JP2021525531A5 - - Google Patents
Info
- Publication number
- JP2021525531A5 JP2021525531A5 JP2020567085A JP2020567085A JP2021525531A5 JP 2021525531 A5 JP2021525531 A5 JP 2021525531A5 JP 2020567085 A JP2020567085 A JP 2020567085A JP 2020567085 A JP2020567085 A JP 2020567085A JP 2021525531 A5 JP2021525531 A5 JP 2021525531A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- seq
- nucleotide sequence
- raav
- shrna
- Prior art date
Links
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862679585P | 2018-06-01 | 2018-06-01 | |
| US62/679,585 | 2018-06-01 | ||
| US201962809539P | 2019-02-22 | 2019-02-22 | |
| US62/809,539 | 2019-02-22 | ||
| PCT/US2019/035159 WO2019232517A1 (en) | 2018-06-01 | 2019-06-03 | Compositions and methods for treatment of dominant retinitis pigmentosa |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2021525531A JP2021525531A (ja) | 2021-09-27 |
| JP2021525531A5 true JP2021525531A5 (https=) | 2022-06-13 |
| JPWO2019232517A5 JPWO2019232517A5 (https=) | 2022-06-13 |
| JP7563694B2 JP7563694B2 (ja) | 2024-10-08 |
Family
ID=68698456
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020567085A Active JP7563694B2 (ja) | 2018-06-01 | 2019-06-03 | 優性網膜色素変性症の処置のための組成物および方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US12203074B2 (https=) |
| EP (1) | EP3802836A4 (https=) |
| JP (1) | JP7563694B2 (https=) |
| CN (2) | CN112204145B (https=) |
| AU (1) | AU2019276645B2 (https=) |
| CA (1) | CA3102095A1 (https=) |
| IL (1) | IL279091B1 (https=) |
| WO (1) | WO2019232517A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2021212686A1 (zh) * | 2020-04-21 | 2021-10-28 | 北京中因科技有限公司 | RHO-adRP基于基因编辑的方法和组合物 |
| CN114521214A (zh) * | 2020-07-24 | 2022-05-20 | 尔知渃米斯股份有限公司 | 视紫红质转录体特异性反式剪接核酶及其用途 |
| JP7334346B2 (ja) * | 2020-07-24 | 2023-08-28 | アールズィーノミクス・インコーポレイテッド | ロドプシン転写体に特異的なトランススプライシングリボザイム及びその用途 |
| US20220098615A1 (en) * | 2020-09-30 | 2022-03-31 | NGGT, Inc. | Dual functional expression vectors and methods of use thereof |
| WO2024211266A1 (en) * | 2023-04-07 | 2024-10-10 | University Of Houston System | Downregulation of rhodopsin as a therapeutic strategy for peripherin-2-associated inherited retinal disorders |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8551970B2 (en) | 1996-04-02 | 2013-10-08 | Optigen Patents Limited | Genetic suppression and replacement |
| US20040234999A1 (en) | 1996-04-02 | 2004-11-25 | Farrar Gwenyth Jane | Genetic suppression and replacement |
| GB9606961D0 (en) | 1996-04-02 | 1996-06-05 | Farrar Gwyneth J | Genetic strategy III |
| WO2004022722A2 (en) * | 2002-09-06 | 2004-03-18 | Massachusetts Institute Of Technology | Lentiviral vectors, related reagents, and methods of use thereof |
| CA2522730A1 (en) | 2003-04-18 | 2004-11-04 | The Trustees Of The University Of Pennsylvania | Compositions and methods for sirna inhibition of angiopoietin 1 and 2 and their receptor tie2 |
| US20070009899A1 (en) | 2003-10-02 | 2007-01-11 | Mounts William M | Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases |
| EP1735442A2 (en) | 2004-03-24 | 2006-12-27 | Oncotherapy Science, Inc. | Compositions and methods for treating pancreatic cancer |
| US20080221057A1 (en) | 2007-02-16 | 2008-09-11 | Wyeth | Secreted protein ccdc80 regulates adipocyte differentiation |
| US8257969B2 (en) | 2007-04-12 | 2012-09-04 | The Provost Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth | Genetic suppression and replacement |
| US20110129825A1 (en) | 2007-08-03 | 2011-06-02 | Melba Stinnett Ketchum | Compositions, methods and systems for the simultaneous determination of parentage, identity, sex, genotype and/or phenotype and breed determination in animals |
| US8450473B2 (en) * | 2009-04-30 | 2013-05-28 | The Research Foundation Of State University Of New York | Compositions and methods for therapy of macular degeneration |
| CN102061308B (zh) | 2010-10-29 | 2013-05-08 | 北京未名凯拓作物设计中心有限公司 | 一种用百草枯筛选转基因植物的方法 |
