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 (https=) | 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 (https=) |
| EP (1) | EP3897745A1 (https=) |
| JP (1) | JP2022514955A (https=) |
| KR (1) | KR102942447B1 (https=) |
| CN (1) | CN113518825A (https=) |
| AU (1) | AU2019417697A1 (https=) |
| BR (1) | BR112021012240A2 (https=) |
| WO (1) | WO2020139796A1 (https=) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN111093715A (zh) * | 2017-09-18 | 2020-05-01 | 儿童医院医疗中心 | 强绝缘子和其在基因递送中的用途 |
| EP4329822A1 (en) | 2021-04-26 | 2024-03-06 | CSL Behring L.L.C. | Lentiviral vectors useful for the treatment of disease |
| EP4402264A4 (en) * | 2021-09-16 | 2025-10-15 | Agency Science Tech & Res | 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 |
| EP4720100A1 (en) | 2023-05-25 | 2026-04-08 | CSL Behring LLC | Modified vectors |
| WO2025077575A1 (zh) * | 2023-10-08 | 2025-04-17 | 上海齐鲁制药研究中心有限公司 | 截短的绝缘子及其用于重组蛋白的瞬时表达的用途 |
| WO2025215591A1 (en) | 2024-04-12 | 2025-10-16 | Csl Behring L.L.C. | Modified vectors for xla gene therapy |
| CN120536433A (zh) * | 2024-05-20 | 2025-08-26 | 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 |
|---|---|---|---|---|
| US5958928A (en) | 1995-03-27 | 1999-09-28 | Chugai Seiyaku Kabushiki Kaisha | Pharmaceutical agents containing methotrexate derivative |
| 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 |
| EP2933332A1 (en) | 2004-06-28 | 2015-10-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| MX2007010008A (es) * | 2005-02-16 | 2008-01-18 | Lentigen Corp | Vectores lentivirales y su uso. |
| WO2007098089A2 (en) | 2006-02-17 | 2007-08-30 | Novacea, Inc. | Treatment of hyperproliferative diseases with methotrexate n-oxide and analogs |
| CN103849629B (zh) | 2006-06-21 | 2017-06-09 | 尤尼克尔Ip股份有限公司 | 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体 |
| BR122020021379B1 (pt) | 2008-10-24 | 2021-05-11 | Sarepta Therapeutics, Inc. | oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular |
| TR201816523T4 (tr) | 2009-11-12 | 2018-11-21 | Univ Western Australia | Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler. |
| 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 |
| DK2699247T3 (en) * | 2011-04-20 | 2018-06-14 | Univ California | PROCEDURE FOR COMBINED CONDITIONING AND CHEMOS SELECTION IN A SIMPLE CYCLE |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| HUE054260T2 (hu) | 2012-09-06 | 2021-08-30 | Univ Chicago | Antiszensz polinukleotidok exon-ugrás indukálására és eljárások disztrófiák kezelésére |
| US20140329762A1 (en) | 2013-03-15 | 2014-11-06 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| US20140363454A1 (en) | 2013-06-06 | 2014-12-11 | Igenica Biotherapeutics, Inc. | Antibody-Drug Conjugates, Compositions and Methods of Use |
| MX374532B (es) * | 2013-06-17 | 2025-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos. |
| JP7103750B2 (ja) * | 2013-12-12 | 2022-07-20 | ザ・ブロード・インスティテュート・インコーポレイテッド | ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用 |
| 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 |
| PL3134432T3 (pl) | 2014-04-25 | 2020-10-19 | Bluebird Bio, Inc. | Promotor mnd chimeryczne receptory antygenowe |
| PL3294893T3 (pl) | 2015-05-13 | 2024-04-29 | Csl Behring Gene Therapy, Inc. | Bioprodukcja wektorów lentiwirusowych |
| HK1247963A1 (zh) | 2015-05-18 | 2018-10-05 | Csl贝林基因治疗股份有限公司 | 用於hiv的治疗的基因疗法及其用途 |
| CN108495640B (zh) * | 2016-02-19 | 2022-11-04 | 加利福尼亚大学董事会 | 短发夹rna(shrna734)及其用于阳性选择和消除遗传修饰的细胞的用途 |
| 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 |
| IL263744B2 (en) | 2016-06-17 | 2023-11-01 | Magenta Therapeutics Inc | Compositions and methods for the depletion of cd117 plus cells |
| CN111164211B (zh) * | 2017-07-18 | 2024-08-02 | 杰特贝林基因治疗股份有限公司 | 用于治疗β-血红蛋白病的组合物和方法 |
| 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 EP EP19842992.0A patent/EP3897745A1/en active Pending
- 2019-12-23 JP JP2021536371A patent/JP2022514955A/ja active Pending
- 2019-12-23 KR KR1020217023216A patent/KR102942447B1/ko active Active
- 2019-12-23 BR BR112021012240A patent/BR112021012240A2/pt unknown
- 2019-12-23 WO PCT/US2019/068233 patent/WO2020139796A1/en not_active Ceased
- 2019-12-23 AU AU2019417697A patent/AU2019417697A1/en active Pending
- 2019-12-23 CN CN201980092125.2A patent/CN113518825A/zh active Pending
-
2021
- 2021-06-21 US US17/353,586 patent/US20210316013A1/en active Pending
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