JP2022514955A5 - - Google Patents
Info
- Publication number
- JP2022514955A5 JP2022514955A5 JP2021536371A JP2021536371A JP2022514955A5 JP 2022514955 A5 JP2022514955 A5 JP 2022514955A5 JP 2021536371 A JP2021536371 A JP 2021536371A JP 2021536371 A JP2021536371 A JP 2021536371A JP 2022514955 A5 JP2022514955 A5 JP 2022514955A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- sequence
- acid sequence
- expression vector
- vector according
- 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
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862784508P | 2018-12-23 | 2018-12-23 | |
| US62/784,508 | 2018-12-23 | ||
| PCT/US2019/068233 WO2020139796A1 (en) | 2018-12-23 | 2019-12-23 | Haematopoietic stem cell-gene therapy for wiskott-aldrich syndrome |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022514955A JP2022514955A (ja) | 2022-02-16 |
| JP2022514955A5 true JP2022514955A5 (enExample) | 2023-01-05 |
Family
ID=69326699
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021536371A Pending JP2022514955A (ja) | 2018-12-23 | 2019-12-23 | ウィスコット・アルドリッチ症候群の造血幹細胞遺伝子治療 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20210316013A1 (enExample) |
| EP (1) | EP3897745A1 (enExample) |
| JP (1) | JP2022514955A (enExample) |
| KR (1) | KR102942447B1 (enExample) |
| CN (1) | CN113518825A (enExample) |
| AU (1) | AU2019417697A1 (enExample) |
| BR (1) | BR112021012240A2 (enExample) |
| WO (1) | WO2020139796A1 (enExample) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11970707B2 (en) * | 2017-09-18 | 2024-04-30 | Children's Hospital Medical Center | Strong insulator and uses thereof in gene delivery |
| WO2022232191A1 (en) | 2021-04-26 | 2022-11-03 | Csl Behring L.L.C. | Lentiviral vectors useful for the treatment of disease |
| WO2023043375A2 (en) * | 2021-09-16 | 2023-03-23 | Agency For Science, Technology And Research | Modulation of tjp1 expression to treat liver diseases |
| CN115838765A (zh) * | 2021-09-18 | 2023-03-24 | 济南赛尔生物科技股份有限公司 | 一种治疗异染性脑白质营养不良的慢病毒载体 |
| CN114990163A (zh) * | 2022-03-31 | 2022-09-02 | 中海峡(福建)细胞生物科技有限公司 | 用于干细胞基因修饰的慢病毒载体及其构建方法和应用 |
| WO2024171034A2 (en) * | 2023-02-13 | 2024-08-22 | Csl Behring Llc | Optimised polynucleotides |
| CN121399150A (zh) | 2023-05-25 | 2026-01-23 | 美国杰特贝林生物制品有限公司 | 经修饰载体 |
| EP4790141A1 (en) * | 2023-10-08 | 2026-08-12 | Shanghai Qilu Pharmaceutical Research and Development Centre Ltd. | Truncated insulator and use thereof in transient expression of recombinant protein |
| WO2025215591A1 (en) | 2024-04-12 | 2025-10-16 | Csl Behring L.L.C. | Modified vectors for xla gene therapy |
| WO2025241977A1 (zh) * | 2024-05-20 | 2025-11-27 | 上海本导基因技术有限公司 | 一种能够调节基因表达的调控元件及应用 |
| WO2026003694A1 (en) * | 2024-06-24 | 2026-01-02 | Csl Behring Llc | Methods of enveloped virus transduction |
Family Cites Families (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR100400509B1 (ko) | 1995-03-27 | 2004-04-03 | 쥬가이 세이야쿠 가부시키가이샤 | 메토트렉세이트유도체를함유하는약제 |
| US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
| EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
| WO2005080581A2 (en) | 2004-02-17 | 2005-09-01 | University Of Florida Research Foundation, Inc. | Insulated herpesvirus-derived gene expression cassettes for sustained and regulatable gene expression |
| ES2627917T3 (es) | 2004-06-28 | 2017-08-01 | The University Of Western Australia | Oligonucleótidos no codificantes para la inducción de la omisión exónica y métodos de uso de los mismos |
