JPWO2019246544A5 - - Google Patents
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- JPWO2019246544A5 JPWO2019246544A5 JP2020570894A JP2020570894A JPWO2019246544A5 JP WO2019246544 A5 JPWO2019246544 A5 JP WO2019246544A5 JP 2020570894 A JP2020570894 A JP 2020570894A JP 2020570894 A JP2020570894 A JP 2020570894A JP WO2019246544 A5 JPWO2019246544 A5 JP WO2019246544A5
- Authority
- JP
- Japan
- Prior art keywords
- cells
- item
- dna
- itr
- construct
- 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.)
- Pending
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- 210000004027 cells Anatomy 0.000 claims description 144
- 229920003013 deoxyribonucleic acid Polymers 0.000 claims description 135
- 150000007523 nucleic acids Chemical class 0.000 claims description 48
- 108020004707 nucleic acids Proteins 0.000 claims description 43
- 229920001850 Nucleic acid sequence Polymers 0.000 claims description 35
- 239000002773 nucleotide Substances 0.000 claims description 28
- 125000003729 nucleotide group Chemical group 0.000 claims description 28
- 230000002459 sustained Effects 0.000 claims description 23
- 229920001972 Concatemer Polymers 0.000 claims description 21
- 241000125945 Protoparvovirus Species 0.000 claims description 16
- 238000003780 insertion Methods 0.000 claims description 15
- 238000006467 substitution reaction Methods 0.000 claims description 15
- 230000000295 complement Effects 0.000 claims description 13
- 229920000160 (ribonucleotides)n+m Polymers 0.000 claims description 10
- 241000700605 Viruses Species 0.000 claims description 10
- 239000008194 pharmaceutical composition Substances 0.000 claims description 10
- 102000004169 proteins and genes Human genes 0.000 claims description 10
- 108090000623 proteins and genes Proteins 0.000 claims description 10
- 102000018120 Recombinases Human genes 0.000 claims description 9
- 108010091086 Recombinases Proteins 0.000 claims description 9
- 238000000338 in vitro Methods 0.000 claims description 8
- 230000001419 dependent Effects 0.000 claims description 7
- -1 RNAi Polymers 0.000 claims description 6
- 201000010099 disease Diseases 0.000 claims description 6
- 230000003612 virological Effects 0.000 claims description 6
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 5
- 229920000033 CRISPR Polymers 0.000 claims description 5
- 238000010354 CRISPR gene editing Methods 0.000 claims description 5
- 210000000349 Chromosomes Anatomy 0.000 claims description 5
- 210000002889 Endothelial Cells Anatomy 0.000 claims description 5
- 229920002391 Guide RNA Polymers 0.000 claims description 5
- 108020005004 Guide RNA Proteins 0.000 claims description 5
- 210000003494 Hepatocytes Anatomy 0.000 claims description 5
- 210000002510 Keratinocytes Anatomy 0.000 claims description 5
- 210000004072 Lung Anatomy 0.000 claims description 5
- 210000004413 Myocytes, Cardiac Anatomy 0.000 claims description 5
- 210000000329 Myocytes, Smooth Muscle Anatomy 0.000 claims description 5
- 229920000890 Palindromic sequence Polymers 0.000 claims description 5
- 210000002307 Prostate Anatomy 0.000 claims description 5
- 230000025458 RNA interference Effects 0.000 claims description 5
- 229920001891 Small hairpin RNA Polymers 0.000 claims description 5
- 210000000952 Spleen Anatomy 0.000 claims description 5
- 108020005202 Viral DNA Proteins 0.000 claims description 5
- 229930002945 all-trans-retinaldehyde Natural products 0.000 claims description 5
- 210000002449 bone cell Anatomy 0.000 claims description 5
- 201000011510 cancer Diseases 0.000 claims description 5
- 210000002919 epithelial cells Anatomy 0.000 claims description 5
- 210000003292 kidney cell Anatomy 0.000 claims description 5
- 229920001239 microRNA Polymers 0.000 claims description 5
- 230000002107 myocardial Effects 0.000 claims description 5
