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 (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 |
---|---|---|---|---|
IL298138A (en) * | 2020-05-12 | 2023-01-01 | Univ 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 |
---|---|---|---|---|
WO2006069584A2 (en) * | 2004-12-29 | 2006-07-06 | Exiqon A/S | NOVEL OLIGONUCLEOTIDE COMPOSITIONS AND PROBE SEQUENCES USEFUL FOR DETECTION AND ANALYSIS OF microRNAs AND THEIR TARGET mRNAs |
ES2564823T3 (es) * | 2005-05-27 | 2016-03-29 | Ospedale San Raffaele S.R.L. | Vector génico que comprende miARN |
JP5102283B2 (ja) * | 2006-04-07 | 2012-12-19 | ゲオルク−アウグスト−ウニヴェルジテート・ゲッティンゲン・シュティフトゥング・エッフェントリヒェン・レッヒツ | タンパク質置換治療のための合成mecp2配列 |
US9415121B2 (en) * | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
AU2013243946A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of membrane proteins |
EP3452101A2 (en) * | 2016-05-04 | 2019-03-13 | CureVac AG | Rna encoding a therapeutic protein |
SG10201912976WA (en) * | 2017-02-28 | 2020-02-27 | Univ Pennsylvania | Adeno-associated virus (aav) clade f vector and uses therefor |
US20210101938A1 (en) * | 2017-03-24 | 2021-04-08 | The University Court Of The University Of Edinburgh | Mecp2 based therapy |
KR20200015701A (ko) * | 2017-06-06 | 2020-02-12 | 유니버시티 오브 매사추세츠 | 레트 증후군에서의 MeCP2의 안전한 발현을 위한 자가-조절 AAV 벡터 |
-
2020
- 2020-04-23 KR KR1020217037510A patent/KR20220003553A/ko unknown
- 2020-04-23 JP JP2021563234A patent/JP2022530095A/ja active Pending
- 2020-04-23 SG SG11202111279QA patent/SG11202111279QA/en unknown
- 2020-04-23 CN CN202080046692.7A patent/CN114026236A/zh active Pending
- 2020-04-23 US US17/605,827 patent/US20220202960A1/en active Pending
- 2020-04-23 WO PCT/US2020/029642 patent/WO2020219766A1/en unknown
- 2020-04-23 CA CA3133889A patent/CA3133889A1/en active Pending
- 2020-04-23 EP EP20795847.1A patent/EP3973060A4/en active Pending
- 2020-04-23 AU AU2020261051A patent/AU2020261051A1/en active Pending
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