KR20230134477A - 말초 미엘린 단백질-22의 발현 억제를 위한 생성물및 방법 - Google Patents

말초 미엘린 단백질-22의 발현 억제를 위한 생성물및 방법 Download PDF

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KR20230134477A
KR20230134477A KR1020237022382A KR20237022382A KR20230134477A KR 20230134477 A KR20230134477 A KR 20230134477A KR 1020237022382 A KR1020237022382 A KR 1020237022382A KR 20237022382 A KR20237022382 A KR 20237022382A KR 20230134477 A KR20230134477 A KR 20230134477A
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aav
rna
synthetic
artificial sequence
het
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스캇 퀀턴 하퍼
클레오파스 클레오파
마리나 스타브루
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더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈
더 사이프러스 파운데이션 포 머스큘러 디스트로피 리서치 디/비/에이 더 사이프러스 인스티튜트 오브 뉴롤로지 앤드 제네틱스
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KR1020237022382A 2020-12-01 2021-11-30 말초 미엘린 단백질-22의 발현 억제를 위한 생성물및 방법 Pending KR20230134477A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063120190P 2020-12-01 2020-12-01
US63/120,190 2020-12-01
PCT/US2021/061177 WO2022119826A1 (en) 2020-12-01 2021-11-30 Products and methods for inhibition of expression of peripheral myelin protein-22

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KR20230134477A true KR20230134477A (ko) 2023-09-21

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KR1020237022382A Pending KR20230134477A (ko) 2020-12-01 2021-11-30 말초 미엘린 단백질-22의 발현 억제를 위한 생성물및 방법

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US (1) US20240318172A1 (https=)
EP (1) EP4256069A4 (https=)
JP (1) JP2023553005A (https=)
KR (1) KR20230134477A (https=)
CN (1) CN116917492A (https=)
AU (1) AU2021390471A1 (https=)
CA (1) CA3203748A1 (https=)
IL (1) IL303308A (https=)
WO (1) WO2022119826A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240103029A (ko) * 2021-11-18 2024-07-03 노파르티스 아게 샤르코 마리 투스 질환 치료용 pmp22 표적화 화합물
WO2024229211A2 (en) 2023-05-02 2024-11-07 Research Institute At Nationwide Children's Hospital A modular system to convert therapeutic microrna expression cassettes from polymerase iii-based to polymerase ii-based promoters
WO2025150052A1 (en) * 2024-01-12 2025-07-17 Potentia Ltd. Targeted delivery of drug conjugates to schwann cells and treatment methods in schwann cell-related diseases
WO2025242147A1 (zh) * 2024-05-21 2025-11-27 北京安龙生物医药有限公司 靶向外周髓鞘蛋白-22基因的寡核苷酸及其用途

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012145597A2 (en) * 2011-04-21 2012-10-26 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of myotilin
WO2017156242A1 (en) * 2016-03-09 2017-09-14 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting pmp22 expression
JP7305534B2 (ja) * 2016-07-06 2023-07-10 バーテックス ファーマシューティカルズ インコーポレイテッド 疼痛関連障害を処置するための物質及び方法
KR101872105B1 (ko) * 2016-09-05 2018-06-27 사회복지법인 삼성생명공익재단 샤르코 마리 투스병 치료용 약학 조성물
AU2018224380A1 (en) * 2017-02-22 2019-08-29 Crispr Therapeutics Ag Compositions and methods for treatment of proprotein convertase subtilisin/kexin type 9 (PCSK9)-related disorders
JP2020536501A (ja) * 2017-09-28 2020-12-17 ツールゲン インコーポレイテッドToolgen Incorporated 遺伝子発現調節のための人為的なゲノム操作
WO2020047268A1 (en) * 2018-08-29 2020-03-05 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of mutant gars protein
EP3628735A1 (en) * 2018-09-25 2020-04-01 Centre National De La Recherche Scientifique Antisense rna targeting pmp22 for the treatment of charcot-marie-tooth 1a disease
JP7561129B2 (ja) * 2018-12-21 2024-10-03 アイオーニス ファーマシューティカルズ, インコーポレーテッド Pmp22の発現を低減するための化合物及び方法
KR20200124038A (ko) * 2019-04-23 2020-11-02 사회복지법인 삼성생명공익재단 마이크로rna-381을 유효성분으로 포함하는, pmp22 과발현에 의한 말초신경병증의 예방 또는 치료용 약학적 조성물

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JP2023553005A (ja) 2023-12-20
IL303308A (en) 2023-07-01
AU2021390471A9 (en) 2023-08-17
EP4256069A1 (en) 2023-10-11
AU2021390471A1 (en) 2023-06-29
CA3203748A1 (en) 2022-06-09
CN116917492A (zh) 2023-10-20
US20240318172A1 (en) 2024-09-26
EP4256069A4 (en) 2025-08-06
WO2022119826A1 (en) 2022-06-09

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