- 210000002569 neurons Anatomy 0.000 claims description 5
- OZAIFHULBGXAKX-UHFFFAOYSA-N precursor Substances N#CC(C)(C)N=NC(C)(C)C#N OZAIFHULBGXAKX-UHFFFAOYSA-N 0.000 claims description 5
- 238000005215 recombination Methods 0.000 claims description 5
- 230000000717 retained Effects 0.000 claims description 5
- 230000002207 retinal Effects 0.000 claims description 5
- 235000020945 retinal Nutrition 0.000 claims description 5
- 239000011604 retinal Substances 0.000 claims description 5
- 210000002363 skeletal muscle cell Anatomy 0.000 claims description 5
- 210000000130 stem cell Anatomy 0.000 claims description 5
- 230000001225 therapeutic Effects 0.000 claims description 5
- 210000004881 tumor cells Anatomy 0.000 claims description 5
- 210000001161 Embryo, Mammalian Anatomy 0.000 claims description 4
- 210000003969 blast cell Anatomy 0.000 claims description 4
- 230000002950 deficient Effects 0.000 claims description 4
- 239000000835 fiber Substances 0.000 claims description 4
- 239000000203 mixture Substances 0.000 claims 19
- 101700066277 COS-1 Proteins 0.000 claims 2
- 241000699802 Cricetulus griseus Species 0.000 claims 2
- 210000001672 Ovary Anatomy 0.000 claims 2
- 210000000234 Capsid Anatomy 0.000 claims 1
- 239000003153 chemical reaction reagent Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 102100008005 GPR180 Human genes 0.000 description 31
- 101710028548 GPR180 Proteins 0.000 description 31
- 230000024881 catalytic activity Effects 0.000 description 7
- 239000002679 microRNA Substances 0.000 description 3
- 239000004055 small Interfering RNA Substances 0.000 description 3
- 125000004122 cyclic group Chemical group 0.000 description 2
- 230000001939 inductive effect Effects 0.000 description 2
- 210000002966 Serum Anatomy 0.000 description 1
- 230000001580 bacterial Effects 0.000 description 1
- 238000004520 electroporation Methods 0.000 description 1
- 210000002308 embryonic cell Anatomy 0.000 description 1
- 210000002950 fibroblast Anatomy 0.000 description 1
- 238000001638 lipofection Methods 0.000 description 1
- 238000001890 transfection Methods 0.000 description 1
- 230000001131 transforming Effects 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024086213A JP2024112967A (ja) | 2018-06-22 | 2024-05-28 | 細胞内で存続する遺伝子送達のためのベクター |
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862688982P | 2018-06-22 | 2018-06-22 | |
| US62/688,982 | 2018-06-22 | ||
| US201862689453P | 2018-06-25 | 2018-06-25 | |
| US62/689,453 | 2018-06-25 | ||
| US201862697750P | 2018-07-13 | 2018-07-13 | |
| US62/697,750 | 2018-07-13 | ||
| US201862717310P | 2018-08-10 | 2018-08-10 | |
| US62/717,310 | 2018-08-10 | ||
| PCT/US2019/038515 WO2019246544A2 (fr) | 2018-06-22 | 2019-06-21 | Vecteurs pour l'administration de gènes qui persistent dans les cellules |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024086213A Division JP2024112967A (ja) | 2018-06-22 | 2024-05-28 | 細胞内で存続する遺伝子送達のためのベクター |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021528959A JP2021528959A (ja) | 2021-10-28 |
| JPWO2019246544A5 true JPWO2019246544A5 (fr) | 2022-06-27 |
Family
ID=67297281
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020570894A Pending JP2021528959A (ja) | 2018-06-22 | 2019-06-21 | 細胞内で存続する遺伝子送達のためのベクター |
| JP2024086213A Pending JP2024112967A (ja) | 2018-06-22 | 2024-05-28 | 細胞内で存続する遺伝子送達のためのベクター |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024086213A Pending JP2024112967A (ja) | 2018-06-22 | 2024-05-28 | 細胞内で存続する遺伝子送達のためのベクター |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20210269828A1 (fr) |
| EP (1) | EP3810782A2 (fr) |
| JP (2) | JP2021528959A (fr) |
| CN (1) | CN112639110A (fr) |
| AU (1) | AU2019290228A1 (fr) |
| CA (1) | CA3104113A1 (fr) |
