CN112004925B - 具有降低的肝向性的AAV9和AAVrh74的杂合重组腺相关病毒血清型 - Google Patents
具有降低的肝向性的AAV9和AAVrh74的杂合重组腺相关病毒血清型 Download PDFInfo
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- CN112004925B CN112004925B CN201980023352.XA CN201980023352A CN112004925B CN 112004925 B CN112004925 B CN 112004925B CN 201980023352 A CN201980023352 A CN 201980023352A CN 112004925 B CN112004925 B CN 112004925B
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP18305399.0 | 2018-04-05 | ||
| EP18305399 | 2018-04-05 | ||
| PCT/EP2019/058560 WO2019193119A1 (en) | 2018-04-05 | 2019-04-04 | HYBRID RECOMBINANT ADENO-ASSOCIATED VIRUS SEROTYPE BETWEEN AAV9 AND AAVrh74 WITH REDUCED LIVER TROPISM |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN112004925A CN112004925A (zh) | 2020-11-27 |
| CN112004925B true CN112004925B (zh) | 2024-09-27 |
Family
ID=62063468
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980023352.XA Active CN112004925B (zh) | 2018-04-05 | 2019-04-04 | 具有降低的肝向性的AAV9和AAVrh74的杂合重组腺相关病毒血清型 |
| CN201980095204.9A Pending CN113795574A (zh) | 2018-04-05 | 2019-10-04 | 具有降低的肝向性和增加的肌肉转导的AAV9和AAVrh74之间的肽修饰的杂合重组腺相关病毒血清型 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980095204.9A Pending CN113795574A (zh) | 2018-04-05 | 2019-10-04 | 具有降低的肝向性和增加的肌肉转导的AAV9和AAVrh74之间的肽修饰的杂合重组腺相关病毒血清型 |
Country Status (13)
| Country | Link |
|---|---|
| US (3) | US12037362B2 (https=) |
| EP (2) | EP3775173A1 (https=) |
| JP (2) | JP7374119B2 (https=) |
| KR (2) | KR20210015770A (https=) |
| CN (2) | CN112004925B (https=) |
| AU (2) | AU2019249890B2 (https=) |
| BR (2) | BR112020020223A2 (https=) |
| CA (2) | CA3093347A1 (https=) |
| IL (2) | IL277740A (https=) |
| MA (1) | MA51353B1 (https=) |
| TN (1) | TN2020000187A1 (https=) |
| WO (2) | WO2019193119A1 (https=) |
| ZA (1) | ZA202005422B (https=) |
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| US20210363193A1 (en) * | 2018-04-27 | 2021-11-25 | Universität Heidelberg | Modified aav capsid polypeptides for treatment of muscular diseases |
| US12466864B2 (en) | 2019-03-25 | 2025-11-11 | Genethon | Production of large-sized quasidystrophins using overlapping AAV vectors |
| CN114127088B (zh) * | 2019-04-23 | 2024-05-14 | 国家医疗保健研究所 | 变体衣壳蛋白和腺相关病毒aav变体及其药物组合物 |
| JP7651562B2 (ja) | 2019-09-19 | 2025-03-26 | ジェネトン | Fkrpの心毒性を軽減する遺伝子治療発現系 |
| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| US12188039B2 (en) | 2019-11-08 | 2025-01-07 | The Board Of Regents Of The University Of Texas System | Recombinant adeno-associated viral vector for gene delivery |
| EP4073257A1 (en) * | 2019-12-09 | 2022-10-19 | UCL Business Ltd | Gene therapy composition and treatment for myh7-linked cardiomyopathy |
| EP4077684A1 (en) | 2019-12-18 | 2022-10-26 | Genethon | Production of recombinant viral vectors from plant hairy roots |
| WO2021229255A1 (en) | 2020-05-14 | 2021-11-18 | Genethon | Tools and method for preventing a a¥ neutralization by antibodies |
| EP3913060A1 (en) | 2020-05-22 | 2021-11-24 | Genethon | Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy |
