JP2022501044A5 - - Google Patents
Info
- Publication number
- JP2022501044A5 JP2022501044A5 JP2021515639A JP2021515639A JP2022501044A5 JP 2022501044 A5 JP2022501044 A5 JP 2022501044A5 JP 2021515639 A JP2021515639 A JP 2021515639A JP 2021515639 A JP2021515639 A JP 2021515639A JP 2022501044 A5 JP2022501044 A5 JP 2022501044A5
- Authority
- JP
- Japan
- Prior art keywords
- polynucleotide
- particle
- seq
- nucleotide sequence
- ribozyme
- 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
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862734435P | 2018-09-21 | 2018-09-21 | |
| US62/734,435 | 2018-09-21 | ||
| PCT/US2019/052272 WO2020061528A1 (en) | 2018-09-21 | 2019-09-20 | Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022501044A JP2022501044A (ja) | 2022-01-06 |
| JP2022501044A5 true JP2022501044A5 (cg-RX-API-DMAC10.html) | 2022-09-28 |
| JP7546295B2 JP7546295B2 (ja) | 2024-09-06 |
Family
ID=69887937
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021515639A Active JP7546295B2 (ja) | 2018-09-21 | 2019-09-20 | ヒトアンジェルマン症候群において父方ｕｂｅ３ａ遺伝子発現を復活させるための組成物および方法 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US12221609B2 (cg-RX-API-DMAC10.html) |
| EP (1) | EP3833398A4 (cg-RX-API-DMAC10.html) |
| JP (1) | JP7546295B2 (cg-RX-API-DMAC10.html) |
| KR (1) | KR102782761B1 (cg-RX-API-DMAC10.html) |
| CN (2) | CN119592561A (cg-RX-API-DMAC10.html) |
| AU (2) | AU2019344006B2 (cg-RX-API-DMAC10.html) |
| CA (1) | CA3113648A1 (cg-RX-API-DMAC10.html) |
| IL (1) | IL281563B2 (cg-RX-API-DMAC10.html) |
| MX (1) | MX2021003303A (cg-RX-API-DMAC10.html) |
| WO (1) | WO2020061528A1 (cg-RX-API-DMAC10.html) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3833398A4 (en) | 2018-09-21 | 2021-12-22 | University of Connecticut | COMPOSITIONS AND METHODS OF RESTORING PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN'S SYNDROME IN HUMANS |
| WO2021222232A1 (en) * | 2020-04-28 | 2021-11-04 | The Trustees Of The University Of Pennsylvania | Compositions and uses thereof for treatment of angelman syndrome |
| US20240318198A1 (en) * | 2021-02-03 | 2024-09-26 | The Board Of Regents Of The University Of Texas System | Gene therapy for angelman syndrome |
| CN119384504A (zh) * | 2022-03-07 | 2025-01-28 | 康涅狄格大学 | 靶向SNORD115位置以恢复快乐木偶综合征中父系UBE3A基因表达的shRNA |
| KR102986878B1 (ko) | 2022-04-27 | 2026-07-06 | 중앙대학교 산학협력단 | CRISPR-Cas12f1 시스템을 이용한 유전체의 단일 뉴클레오타이드 편집 방법 및 이의 용도 |
Family Cites Families (40)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| FR2705686B1 (fr) | 1993-05-28 | 1995-08-18 | Transgene Sa | Nouveaux adénovirus défectifs et lignées de complémentation correspondantes. |
| FR2711523B1 (fr) | 1993-10-26 | 1996-02-16 | Transgene Sa | Procédé de préparation d'un aérosol viral. |
| JPH10501681A (ja) | 1994-02-22 | 1998-02-17 | ダナ−ファーバー キャンサー インスティチュート | 核酸送達システムならびにその合成および使用方法 |
| US5837520A (en) | 1995-03-07 | 1998-11-17 | Canji, Inc. | Method of purification of viral vectors |
| FR2737222B1 (fr) | 1995-07-24 | 1997-10-17 | Transgene Sa | Nouveaux vecteurs viraux et lignee pour la therapie genique |
| US5898031A (en) | 1996-06-06 | 1999-04-27 | Isis Pharmaceuticals, Inc. | Oligoribonucleotides for cleaving RNA |
| JP2000514290A (ja) | 1996-07-01 | 2000-10-31 | ローヌ―プーラン・ロレ・エス・アー | 組換えアデノウイルスの製造方法 |
| EP0968284B1 (en) | 1996-11-20 | 2006-12-13 | Introgen Therapeutics, Inc. | An improved method for the production and purification of adenoviral vectors |
| IL130198A (en) | 1996-12-13 | 2005-09-25 | Schering Corp | Methods for purifying viruses |
| FR2763958A1 (fr) | 1997-05-29 | 1998-12-04 | Transgene Sa | Produit de combinaison associant un acide nucleique a une substance desorganisant la matrice extracellulaire pour la therapie genique |
| AU707186B2 (en) | 1997-07-01 | 1999-07-01 | Transgene S.A. | Compositions useful for transferring therapeutically active substances into a target cell, and their use in gene therapy |
| AU752658B2 (en) | 1998-05-06 | 2002-09-26 | Association Francaise Contre Les Myopathies | Use of a nuclease inhibitor or interleukin-10 (IL-10) for the preparation of a therapeutic composition for improving transfection of a polynucleotide into a cell and compositions useful in gene therapy |
| JP3864610B2 (ja) | 1998-05-21 | 2007-01-10 | 旭硝子株式会社 | 水分散型撥水撥油剤組成物およびその製造方法 |
