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/fr
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 (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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

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