JPWO2019195423A5 - - Google Patents
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- JPWO2019195423A5 JPWO2019195423A5 JP2020552312A JP2020552312A JPWO2019195423A5 JP WO2019195423 A5 JPWO2019195423 A5 JP WO2019195423A5 JP 2020552312 A JP2020552312 A JP 2020552312A JP 2020552312 A JP2020552312 A JP 2020552312A JP WO2019195423 A5 JPWO2019195423 A5 JP WO2019195423A5
- Authority
- JP
- Japan
- Prior art keywords
- capsid protein
- growth factor
- aav
- factor
- aav vector
- 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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- 102100023321 Ceruloplasmin Human genes 0.000 claims 13
- 239000013607 AAV vector Substances 0.000 claims 11
- 150000001413 amino acids Chemical class 0.000 claims 8
- 150000007523 nucleic acids Chemical class 0.000 claims 7
- 108020004707 nucleic acids Proteins 0.000 claims 6
- 102000039446 nucleic acids Human genes 0.000 claims 6
- -1 Erythropoetin Proteins 0.000 claims 5
- 239000008194 pharmaceutical composition Substances 0.000 claims 4
- 238000006467 substitution reaction Methods 0.000 claims 4
- 101710163270 Nuclease Proteins 0.000 claims 3
- 210000004027 cell Anatomy 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 238000010362 genome editing Methods 0.000 claims 3
- 239000003102 growth factor Substances 0.000 claims 3
- 239000002773 nucleotide Substances 0.000 claims 3
- 125000003729 nucleotide group Chemical group 0.000 claims 3
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- 201000000053 blastoma Diseases 0.000 claims 2
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- 201000008184 embryoma Diseases 0.000 claims 2
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Priority Applications (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
JP2024006511A JP2024054142A (ja) | 2018-04-03 | 2024-01-19 | 眼組織を標的とするウイルスベクター |
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862652108P | 2018-04-03 | 2018-04-03 | |
US62/652,108 | 2018-04-03 | ||
PCT/US2019/025584 WO2019195423A1 (en) | 2018-04-03 | 2019-04-03 | Virus vectors for targeting ophthalmic tissues |
Related Child Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JP2024006511A Division JP2024054142A (ja) | 2018-04-03 | 2024-01-19 | 眼組織を標的とするウイルスベクター |
Publications (3)
Publication Number | Publication Date |
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JP2021519583A JP2021519583A (ja) | 2021-08-12 |
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Families Citing this family (11)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
ES2865487T3 (es) | 2015-09-28 | 2021-10-15 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores virales que evaden los anticuerpos |
BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
BR112020020195A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
EP3775173A1 (en) * | 2018-04-05 | 2021-02-17 | Genethon | Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism |
AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
CA3157700A1 (en) | 2019-10-17 | 2021-04-22 | Stridebio, Inc. | Adeno-associated viral vectors for treatment of niemann-pick disease type c |
TWI819268B (zh) * | 2020-01-16 | 2023-10-21 | 詹姆斯 W 希爾 | 藉由基因轉導改變眼睛顏色 |
WO2022018516A1 (en) * | 2020-07-21 | 2022-01-27 | Inspirar Limited | Composition and method for treating eye diseases |
TW202242124A (zh) | 2021-01-14 | 2022-11-01 | 美商史崔德生物公司 | 靶向t細胞之aav載體 |
CN117801116A (zh) * | 2022-09-30 | 2024-04-02 | 上海玮美基因科技有限责任公司 | 一种融合型新型腺相关病毒及其应用 |
WO2024124019A2 (en) | 2022-12-07 | 2024-06-13 | Ginkgo Bioworks, Inc. | Aav vectors targeting hematopoietic stem cells |
Family Cites Families (43)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4501729A (en) | 1982-12-13 | 1985-02-26 | Research Corporation | Aerosolized amiloride treatment of retained pulmonary secretions |
WO1988002026A1 (en) | 1986-09-08 | 1988-03-24 | Applied Biotechnology, Inc. | Empty viral capsid vaccines |
US4968603A (en) | 1986-12-31 | 1990-11-06 | The Regents Of The University Of California | Determination of status in neoplastic disease |
WO1990005142A1 (en) | 1988-11-10 | 1990-05-17 | Imperial Cancer Research Technology Ltd. | Polypeptides |
US5916563A (en) | 1988-11-14 | 1999-06-29 | United States Of America | Parvovirus protein presenting capsids |
US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
ES2026826A6 (es) | 1991-03-26 | 1992-05-01 | Ercros Sa | Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados. |
