1 (hr) | Modificirani faktor ix, te pripravci, postupci i upotreba za prijenos gena u stanice, organe i tkiva | |
| Suzuki et al. | Rice MYB protein OSMYB5 specifically binds to the AACA motif conserved among promoters of genes for storage protein glutelin | |
| JP7796702B2 (ja) | スペーサーを含む核酸分子およびその使用の方法 | |
| Wang et al. | Impact of different promoters on episomal vectors harbouring characteristic motifs of matrix attachment regions | |
| Richardson et al. | Requirement for either early region 1a or early region 1b adenovirus gene products in the helper effect for adeno-associated virus | |
| JP2021534814A5 (https=) | ||
| Suzuki et al. | Preferable sites and orientations of transgene inserted in the adenovirus vector genome: The E3 site may be unfavorable for transgene position | |
| Cooper | Muscle-specific splicing of a heterologous exon mediated by a single muscle-specific splicing enhancer from the cardiac troponin T gene | |
| WO2020142714A1 (en) | Aav expression cassette and aav vectors comprising the same | |
| CN102234647B (zh) | 一个水稻逆境诱导启动子kt619p的鉴定和应用 | |
| Buvoli et al. | Potential limitations of transcription terminators used as transgene insulators in adenoviral vectors | |
| Haun et al. | Characterization of the human ADP-ribosylation factor 3 promoter. Transcriptional regulation of a TATA-less promoter. | |
| Strauss et al. | The MDR1 downstream promoter contains sequence-specific binding sites for wild-type p53 | |
| CN116981776A (zh) | 使用合成启动子控制多基因表达 | |
| Astell et al. | Sequence of the 3′ terminus of the genome from kilham rat virus, a nondefective parvovirus | |
| CN102725408B (zh) | 猪肌抑素基因启动子及其应用 | |
| Rockhold et al. | Structure of two Solanum bulbocastanum polyubiquitin genes and expression of their promoters in transgenic potatoes | |
| Kucharova et al. | Design and optimization of short DNA sequences that can be used as 5′ fusion partners for high-level expression of heterologous genes in Escherichia coli | |
| Forde | AT-rich elements (ATREs) in the promoter regions of nodulin and other higher plant genes: a novel class of cis-acting regulatory element? | |
| CN103194450A (zh) | 一种提高肌细胞生成素（MyoG）基因启动子活性的方法 |