KR20240154664A - 안젤만 증후군에서 부계 UBE3A 유전자 발현을 회복하기 위한 UBE3A-ATS를 표적으로 하는 shRNA - Google Patents

안젤만 증후군에서 부계 UBE3A 유전자 발현을 회복하기 위한 UBE3A-ATS를 표적으로 하는 shRNA Download PDF

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KR20240154664A
KR20240154664A KR1020247033383A KR20247033383A KR20240154664A KR 20240154664 A KR20240154664 A KR 20240154664A KR 1020247033383 A KR1020247033383 A KR 1020247033383A KR 20247033383 A KR20247033383 A KR 20247033383A KR 20240154664 A KR20240154664 A KR 20240154664A
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seq
polynucleotide
shrna
article
expression vector
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Korean (ko)
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스토미 챔벌린
노엘 저먼
피터 페리노
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유니버시티 오브 코네티컷
오비드 테라퓨틱스 인크.
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Publication of KR20240154664A publication Critical patent/KR20240154664A/ko
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KR1020247033383A 2022-03-07 2023-03-07 안젤만 증후군에서 부계 UBE3A 유전자 발현을 회복하기 위한 UBE3A-ATS를 표적으로 하는 shRNA Pending KR20240154664A (ko)

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
KR20240154664A true KR20240154664A (ko) 2024-10-25

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KR1020247033383A Pending KR20240154664A (ko) 2022-03-07 2023-03-07 안젤만 증후군에서 부계 UBE3A 유전자 발현을 회복하기 위한 UBE3A-ATS를 표적으로 하는 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

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CN119053703A (zh) 2024-11-29
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
IL315364A (en) 2024-11-01
US20230332157A1 (en) 2023-10-19
WO2023172534A3 (en) 2023-10-19

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