JP2025512709A - 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 - Google Patents

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 Download PDF

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JP2025512709A
JP2025512709A JP2024553306A JP2024553306A JP2025512709A JP 2025512709 A JP2025512709 A JP 2025512709A JP 2024553306 A JP2024553306 A JP 2024553306A JP 2024553306 A JP2024553306 A JP 2024553306A JP 2025512709 A JP2025512709 A JP 2025512709A
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seq
polynucleotide
ube3a
shrna
expression vector
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JP2024553306A
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JP2025512709A5 (https=
JPWO2023172534A5 (https=
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チェンバレン，ストーミー
ジャーメイン，ノエル
ペリノ，ピーター
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Ovid Therapeutics Inc
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Ovid Therapeutics Inc
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Publication of JP2025512709A5 publication Critical patent/JP2025512709A5/ja
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
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 (3)

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JP2025512709A true JP2025512709A (ja) 2025-04-22
JP2025512709A5 JP2025512709A5 (https=) 2026-03-16
JPWO2023172534A5 JPWO2023172534A5 (https=) 2026-03-16

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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 (https=)
EP (1) EP4473114A2 (https=)
JP (1) JP2025512709A (https=)
KR (1) KR20240154664A (https=)
CN (1) CN119053703A (https=)
AU (1) AU2023232014A1 (https=)
CA (1) CA3254296A1 (https=)
IL (1) IL315364A (https=)
MX (1) MX2024010821A (https=)
WO (1) WO2023172534A2 (https=)

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
US12467929B2 (en) * 2020-04-27 2025-11-11 Biogen Ma Inc. Methods of detecting UBE3A protein

Also Published As

Publication number Publication date
CN119053703A (zh) 2024-11-29
MX2024010821A (es) 2024-12-06
AU2023232014A1 (en) 2024-09-26
EP4473114A2 (en) 2024-12-11
CA3254296A1 (en) 2023-09-14
WO2023172534A2 (en) 2023-09-14
KR20240154664A (ko) 2024-10-25
IL315364A (en) 2024-11-01
US20230332157A1 (en) 2023-10-19
WO2023172534A3 (en) 2023-10-19

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