CN114555069A - 寡核苷酸和用于治疗神经学疾病的方法 - Google Patents

寡核苷酸和用于治疗神经学疾病的方法 Download PDF

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CN114555069A
CN114555069A CN202080054507.9A CN202080054507A CN114555069A CN 114555069 A CN114555069 A CN 114555069A CN 202080054507 A CN202080054507 A CN 202080054507A CN 114555069 A CN114555069 A CN 114555069A
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oligonucleotide
linkages
seq
stmn2
qsn
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S.辛克利
D.布朗
S.阿格拉瓦尔
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Quelis Co
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CN202080054507.9A 2019-06-03 2020-06-03 寡核苷酸和用于治疗神经学疾病的方法 Pending CN114555069A (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962856264P 2019-06-03 2019-06-03
US62/856,264 2019-06-03
US201962914252P 2019-10-11 2019-10-11
US62/914,252 2019-10-11
US201962949817P 2019-12-18 2019-12-18
US62/949,817 2019-12-18
PCT/US2020/035811 WO2020247419A2 (en) 2019-06-03 2020-06-03 Oligonucleotides and methods of use for treating neurological diseases

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US (1) US20220333105A1 (https=)
EP (1) EP3976010A4 (https=)
JP (2) JP7795914B2 (https=)
KR (1) KR20220033472A (https=)
CN (1) CN114555069A (https=)
AU (1) AU2020288555A1 (https=)
BR (1) BR112021024463A2 (https=)
CA (1) CA3142526A1 (https=)
IL (1) IL288574A (https=)
MX (1) MX2021014868A (https=)
PH (1) PH12021553024A1 (https=)
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WO2022018155A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Lna oligonucleotides for splice modulation of stmn2
WO2022018187A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Oligonucleotides targeting rna binding protein sites
AU2022255175A1 (en) * 2021-04-06 2023-11-23 Trace Neuroscience, Inc. Compositions and methods for treating tdp-43 proteinopathy
KR20240052763A (ko) * 2021-08-11 2024-04-23 아버 바이오테크놀로지스, 인크. 스타스민 2 (stmn2)를 표적화하는 rna 가이드를 포함하는 유전자 편집 시스템 및 이의 용도
AU2022402929A1 (en) * 2021-12-03 2024-06-13 Quralis Corporation Splice switcher antisense oligonucleotides with modified backbone chemistries
GB202208387D0 (en) * 2022-06-08 2022-07-20 Ucl Business Ltd Modified U7 snRNA construct
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WO2024124203A2 (en) * 2022-12-09 2024-06-13 Auttx, Llc Targeted treatment of spliceopathy-induced neurological disorders
WO2024173645A1 (en) * 2023-02-15 2024-08-22 Arbor Biotechnologies, Inc. Gene editing method for inhibiting aberrant splicing in stathmin 2 (stmn2) transcript
WO2025184347A1 (en) * 2024-02-27 2025-09-04 Emory University Network analysis of the cerebrospinal fluid proteome of sporadic and familial forms of amyotrophic lateral sclerosis and methods of treatment of the same

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EP3976010A2 (en) 2022-04-06
US20220333105A1 (en) 2022-10-20
WO2020247419A2 (en) 2020-12-10
KR20220033472A (ko) 2022-03-16
JP2022536085A (ja) 2022-08-12
BR112021024463A2 (pt) 2022-03-08
MX2021014868A (es) 2022-03-25
EP3976010A4 (en) 2023-08-02
PH12021553024A1 (en) 2023-08-23
CA3142526A1 (en) 2020-12-10
WO2020247419A3 (en) 2021-01-14
JP2026002847A (ja) 2026-01-08
AU2020288555A1 (en) 2022-01-20
IL288574A (en) 2022-02-01

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