JP2025512709A5 - - Google Patents

Info

Publication number
JP2025512709A5
JP2025512709A5 JP2024553306A JP2024553306A JP2025512709A5 JP 2025512709 A5 JP2025512709 A5 JP 2025512709A5 JP 2024553306 A JP2024553306 A JP 2024553306A JP 2024553306 A JP2024553306 A JP 2024553306A JP 2025512709 A5 JP2025512709 A5 JP 2025512709A5
Authority
JP
Japan
Prior art keywords
polynucleotide
expression vector
seq
pharmaceutical composition
shrna
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
JP2024553306A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025512709A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2023/014679 external-priority patent/WO2023172534A2/en
Publication of JP2025512709A publication Critical patent/JP2025512709A/ja
Publication of JP2025512709A5 publication Critical patent/JP2025512709A5/ja
Pending legal-status Critical Current

Links

JP2024553306A 2022-03-07 2023-03-07 アンジェルマン症候群における父方由来ＵＢＥ３Ａ遺伝子発現を回復させるためのＵＢＥ３Ａ－ＡＴＳを標的とするｓｈＲＮＡ Pending JP2025512709A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263317154P 2022-03-07 2022-03-07
US63/317,154 2022-03-07
PCT/US2023/014679 WO2023172534A2 (en) 2022-03-07 2023-03-07 shRNA TARGETING UBE3A-ATS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME

Publications (2)

Publication Number Publication Date
JP2025512709A JP2025512709A (ja) 2025-04-22
JP2025512709A5 true JP2025512709A5 (cg-RX-API-DMAC7.html) 2026-03-16

Family

ID=87935840

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024553306A Pending JP2025512709A (ja) 2022-03-07 2023-03-07 アンジェルマン症候群における父方由来ＵＢＥ３Ａ遺伝子発現を回復させるためのＵＢＥ３Ａ－ＡＴＳを標的とするｓｈＲＮＡ

Country Status (10)

Country Link
US (1) US20230332157A1 (cg-RX-API-DMAC7.html)
EP (1) EP4473114A2 (cg-RX-API-DMAC7.html)
JP (1) JP2025512709A (cg-RX-API-DMAC7.html)
KR (1) KR20240154664A (cg-RX-API-DMAC7.html)
CN (1) CN119053703A (cg-RX-API-DMAC7.html)
AU (1) AU2023232014A1 (cg-RX-API-DMAC7.html)
CA (1) CA3254296A1 (cg-RX-API-DMAC7.html)
IL (1) IL315364A (cg-RX-API-DMAC7.html)
MX (1) MX2024010821A (cg-RX-API-DMAC7.html)
WO (1) WO2023172534A2 (cg-RX-API-DMAC7.html)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025101991A1 (en) * 2023-11-09 2025-05-15 Ionis Pharmaceuticals, Inc. Methods for modulating ube3a-ats

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8058516B2 (en) * 2000-07-19 2011-11-15 Monsanto Technology Llc Rice metallothionein promoters
WO2017083722A1 (en) * 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy
EP4143581A1 (en) * 2020-04-27 2023-03-08 Biogen MA Inc. Methods of detecting ube3a protein

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