CN111433361B - 天使综合征反义治疗 - Google Patents

天使综合征反义治疗 Download PDF

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CN111433361B
CN111433361B CN201880077892.1A CN201880077892A CN111433361B CN 111433361 B CN111433361 B CN 111433361B CN 201880077892 A CN201880077892 A CN 201880077892A CN 111433361 B CN111433361 B CN 111433361B
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artificial sequence
synthetic construct
rna
dna
aso
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CN111433361A (zh
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S·V·迪多
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Texas A&M University System
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CN201880077892.1A 2017-12-01 2018-11-30 天使综合征反义治疗 Active CN111433361B (zh)

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US201762593431P 2017-12-01 2017-12-01
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US201862676034P 2018-05-24 2018-05-24
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PCT/US2018/063416 WO2019109001A1 (en) 2017-12-01 2018-11-30 Angelman syndrome antisense treatment

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EP3758714A4 (en) * 2018-02-27 2021-12-01 The University of North Carolina at Chapel Hill METHODS AND COMPOSITIONS FOR TREATMENT OF ANGELMAN'S SYNDROME
TW202536179A (zh) 2019-03-29 2025-09-16 美商Ionis製藥公司 用於調節ube3a-ats之化合物及方法
TW202216996A (zh) * 2020-06-29 2022-05-01 美商Ionis製藥公司 調節ｐｌｐ１之化合物及方法
CA3197664A1 (en) * 2020-10-06 2022-04-14 University Of Maryland, Baltimore Rapid diagnostic electrochemical biosensing targeted with antisense oligonucleotides
CN119384504A (zh) * 2022-03-07 2025-01-28 康涅狄格大学 靶向SNORD115位置以恢复快乐木偶综合征中父系UBE3A基因表达的shRNA
KR20250107262A (ko) 2023-01-31 2025-07-11 후지필름 가부시키가이샤 경화성 조성물, 경화물, 광학 재료, 회절 광학 소자 및 화합물
WO2025100980A1 (ko) * 2023-11-08 2025-05-15 한국과학기술원 Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도
WO2025217466A1 (en) * 2024-04-11 2025-10-16 Ultragenyx Pharmaceutical Inc. Dosing regimens for antisense treatment of angelman syndrome
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