| WO (1) | WO2019246544A2 (fr) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2018313921B2 (en) | 2017-08-09 | 2024-12-19 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof |
| US20220243212A1 (en) * | 2019-06-21 | 2022-08-04 | Asklepios Biopharmaceutical, Inc. | Production of vectors using phage origin of replication |
| CZ308738B6 (cs) * | 2020-01-20 | 2021-04-14 | PortalGene s.r.o. | Způsob úpravy molekuly nukleové kyseliny pro usnadění jejího přenosu do hostitelských buněk a upravená molekula nukleové kyseliny |
| AU2021244559A1 (en) * | 2020-03-24 | 2022-11-17 | Generation Bio Co. | Non-viral DNA vectors and uses thereof for expressing Gaucher therapeutics |
| CN115667530A (zh) * | 2020-03-24 | 2023-01-31 | 世代生物公司 | 非病毒dna载体和其用于表达因子ix治疗剂的用途 |
| US20230272432A1 (en) | 2020-07-27 | 2023-08-31 | Anjarium Biosciences Ag | Compositions of dna molecules, methods of making therefor, and methods of use thereof |
| GB202014751D0 (en) | 2020-09-18 | 2020-11-04 | Lightbio Ltd | Targeting vector |
| EP4317424A1 (fr) * | 2021-03-29 | 2024-02-07 | Kaneka Corporation | Vecteur, procédé de préparation d'adn linéaire fermé à liaison covalente l'utilisant, procédé de préparation de vecteur parvoviral et cellule de production de vecteur parvoviral |
| AU2022211795A1 (en) * | 2021-08-06 | 2023-02-23 | Syte.Bio Inc. | Hairpin loop ended self-complementary double-stranded covalently closed linear dna vector, manufacturing system and process, and uses of said resulting dna vector |
| CN116606834A (zh) * | 2023-04-10 | 2023-08-18 | 天津中合基因科技有限公司 | 多种具有切割连接活性的原端酶及其应用 |
| US20240376494A1 (en) * | 2023-05-08 | 2024-11-14 | Wisconsin Alumni Research Foundation | Recombinant aav constructs for increased transgene expression |
Family Cites Families (46)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4946787A (en) | 1985-01-07 | 1990-08-07 | Syntex (U.S.A.) Inc. | N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US5049386A (en) | 1985-01-07 | 1991-09-17 | Syntex (U.S.A.) Inc. | N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4795700A (en) | 1985-01-25 | 1989-01-03 | California Institute Of Technology | Nucleic acid probes and methods of using same |
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US4968603A (en) | 1986-12-31 | 1990-11-06 | The Regents Of The University Of California | Determination of status in neoplastic disease |
| WO1990005142A1 (fr) | 1988-11-10 | 1990-05-17 | Imperial Cancer Research Technology Ltd. | Polypeptides |
| US5194376A (en) | 1989-02-28 | 1993-03-16 | University Of Ottawa | Baculovirus expression system capable of producing foreign gene proteins at high levels |
| FR2675803B1 (fr) | 1991-04-25 | 1996-09-06 | Genset Sa | Oligonucleotides fermes, antisens et sens et leurs applications. |
| US5478745A (en) * | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869248A (en) | 1994-03-07 | 1999-02-09 | Yale University | Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences |
| US5599706A (en) | 1994-09-23 | 1997-02-04 | Stinchcomb; Dan T. | Ribozymes targeted to apo(a) mRNA |
| US6040183A (en) | 1995-06-07 | 2000-03-21 | University Of North Carloina At Chapel Hill | Helper virus-free AAV production |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US6083702A (en) | 1995-12-15 | 2000-07-04 | Intronn Holdings Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| CA2240494C (fr) | 1995-12-15 | 2007-03-13 | Lloyd G. Mitchell | Molecules therapeutiques produites par trans-epissure |
| US5962313A (en) | 1996-01-18 | 1999-10-05 | Avigen, Inc. | Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme |
| EP0924298A1 (fr) | 1997-12-18 | 1999-06-23 | Stichting Instituut voor Dierhouderij en Diergezondheid (ID-DLO) | Expression de proteine dans des systèmes d'expression faisant appel aux baculovirus |
| US6225060B1 (en) | 1998-03-04 | 2001-05-01 | Onyx Pharmaceuticals, Inc. | Baculovirus expression system and method for high throughput expression of genetic material |
