JPWO2020219766A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2020219766A5 JPWO2020219766A5 JP2021563234A JP2021563234A JPWO2020219766A5 JP WO2020219766 A5 JPWO2020219766 A5 JP WO2020219766A5 JP 2021563234 A JP2021563234 A JP 2021563234A JP 2021563234 A JP2021563234 A JP 2021563234A JP WO2020219766 A5 JPWO2020219766 A5 JP WO2020219766A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- raav
- sequence
- sequences
- mirna target
- 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 |
|---|---|---|---|
| US201962837947P | 2019-04-24 | 2019-04-24 | |
| US62/837,947 | 2019-04-24 | ||
| PCT/US2020/029642 WO2020219766A1 (en) | 2019-04-24 | 2020-04-23 | Compositions useful in treatment of rett syndrome |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022530095A JP2022530095A (ja) | 2022-06-27 |
| JPWO2020219766A5 true JPWO2020219766A5 (ru) | 2023-04-28 |
Family
ID=72941315
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021563234A Pending JP2022530095A (ja) | 2019-04-24 | 2020-04-23 | レット症候群の治療に有用な組成物 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20220202960A1 (ru) |
| EP (1) | EP3973060A4 (ru) |
| JP (1) | JP2022530095A (ru) |
| KR (1) | KR20220003553A (ru) |
| CN (1) | CN114026236A (ru) |
| AU (1) | AU2020261051A1 (ru) |
| CA (1) | CA3133889A1 (ru) |
| SG (1) | SG11202111279QA (ru) |
| WO (1) | WO2020219766A1 (ru) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2021270447A1 (en) * | 2020-05-12 | 2023-01-05 | The Trustees Of The University Of Pennsylvania | Compositions for DRG-specific reduction of transgene expression |
| WO2022094078A1 (en) * | 2020-10-28 | 2022-05-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
| WO2023205657A2 (en) * | 2022-04-18 | 2023-10-26 | City Of Hope | Compositions for restoring mecp2 gene function and methods of use thereof |
| WO2024081551A1 (en) * | 2022-10-10 | 2024-04-18 | Passage Bio, Inc. | Method of purifying full recombinant aav particles |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1959012A3 (en) * | 2004-12-29 | 2009-12-30 | Exiqon A/S | Novel oligonucleotide compositions and probe sequences useful for detection and analysis of microRNAs and their target mRNAs |
| US10000757B2 (en) * | 2005-05-27 | 2018-06-19 | Ospedale San Raffaele S.R.L. | Gene vector |
| DK2010563T3 (da) * | 2006-04-07 | 2011-05-23 | Univ Goettingen Georg August | Syntetisk MeCP2 sekvens til proteinsubstitutionsterapi |
| US9415121B2 (en) * | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| EP2833923A4 (en) * | 2012-04-02 | 2016-02-24 | Moderna Therapeutics Inc | MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF PROTEINS |
| WO2017191274A2 (en) * | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
| ES2971872T3 (es) * | 2017-02-28 | 2024-06-10 | Univ Pennsylvania | Vector de clado F de virus adenoasociado (AAV) y usos para el mismo |
| CA3057425A1 (en) * | 2017-03-24 | 2018-09-27 | The University Court Of The University Of Edinburgh | Mecp2 based therapy |
| CA3066623A1 (en) * | 2017-06-06 | 2019-12-13 | University Of Massachusetts | Self-regulating aav vectors for safe expression of mecp2 in rett syndrome |
-
2020
- 2020-04-23 EP EP20795847.1A patent/EP3973060A4/en active Pending
- 2020-04-23 US US17/605,827 patent/US20220202960A1/en active Pending
- 2020-04-23 JP JP2021563234A patent/JP2022530095A/ja active Pending
- 2020-04-23 CN CN202080046692.7A patent/CN114026236A/zh active Pending
- 2020-04-23 KR KR1020217037510A patent/KR20220003553A/ko unknown
- 2020-04-23 WO PCT/US2020/029642 patent/WO2020219766A1/en unknown
- 2020-04-23 AU AU2020261051A patent/AU2020261051A1/en active Pending
- 2020-04-23 CA CA3133889A patent/CA3133889A1/en active Pending
- 2020-04-23 SG SG11202111279QA patent/SG11202111279QA/en unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| Powell et al. | Viral expression cassette elements to enhance transgene target specificity and expression in gene therapy | |
| Daya et al. | Gene therapy using adeno-associated virus vectors | |
| US11344608B2 (en) | Factor IX gene therapy | |
| JP2020537544A5 (ru) | ||
| JP2024009857A5 (ru) | ||
| JPWO2020132455A5 (ru) | ||
| JP2018108113A5 (ru) | ||
| JP2018522529A5 (ru) | ||
| JP2020522269A5 (ru) | ||
| JP6824169B2 (ja) | 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用 | |
| WO2023283962A1 (en) | Modified aav capsid for gene therapy and methods thereof | |
| JP2023544947A (ja) | 昆虫細胞内で重複オープンリーディングフレームを含む遺伝子を発現するための発現カセット及びその使用 | |
| CN118434877A (zh) | 用于核纤层蛋白a相关缺陷的基因疗法 | |
| Gan et al. | Gene delivery with viral vectors for cerebrovascular diseases | |
| JPWO2020223231A5 (ru) | ||
| JPWO2020223232A5 (ru) | ||
| JPWO2020219766A5 (ru) | ||
| JPWO2021067448A5 (ru) | ||
| US20220213494A1 (en) | Dual leucine zipper kinase inhibitors for gene therapy | |
| US20220010332A1 (en) | Intron fragments | |
| JPWO2020227166A5 (ru) | ||
| JPWO2021231579A5 (ru) | ||
| JPWO2022036220A5 (ru) | ||
| JPWO2022076803A5 (ru) | ||
| WO2024220722A1 (en) | Compositions and methods for tissue- specific expression of gene therapies |