| CN102061305B (zh) * | 2010-12-15 | 2012-07-18 | 深圳市百恩维生物科技有限公司 | 一种多ShRNA高效协同沉默基因方法及载体 |
| US20130064815A1 (en) | 2011-09-12 | 2013-03-14 | The Trustees Of Princeton University | Inducing apoptosis in quiescent cells |
| EP2855684A1 (en) | 2012-05-25 | 2015-04-08 | Commissariat à l'Énergie Atomique et aux Énergies Alternatives | Vector for the selective silencing of a gene in astrocytes |
| WO2014138792A1 (en) | 2013-03-14 | 2014-09-18 | Commonwealth Scientific And Industrial Research Organisation | Double-stranded rna |
| EP3071592B1 (en) | 2013-11-20 | 2021-01-06 | Fondazione Telethon | Artificial dna-binding proteins and uses thereof |
| FI3628334T3 (fi) | 2014-03-21 | 2023-09-15 | Genzyme Corp | Geenihoito verkkokalvon pigmenttirappeuma |
| IL248102B (en) * | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| BR112017017178A2 (pt) * | 2015-02-26 | 2018-04-03 | Ionis Pharmaceuticals Inc | moduladores específicos de alelo de rodopsina de p23h |
| PL3265568T3 (pl) | 2015-03-06 | 2020-11-30 | Massachusetts Eye & Ear Infirmary | Terapie augmentacji genów w przypadku wrodzonego zwyrodnienia siatkówki spowodowanego mutacjami genu prpf31 |
| EP3289080B1 (en) | 2015-04-30 | 2021-08-25 | The Trustees of Columbia University in the City of New York | Gene therapy for autosomal dominant diseases |
| WO2017137493A1 (en) | 2016-02-09 | 2017-08-17 | Fondazione Telethon | Synthetic promoters and uses thereof |
| US20170348387A1 (en) | 2016-02-29 | 2017-12-07 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for nphp5 lca-ciliopathy |
| CN109154002B (zh) | 2016-03-01 | 2022-08-30 | 佛罗里达大学研究基金会有限公司 | 用于治疗显性视网膜色素变性的aav载体 |
-
2019
- 2019-06-03 CA CA3102095A patent/CA3102095A1/en active Pending
- 2019-06-03 WO PCT/US2019/035159 patent/WO2019232517A1/en not_active Ceased
- 2019-06-03 CN CN201980036506.9A patent/CN112204145B/zh active Active
- 2019-06-03 CN CN202410254122.7A patent/CN118480544A/zh active Pending
- 2019-06-03 US US17/059,815 patent/US12203074B2/en active Active
- 2019-06-03 EP EP19812143.6A patent/EP3802836A4/en active Pending
- 2019-06-03 JP JP2020567085A patent/JP7563694B2/ja active Active
- 2019-06-03 AU AU2019276645A patent/AU2019276645B2/en active Active
-
2020
- 2020-11-30 IL IL279091A patent/IL279091B1/en unknown
-
2024
- 2024-12-10 US US18/976,035 patent/US20250215437A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2021525531A5 (https=) | ||
| JP7651190B2 (ja) | 遺伝子操作rnaを用いた内因性adarによる標的化rna編集 | |
| JP7094236B2 (ja) | 変異aav、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法 | |
| JP2022500066A5 (https=) | ||
| JP2021502060A5 (https=) | ||
| IL279091B1 (en) | Compositions and methods for treating dominant retinitis pigmentosa | |
| JP7546295B2 (ja) | 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 | |
| JP2024119830A5 (https=) | ||
| JPWO2020069461A5 (https=) | ||
| CN113195719A (zh) | 用于增加蛋白质表达和/或治疗单倍剂量不足症的方法及组合物 | |
| JP2021533801A5 (https=) | ||
| JP2023532864A (ja) | 導入遺伝子発現系 | |
| JP2021534796A5 (https=) | ||
| JPWO2019232517A5 (https=) | ||
| US12415850B2 (en) | Compositions and methods for regulating production of an antibody like protein and ribonucleic acid | |
| EP4709862A2 (en) | Rnai targeting kif1a missense mutations for treatment of kif1a associated neurological disorder | |
| WO2024078345A1 (zh) | snRNA核酸分子及其应用 | |
| JPWO2022015715A5 (https=) | ||
| WO2023172535A2 (en) | shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME | |
| EP4473114A2 (en) | Shrna targeting ube3a-ats to restore paternal ube3a gene expression in angelman syndrome | |
| JPWO2020047476A5 (https=) | ||
| WO2025168955A1 (en) | Therapy for dravet syndrome by targeting scn1a antisense transcript | |
| WO2025065890A1 (zh) | 一种重组腺相关病毒颗粒、重组腺相关病毒载体系统及其应用 | |
| WO2024254243A2 (en) | Rnai targeting ppp2r5d missense mutations for treatment of jordan's syndrome | |
| JPWO2020077412A5 (https=) |