| WO1999064577A1 (fr) * | 1998-06-09 | 1999-12-16 | Merck & Co., Inc. | Nouveaux vecteurs adenoviraux utilises en therapie genique |
| DE19826758C1 (de) | 1998-06-15 | 1999-10-21 | Soft Gene Gmbh | Darstellung von linearen kovalent geschlossenen DNA-Molekülen als Expressionskonstrukte |
| DE19935756A1 (de) | 1999-07-27 | 2001-02-08 | Mologen Forschungs Entwicklung | Kovalent geschlossenes Nukleinsäuremolekül zur Immunstimulation |
| JP4860886B2 (ja) | 2000-06-01 | 2012-01-25 | ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 二本鎖パルボウイルスベクター |
| US6623729B2 (en) | 2001-07-09 | 2003-09-23 | Korea Advanced Institute Of Science And Technology | Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer |
| ITRM20020253A1 (it) | 2002-05-08 | 2003-11-10 | Univ Roma | Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| IL166612A0 (en) | 2002-08-29 | 2006-01-15 | Univ Leland Stanford Junior | Circular nucleic acid vectors and methods for making and using the same |
| FR2874384B1 (fr) | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
| US7892824B2 (en) | 2007-01-18 | 2011-02-22 | University Of Missouri-Columbia | Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| KR101414713B1 (ko) | 2007-10-11 | 2014-07-03 | 삼성전자주식회사 | 리가제 및 엔도뉴클레아제의 존재하에서 롤링서클 증폭에의하여 표적 핵산을 증폭하는 방법 |
| GB0901593D0 (en) * | 2009-01-30 | 2009-03-11 | Touchlight Genetics Ltd | Production of closed linear DNA |
| KR101607702B1 (ko) | 2009-05-29 | 2016-03-31 | 삼성디스플레이 주식회사 | 액정 표시 장치 |
| EP2524037B1 (fr) | 2010-01-12 | 2018-05-16 | The University Of North Carolina At Chapel Hill | Répétitions terminales inversées restrictives pour vecteurs viraux |
| GB201013153D0 (en) | 2010-08-04 | 2010-09-22 | Touchlight Genetics Ltd | Primer for production of closed linear DNA |
| WO2012109570A1 (fr) | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Vecteurs viraux à profil de transduction modifiée et procédés pour les préparer et les utiliser |
| EP2500434A1 (fr) * | 2011-03-12 | 2012-09-19 | Association Institut de Myologie | Vecteurs AAV sans capside, compositions et procédés pour la production des vecteurs et la thérapie génique |
| JP6165752B2 (ja) | 2011-10-28 | 2017-07-19 | ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | アデノ随伴ウイルスの産生のための細胞株 |
| US9862954B2 (en) * | 2012-11-22 | 2018-01-09 | Mediphage Bioceuticals, Inc. | DNA vector production system |
| US9290778B2 (en) * | 2013-11-22 | 2016-03-22 | Mediphage Bioceuticals, Inc. | DNA vector production system |
| JP2015014459A (ja) | 2013-07-03 | 2015-01-22 | 日本電波工業株式会社 | 放射線測定装置 |
| EP3221456B1 (fr) * | 2014-11-21 | 2021-09-22 | University of Florida Research Foundation, Inc. | Vecteurs viraux adéno-associés recombinés au génome modifié |
| CA3002524A1 (fr) * | 2015-10-28 | 2017-05-04 | Sangamo Therapeutics, Inc. | Constructions specifiques au foie, cassettes d'expression du facteur viii et leurs methodes d'utilisation |
| ES2898337T3 (es) * | 2016-03-03 | 2022-03-07 | Univ Massachusetts | ADN dúplex lineal de extremos cerrados para la transferencia de genes no víricos |
| CN109475646A (zh) | 2016-03-30 | 2019-03-15 | 因特利亚治疗公司 | 用于crispr/cas成分的脂质纳米颗粒制剂 |
-
2019
- 2019-06-21 EP EP19740133.4A patent/EP3810782A2/fr active Pending
- 2019-06-21 JP JP2020570894A patent/JP2021528959A/ja active Pending
- 2019-06-21 US US17/253,929 patent/US20210269828A1/en active Pending
- 2019-06-21 AU AU2019290228A patent/AU2019290228A1/en active Pending
- 2019-06-21 CA CA3104113A patent/CA3104113A1/fr active Pending
- 2019-06-21 CN CN201980055070.8A patent/CN112639110A/zh active Pending
- 2019-06-21 WO PCT/US2019/038515 patent/WO2019246544A2/fr active Application Filing
-
2024
- 2024-05-28 JP JP2024086213A patent/JP2024112967A/ja active Pending
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