CN119053703A - 靶向UBE3A-ATS以恢复快乐木偶综合征中父系UBE3A基因表达的shRNA - Google Patents

靶向UBE3A-ATS以恢复快乐木偶综合征中父系UBE3A基因表达的shRNA Download PDF

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Publication number
CN119053703A
CN119053703A CN202380026099.XA CN202380026099A CN119053703A CN 119053703 A CN119053703 A CN 119053703A CN 202380026099 A CN202380026099 A CN 202380026099A CN 119053703 A CN119053703 A CN 119053703A
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China
Prior art keywords
seq
polynucleotide
shrna
expression vector
ube3a
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Pending
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CN202380026099.XA
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Chinese (zh)
Inventor
斯托米·张伯伦
诺艾儿·杰曼
彼得·佩里诺
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University of Connecticut
Ovid Therapeutics Inc
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University of Connecticut
Ovid Therapeutics Inc
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Publication of CN119053703A publication Critical patent/CN119053703A/zh
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CN202380026099.XA 2022-03-07 2023-03-07 靶向UBE3A-ATS以恢复快乐木偶综合征中父系UBE3A基因表达的shRNA Pending CN119053703A (zh)

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 (1)

Publication Number Publication Date
CN119053703A true CN119053703A (zh) 2024-11-29

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CN202380026099.XA Pending CN119053703A (zh) 2022-03-07 2023-03-07 靶向UBE3A-ATS以恢复快乐木偶综合征中父系UBE3A基因表达的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

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JP2025512709A (ja) 2025-04-22
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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