| EP0998945A1 (en) | 1998-09-30 | 2000-05-10 | Transgene S.A. | Use of magnesium (Mg2+) for the enhancement of gene delivery in gene therapy |
| AU779267B2 (en) | 1998-12-31 | 2005-01-13 | Centelion S.A.S. | Method for separating viral particles |
| CA2364934C (fr) | 1999-02-22 | 2011-10-18 | Transgene S.A. | Procede d'obtention d'une preparation virale purifiee |
| EP1156832A1 (en) | 1999-12-28 | 2001-11-28 | Transgene S.A. | Use of lithium (li?+ ) for the preparation of a composition for transfection of a polynucleotide into a cell and compositions useful in gene therapy |
| WO2004029212A2 (en) | 2002-09-25 | 2004-04-08 | University Of Massachusetts | In vivo gene silencing by chemically modified and stable sirna |
| AU2004221760B2 (en) | 2003-03-21 | 2010-03-18 | Roche Innovation Center Copenhagen A/S | Short interfering RNA (siRNA) analogues |
| ES2356154T3 (es) | 2003-07-21 | 2011-04-05 | Transgene S.A. | Citoquinas multifuncionales. |
| WO2005073378A1 (en) | 2004-01-30 | 2005-08-11 | Santaris Pharma A/S | MODIFIED SHORT INTERFERING RNA (MODIFIED siRNA) |
| US7361468B2 (en) * | 2004-07-02 | 2008-04-22 | Affymetrix, Inc. | Methods for genotyping polymorphisms in humans |
| US20080213891A1 (en) | 2004-07-21 | 2008-09-04 | Alnylam Pharmaceuticals, Inc. | RNAi Agents Comprising Universal Nucleobases |
| EP1948674A4 (en) | 2005-11-02 | 2009-02-04 | Protiva Biotherapeutics Inc | MODIFIED SIRNA MOLECULES AND APPLICATIONS THEREOF |
| WO2007124452A2 (en) | 2006-04-20 | 2007-11-01 | The Regents Of The University Of California | Methods for expressing multiple sirna and shrna from a single vector |
| WO2010030396A2 (en) | 2008-09-15 | 2010-03-18 | Stanford University | Novel shrna gene therapy for treatment of ischemic heart disease |
| WO2011111072A2 (en) | 2010-03-10 | 2011-09-15 | Transgene Biotek Ltd. | Adeno-associated virus ezh2 shrna (aav ezh2 shrna)therapy for breast cancer |
| US9163236B2 (en) | 2010-12-06 | 2015-10-20 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Pharmaceutical composition comprising NANOG SHRNA, and method of using NANOG SHRNA to treat cancer |
| SG10201604810UA (en) * | 2012-02-07 | 2016-08-30 | Global Bio Therapeutics Inc | Compartmentalized method of nucleic acid delivery and compositions and uses thereof |
| US20130259927A1 (en) | 2012-04-02 | 2013-10-03 | Gradalis, Inc. | Ewing's Sarcoma Bifunctional shRNA Design |
| ES2809199T3 (es) * | 2012-06-25 | 2021-03-03 | Ionis Pharmaceuticals Inc | Modulación de la expresión de UBE3A-ATS |
| CN105120901A (zh) | 2012-07-11 | 2015-12-02 | 宾夕法尼亚大学托管会 | Rpgr x染色体连锁视网膜退化的aav介导的基因治疗 |
| WO2014052855A1 (en) | 2012-09-27 | 2014-04-03 | Population Diagnostics, Inc. | Methods and compositions for screening and treating developmental disorders |
| WO2015089443A2 (en) * | 2013-12-12 | 2015-06-18 | The Brigham And Women's Hospital, Inc. | Treating neurodegenerative disease |
| WO2016086104A1 (en) * | 2014-11-25 | 2016-06-02 | Ionis Pharmaceuticals, Inc. | Modulation of ube3a-ats expression |
| HK1258434A1 (zh) * | 2015-11-12 | 2019-11-08 | F. Hoffmann-La Roche Ag | 来自灵长类物种的标准化神经元细胞测定试验 |
| WO2017189963A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| CN118950038A (zh) | 2018-09-07 | 2024-11-15 | 科慕埃弗西有限公司 | 使用含氯烃从氟卤化锑催化剂除去氟 |
| EP3833398A4 (en) | 2018-09-21 | 2021-12-22 | University of Connecticut | COMPOSITIONS AND METHODS OF RESTORING PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN'S SYNDROME IN HUMANS |
-
2019
- 2019-09-20 EP EP19861996.7A patent/EP3833398A4/en active Pending
- 2019-09-20 WO PCT/US2019/052272 patent/WO2020061528A1/en not_active Ceased
- 2019-09-20 KR KR1020217011023A patent/KR102782761B1/ko active Active
- 2019-09-20 JP JP2021515639A patent/JP7546295B2/ja active Active
- 2019-09-20 US US17/271,982 patent/US12221609B2/en active Active
- 2019-09-20 CN CN202411375936.2A patent/CN119592561A/zh active Pending
- 2019-09-20 CN CN201980076741.9A patent/CN113164624B/zh active Active
- 2019-09-20 AU AU2019344006A patent/AU2019344006B2/en active Active
- 2019-09-20 CA CA3113648A patent/CA3113648A1/en active Pending
- 2019-09-20 MX MX2021003303A patent/MX2021003303A/es unknown
- 2019-09-20 IL IL281563A patent/IL281563B2/en unknown
-
2024
- 2024-12-31 US US19/006,482 patent/US20250136994A1/en active Pending
-
2026
- 2026-01-28 AU AU2026200604A patent/AU2026200604A1/en active Pending
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