| CN116322790A (zh) | 2020-06-09 | 2023-06-23 | 吉尼松公司 | 用于治疗扩张型心肌病的nrf2激活剂 |
| US20230220391A1 (en) | 2020-06-09 | 2023-07-13 | Genethon | CILP-1 Inhibitors for Use in the Treatment of Dilated Cardiomyopathies |
| WO2021250076A1 (en) | 2020-06-09 | 2021-12-16 | Genethon | Treatment of genetic dilated cardiomyopathies |
| US20230256117A1 (en) | 2020-06-19 | 2023-08-17 | Genethon | Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg |
| CA3187635A1 (en) | 2020-07-03 | 2022-01-06 | Genethon | Method for engineering novel hybrid aav capsids through hypervariable regions swapping |
| EP4179096A1 (en) | 2020-07-10 | 2023-05-17 | Genethon | A novel muscle-specific promoter |
| EP4421179A3 (en) | 2020-09-10 | 2024-12-18 | Genethon | Peptide-modified aav capsid |
| US20230272433A1 (en) | 2020-09-29 | 2023-08-31 | Genethon | Enhancing Utrophin Expression in Cell by Inducing Mutations Within Utrophin Regulatory Elements and Therapeutic Use Thereof |
| KR20230117177A (ko) * | 2020-12-03 | 2023-08-07 | 유니버시티 오브 매사추세츠 | 진행성 골화성 섬유이형성증을 위한 신규 유전자 치료제의개발 |
| EP4273236A4 (en) * | 2020-12-29 | 2025-01-08 | Jiangsu Genscript Probio Biotech Co., Ltd. | HEK293T CELL STRAIN WITH HIGH DISPERSIBILITY AND SCREENING METHODS THEREFOR |
| US20240115734A1 (en) * | 2021-02-09 | 2024-04-11 | Affinia Therapeutics Inc. | Recombinant aavs with improved tropism and specificity |
| US20240165271A1 (en) | 2021-03-26 | 2024-05-23 | The Board Of Regents Of The University Of Texas System | Nucleotide editing to reframe dmd transcripts by base editing and prime editing |
| WO2022226301A1 (en) * | 2021-04-23 | 2022-10-27 | The University Of North Carolina At Chapel Hill | Chimeric heart tropic aav capsids |
| EP4381077A1 (en) | 2021-08-04 | 2024-06-12 | Genethon | Hybrid promoters for gene expression in muscles and in the cns |
| CN115232818B (zh) * | 2021-09-18 | 2025-10-21 | 南京启真基因工程有限公司 | 构建dok7基因突变的先天性肌无力模型猪核移植供体细胞的基因编辑系统及其应用 |
| US20230211014A1 (en) * | 2021-12-29 | 2023-07-06 | Genetic Cures for Kids Inc. | AAV Gene Therapy for Spastic Paraplegia |
| CN114276419B (zh) * | 2021-12-30 | 2023-11-17 | 上海勉亦生物科技有限公司 | 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用 |
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| CN116925192B (zh) * | 2022-04-12 | 2025-04-29 | 合肥星眸生物科技有限公司 | 一种融合型腺相关病毒及其应用 |
| WO2023237731A1 (en) | 2022-06-09 | 2023-12-14 | Genethon | N-terminal truncated gde for the treatment of glycogen storage disease iii |
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| CN116041443B (zh) * | 2022-12-30 | 2023-09-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2024173802A2 (en) * | 2023-02-17 | 2024-08-22 | Biogen Ma Inc. | Rgd-containing peptides for delivering payloads |
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| CN105324392A (zh) * | 2013-04-20 | 2016-02-10 | 全国儿童医院研究所 | 外显子2靶向U7snRNA多核苷酸构建体的重组腺相关病毒递送 |
| CN105518145A (zh) * | 2013-07-12 | 2016-04-20 | 费城儿童医院 | Aav载体和用于抗aav(腺相关病毒)中和抗体的检测 |
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| TN2020000187A1 (en) | 2022-04-04 |
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