| EP2573185A3 (en) * | 2005-02-16 | 2013-06-05 | Lentigen Corporation | Lentiviral vectors and their use |
| WO2007098089A2 (en) | 2006-02-17 | 2007-08-30 | Novacea, Inc. | Treatment of hyperproliferative diseases with methotrexate n-oxide and analogs |
| WO2007148971A2 (en) | 2006-06-21 | 2007-12-27 | Amsterdam Molecular Therapeutics B.V. | Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells |
| EP3133160B1 (en) | 2008-10-24 | 2018-12-12 | Sarepta Therapeutics, Inc. | Exon skipping compositions for dmd |
| HUE040445T2 (hu) | 2009-11-12 | 2019-03-28 | Univ Western Australia | Antiszensz molekulák és eljárások betegségek kezelésére |
| US20110294114A1 (en) | 2009-12-04 | 2011-12-01 | Cincinnati Children's Hospital Medical Center | Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells |
| RU2611396C2 (ru) * | 2011-04-20 | 2017-02-21 | Де Реджентс Оф Де Юниверсити Оф Калифорния | Способ совместного кондиционирования и хемоселекции за один цикл |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| WO2014039916A1 (en) | 2012-09-06 | 2014-03-13 | The University Of Chicago | Antisense polynucleotides to induce exon skipping and methods of treating dystrophies |
| HK1220154A1 (zh) | 2013-03-15 | 2017-04-28 | Sarepta Therapeutics, Inc. | 改进的用於治疗肌营养不良的组合物 |
| US20140363454A1 (en) | 2013-06-06 | 2014-12-11 | Igenica Biotherapeutics, Inc. | Antibody-Drug Conjugates, Compositions and Methods of Use |
| KR20250012194A (ko) * | 2013-06-17 | 2025-01-23 | 더 브로드 인스티튜트, 인코퍼레이티드 | 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용 |
| CN111206032B (zh) * | 2013-12-12 | 2024-07-19 | 布罗德研究所有限公司 | 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用 |
| CN104805120A (zh) * | 2014-01-27 | 2015-07-29 | 苟德明 | 一种shRNA-Ago2共表达慢病毒RNAi载体、重组质粒及其构建方法 |
| WO2015164759A2 (en) | 2014-04-25 | 2015-10-29 | Bluebird Bio, Inc. | Mnd promoter chimeric antigen receptors |
| DK3294893T5 (da) | 2015-05-13 | 2024-10-07 | Csl Behring Gene Therapy Inc | Bioproduktion af lentivirale vektorer |
| CA2986469A1 (en) | 2015-05-18 | 2016-11-24 | Calimmune, Inc. | Gene therapeutic for the treatment of hiv and uses thereof |
| WO2017143266A1 (en) * | 2016-02-19 | 2017-08-24 | The Regents Of The University Of California | Short hairpin rna (shrna734) and use of same to positively select and eliminate genetically modified cells |
| CN109641051A (zh) | 2016-06-17 | 2019-04-16 | 美真达治疗公司 | 用于耗尽细胞的组合物和方法 |
| US10111966B2 (en) | 2016-06-17 | 2018-10-30 | Magenta Therapeutics, Inc. | Methods for the depletion of CD117+ cells |
| CA3070242A1 (en) * | 2017-07-18 | 2019-01-24 | Calimmune, Inc. | Compositions and methods for treating beta-hemoglobinopathies |
| JP7470119B2 (ja) * | 2018-08-24 | 2024-04-17 | シーエスエル ベーリング ジーン セラピー インコーポレイテッド | 無血清培地におけるベクター産生 |
-
2019
- 2019-12-23 CN CN201980092125.2A patent/CN113518825A/zh active Pending
- 2019-12-23 AU AU2019417697A patent/AU2019417697A1/en active Pending
- 2019-12-23 JP JP2021536371A patent/JP2022514955A/ja active Pending
- 2019-12-23 EP EP19842992.0A patent/EP3897745A1/en active Pending
- 2019-12-23 KR KR1020217023216A patent/KR102942447B1/ko active Active
- 2019-12-23 WO PCT/US2019/068233 patent/WO2020139796A1/en not_active Ceased
- 2019-12-23 BR BR112021012240A patent/BR112021012240A2/pt unknown
-
2021
- 2021-06-21 US US17/353,586 patent/US20210316013A1/en active Pending
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