JP2022530095A5 - - Google Patents

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Publication number
JP2022530095A5
JP2022530095A5 JP2021563234A JP2021563234A JP2022530095A5 JP 2022530095 A5 JP2022530095 A5 JP 2022530095A5 JP 2021563234 A JP2021563234 A JP 2021563234A JP 2021563234 A JP2021563234 A JP 2021563234A JP 2022530095 A5 JP2022530095 A5 JP 2022530095A5
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JP
Japan
Application number
JP2021563234A
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Japanese (ja)
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JP2022530095A (ja
JPWO2020219766A5 (https=
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Publication date
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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
Publication of JP2022530095A5 publication Critical patent/JP2022530095A5/ja
Pending legal-status Critical Current

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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 (3)

Publication Number Publication Date
JP2022530095A JP2022530095A (ja) 2022-06-27
JPWO2020219766A5 JPWO2020219766A5 (https=) 2023-04-28
JP2022530095A5 true JP2022530095A5 (https=) 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 (https=)
EP (1) EP3973060A4 (https=)
JP (1) JP2022530095A (https=)
KR (1) KR20220003553A (https=)
CN (1) CN114026236A (https=)
AU (1) AU2020261051A1 (https=)
CA (1) CA3133889A1 (https=)
SG (1) SG11202111279QA (https=)
WO (1) WO2020219766A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230304034A1 (en) * 2020-05-12 2023-09-28 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
AU2022369293A1 (en) * 2021-10-18 2024-05-16 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of cdkl5 deficiency disorder (cdd)
US20250312486A1 (en) * 2022-04-18 2025-10-09 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
EP4715058A1 (en) * 2023-05-15 2026-03-25 Genecombio Ltd. Gene therapy for rett syndrome

Family Cites Families (10)

* 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
PL2002003T3 (pl) * 2005-05-27 2016-06-30 Ospedale San Raffaele Srl Wektor genetyczny zawierający mi-RNA
PL2010563T3 (pl) * 2006-04-07 2011-11-30 Georg August Univ Goettingen Stiftung Oeffentlichen Rechts Sekwencja syntetycznego MECP2 do białkowej terapii substytucyjnej
US9415121B2 (en) * 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
AU2013243948A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
EP3452101A2 (en) * 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
CA3052487A1 (en) * 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Influenza vaccines based on aav vectors
JP7455579B2 (ja) * 2017-02-28 2024-03-26 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 新規アデノ随伴ウイルス(aav)クレードfベクター及びその用途
JP2020513831A (ja) * 2017-03-24 2020-05-21 ザ ユニバーシティ コート オブ ザ ユニバーシティ オブ エジンバラ MeCP2発現カセット
US11680275B2 (en) * 2017-06-06 2023-06-20 University Of Massachusetts Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome

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