KR20240155345A - 안젤만 증후군에서 부계 UBE3A 유전자 발현을 회복하기 위한 SNORD115 위치를 표적으로 하는 shRNA - Google Patents

안젤만 증후군에서 부계 UBE3A 유전자 발현을 회복하기 위한 SNORD115 위치를 표적으로 하는 shRNA Download PDF

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KR20240155345A
KR20240155345A KR1020247033390A KR20247033390A KR20240155345A KR 20240155345 A KR20240155345 A KR 20240155345A KR 1020247033390 A KR1020247033390 A KR 1020247033390A KR 20247033390 A KR20247033390 A KR 20247033390A KR 20240155345 A KR20240155345 A KR 20240155345A
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seq
shrna
polynucleotide
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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KR1020247033390A 2022-03-07 2023-03-07 안젤만 증후군에서 부계 UBE3A 유전자 발현을 회복하기 위한 SNORD115 위치를 표적으로 하는 shRNA Pending KR20240155345A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263317155P 2022-03-07 2022-03-07
US63/317,155 2022-03-07
PCT/US2023/014680 WO2023172535A2 (en) 2022-03-07 2023-03-07 shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME

Publications (1)

Publication Number Publication Date
KR20240155345A true KR20240155345A (ko) 2024-10-28

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Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247033390A Pending KR20240155345A (ko) 2022-03-07 2023-03-07 안젤만 증후군에서 부계 UBE3A 유전자 발현을 회복하기 위한 SNORD115 위치를 표적으로 하는 shRNA

Country Status (10)

Country Link
US (1) US20230332150A1 (https=)
EP (1) EP4473116A2 (https=)
JP (1) JP2025509262A (https=)
KR (1) KR20240155345A (https=)
CN (1) CN119384504A (https=)
AU (1) AU2023231550A1 (https=)
CA (1) CA3259398A1 (https=)
IL (1) IL315359A (https=)
MX (1) MX2024010820A (https=)
WO (1) WO2023172535A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025101533A1 (en) * 2023-11-09 2025-05-15 Encoded Therapeutics, Inc. Methods and compositions for increasing expression of ube3a

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150329857A1 (en) * 2011-12-28 2015-11-19 Universite Pierre Et Marie Curie (Paris 6) Rna interference to activate stem cells
PT3717646T (pt) * 2017-12-01 2025-07-28 Texas A & M Univ Sys Tratamento antisense da síndrome de angelman
CA3113648A1 (en) * 2018-09-21 2020-03-26 University Of Connecticut Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome
CA3133294A1 (en) * 2019-03-15 2020-09-24 Mir Scientific, Llc Methods for predicting prostate cancer and uses thereof

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MX2024010820A (es) 2024-12-06
IL315359A (en) 2024-11-01
CN119384504A (zh) 2025-01-28
JP2025509262A (ja) 2025-04-11
WO2023172535A2 (en) 2023-09-14
CA3259398A1 (en) 2023-09-14
AU2023231550A1 (en) 2024-09-26
EP4473116A2 (en) 2024-12-11
US20230332150A1 (en) 2023-10-19
WO2023172535A3 (en) 2023-11-30

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