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
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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
Application number
JP2021563234A
Other languages
English (en)
Japanese (ja)
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JP2022530095A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/029642 external-priority patent/WO2020219766A1/en
Publication of JP2022530095A publication Critical patent/JP2022530095A/ja
Publication of JPWO2020219766A5 publication Critical patent/JPWO2020219766A5/ja
Pending legal-status Critical Current

Links

JP2021563234A 2019-04-24 2020-04-23 レット症候群の治療に有用な組成物 Pending JP2022530095A (ja)

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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

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