JPWO2020219766A5 - - Google Patents
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- 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 (fr) | 2019-04-24 | 2020-04-23 | Compositions utiles dans le traitement du syndrome de rett |
Publications (2)
Publication Number | Publication Date |
---|---|
JP2022530095A JP2022530095A (ja) | 2022-06-27 |
JPWO2020219766A5 true JPWO2020219766A5 (fr) | 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 (fr) |
EP (1) | EP3973060A4 (fr) |
JP (1) | JP2022530095A (fr) |
KR (1) | KR20220003553A (fr) |
CN (1) | CN114026236A (fr) |
AU (1) | AU2020261051A1 (fr) |
CA (1) | CA3133889A1 (fr) |
SG (1) | SG11202111279QA (fr) |
WO (1) | WO2020219766A1 (fr) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JP2023526310A (ja) * | 2020-05-12 | 2023-06-21 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 導入遺伝子発現のdrg特異的低減のための組成物 |
WO2022094078A1 (fr) * | 2020-10-28 | 2022-05-05 | The Trustees Of The University Of Pennsylvania | Compositions utiles dans le traitement du syndrome de rett |
WO2023205657A2 (fr) * | 2022-04-18 | 2023-10-26 | City Of Hope | Compositions pour restaurer la fonction du gène mecp2 et leurs méthodes d'utilisation |
WO2024081551A1 (fr) * | 2022-10-10 | 2024-04-18 | Passage Bio, Inc. | Procédé de purification de particules d'aav entièrement recombinés |
Family Cites Families (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2006069584A2 (fr) * | 2004-12-29 | 2006-07-06 | Exiqon A/S | Nouvelles compositions d'oligonucleotides et sequences de sondes utiles pour la detection et l'analyse de microarn et de leurs marn cibles |
KR20130114758A (ko) * | 2005-05-27 | 2013-10-17 | 오스페달레 산 라파엘 에스.알.엘. | Mi-rna를 포함하는 유전자 벡터 |
CA2647125C (fr) * | 2006-04-07 | 2015-02-03 | Georg-August-Universitaet Goettingen Stiftung Oeffentlichen Rechts | Sequence mecp2 synthetique utilisee dans une therapie de substitution de proteines |
US9415121B2 (en) * | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
DE18200782T1 (de) * | 2012-04-02 | 2021-10-21 | Modernatx, Inc. | Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen |
EP3452101A2 (fr) * | 2016-05-04 | 2019-03-13 | CureVac AG | Arn codant pour une protéine thérapeutique |
HRP20240257T1 (hr) * | 2017-02-28 | 2024-05-24 | The Trustees Of The University Of Pennsylvania | Adeno-povezani virusni (aav) clade f vektor i njegova uporaba |
WO2018172794A1 (fr) * | 2017-03-24 | 2018-09-27 | The University Court Of The University Of Edinburgh | Thérapie à base de mecp2 |
SG11201911737PA (en) * | 2017-06-06 | 2020-01-30 | Univ Massachusetts | Self-regulating aav vectors for safe expression of mecp2 in rett syndrome |
-
2020
- 2020-04-23 CN CN202080046692.7A patent/CN114026236A/zh active Pending
- 2020-04-23 KR KR1020217037510A patent/KR20220003553A/ko unknown
- 2020-04-23 JP JP2021563234A patent/JP2022530095A/ja active Pending
- 2020-04-23 US US17/605,827 patent/US20220202960A1/en active Pending
- 2020-04-23 AU AU2020261051A patent/AU2020261051A1/en active Pending
- 2020-04-23 EP EP20795847.1A patent/EP3973060A4/fr active Pending
- 2020-04-23 WO PCT/US2020/029642 patent/WO2020219766A1/fr unknown
- 2020-04-23 CA CA3133889A patent/CA3133889A1/fr active Pending
- 2020-04-23 SG SG11202111279QA patent/SG11202111279QA/en unknown
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