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 |
US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
US5599706A (en) | 1994-09-23 | 1997-02-04 | Stinchcomb; Dan T. | Ribozymes targeted to apo(a) mRNA |
US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
US6040183A (en) | 1995-06-07 | 2000-03-21 | University Of North Carloina At Chapel Hill | Helper virus-free AAV production |
ES2337853T3 (es) | 1995-06-07 | 2010-04-29 | University Of North Carolina At Chapel Hill | Transduccion de mioblastos por vaa. |
JP4321877B2 (ja) | 1995-12-15 | 2009-08-26 | バークシス コーポレイション | トランス―スプライスにより生成される治療用分子 |
US6083702A (en) | 1995-12-15 | 2000-07-04 | Intronn Holdings Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
US20030215422A1 (en) | 1996-09-11 | 2003-11-20 | John A. Chiorini | Aav4 vector and uses thereof |
US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
WO1999061601A2 (en) | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
CA2347129A1 (en) | 1998-09-22 | 2000-03-30 | University Of Florida | Methods for large-scale production of recombinant aav vectors |
ATE362542T1 (de) | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
CA2348382C (en) | 1998-11-10 | 2013-09-17 | The University Of North Carolina At Chapel Hill | Chimeric parvovirus vectors and methods of making and administering the same |
US7314912B1 (en) | 1999-06-21 | 2008-01-01 | Medigene Aktiengesellschaft | AAv scleroprotein, production and use thereof |
CA2410828C (en) | 2000-06-01 | 2012-01-24 | University Of North Carolina At Chapel Hill | Duplexed parvovirus vectors |
EP1286703B1 (en) | 2000-06-01 | 2009-08-05 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
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 |
AU2003223766A1 (en) | 2002-04-30 | 2003-11-17 | University Of North Carolina At Chapel Hill | Secretion signal vectors |
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. |
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 |
JP4346526B2 (ja) | 2004-08-31 | 2009-10-21 | 株式会社東芝 | 半導体集積回路装置 |
US9114148B2 (en) | 2004-09-09 | 2015-08-25 | University Of Cincinnati | Modulating phosphatase activity in cardiac cells |
EP1828390B1 (en) | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
WO2006119137A1 (en) | 2005-04-29 | 2006-11-09 | The University Of North Carolina At Chapel Hill | Methods and compositions for regulated expression of nucleic acid at post-transcriptional level |
WO2007100465A2 (en) | 2006-02-10 | 2007-09-07 | The University Of Cincinnati | Phosphatase inhibitor protein-1 as a regulator of cardiac function |
JP5575486B2 (ja) | 2007-01-18 | 2014-08-20 | ユニヴァーシティ オブ ミズーリー−コロンビア | 筋鞘にnNOSを回復させる合成ミニ/ミクロジストロフィン遺伝子 |
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 |
ES2634118T3 (es) | 2009-02-11 | 2017-09-26 | The University Of North Carolina At Chapel Hill | Vectores de virus modificados y métodos para fabricar y utilizar los mismos |
DK3693025T3 (da) * | 2011-04-22 | 2022-01-10 | Univ California | Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf |
PL2968605T3 (pl) | 2013-03-15 | 2022-11-07 | The University Of North Carolina At Chapel Hill | Metody i kompozycje dla wektorów aav podwójnie wiążących glikan |
WO2015038958A1 (en) | 2013-09-13 | 2015-03-19 | California Institute Of Technology | Selective recovery |
EP3245220B1 (en) * | 2015-01-14 | 2023-09-20 | The University of North Carolina at Chapel Hill | Methods and compositions for targeted gene transfer |
WO2016134338A1 (en) * | 2015-02-19 | 2016-08-25 | University Of Florida Research Foundation, Inc. | Recombinant aav vectors for gene therapy of human hematopoietic disorders |
RU2758488C2 (ru) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
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2019
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- 2019-04-03 CN CN201980035122.5A patent/CN112533644A/zh active Pending
- 2019-04-03 WO PCT/US2019/025584 patent/WO2019195423A1/en unknown
- 2019-04-03 EP EP19721906.6A patent/EP3773743A1/en active Pending
- 2019-04-03 BR BR112020020341-6A patent/BR112020020341A2/pt unknown
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- 2019-04-03 CA CA3094465A patent/CA3094465A1/en active Pending
- 2019-04-03 US US17/045,087 patent/US20210371469A1/en active Pending
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- 2019-04-03 AU AU2019247191A patent/AU2019247191A1/en active Pending
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