CN114173821A - 用于恢复stmn2水平的方法和组合物 - Google Patents

用于恢复stmn2水平的方法和组合物 Download PDF

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Publication number
CN114173821A
CN114173821A CN202080020187.5A CN202080020187A CN114173821A CN 114173821 A CN114173821 A CN 114173821A CN 202080020187 A CN202080020187 A CN 202080020187A CN 114173821 A CN114173821 A CN 114173821A
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stmn2
agent
tdp
disease
rna
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Chinese (zh)
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约瑟夫·罗伯特·克里姆
弗兰塞斯科·利莫内
伊鲁内·圭拉桑朱昂
路易斯·威廉
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Harvard University
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Harvard University
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CN202080020187.5A 2019-01-14 2020-01-14 用于恢复stmn2水平的方法和组合物 Pending CN114173821A (zh)

Applications Claiming Priority (3)

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US201962792276P 2019-01-14 2019-01-14
US62/792,276 2019-01-14
PCT/US2020/013581 WO2020150290A2 (en) 2019-01-14 2020-01-14 Methods and compositions for restoring stmn2 levels

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US (1) US12496327B2 (https=)
EP (1) EP3911411A4 (https=)
JP (1) JP2022517117A (https=)
CN (1) CN114173821A (https=)
CA (1) CA3126918A1 (https=)
WO (1) WO2020150290A2 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3103429A1 (en) 2018-06-14 2019-12-19 Don W. Cleveland Compounds and methods for increasing stmn2 expression
EP4162051A4 (en) * 2020-06-03 2025-07-09 Quralis Corp TREATMENT OF NEUROLOGICAL DISEASES USING GENE TRANSCRIPT MODULATORS
WO2022018155A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Lna oligonucleotides for splice modulation of stmn2
US20250270548A1 (en) * 2020-12-07 2025-08-28 Perron Institute for Neurological and Translational Science Limited Compositions and methods for treating tardbp associated diseases
CA3215808A1 (en) * 2021-04-22 2022-10-27 Roger Chung Modified polypeptides and uses thereof
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
EP4724426A1 (en) * 2023-06-12 2026-04-15 The General Hospital Corporation Statin derivatives and methods of using the same
WO2025076126A1 (en) * 2023-10-02 2025-04-10 Emory University Agents that bind nucleic acids encoding elavl3 cryptic exons, pharmaceutical compositions, and uses in managing neurological diseases

Citations (4)

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US20090253622A1 (en) * 2006-02-03 2009-10-08 Nederlandse Organisatie Voor Toegepast - Natuurwetenschappelijk Onderzoek Tno Use of tlr3 agonists for the treatment of neurodegenerative disorders
WO2013169793A2 (en) * 2012-05-09 2013-11-14 Ipierian, Inc. Methods and compositions for tdp-43 proteinopathies
US20160291037A1 (en) * 2015-03-31 2016-10-06 Arizona Board Of Regents On Behalf Of Arizona State University Methods for detecting traumatic brain injury
WO2018154337A1 (en) * 2017-02-27 2018-08-30 St George's Hospital Medical School Biomarkers

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CA2653974A1 (en) 2006-05-30 2008-02-14 Mayo Foundation For Medical Education And Research Detecting and treating dementia
CA3103429A1 (en) 2018-06-14 2019-12-19 Don W. Cleveland Compounds and methods for increasing stmn2 expression
JP7795914B2 (ja) 2019-06-03 2026-01-08 クラリス コーポレーション 神経学的疾患を処置するためのオリゴヌクレオチドおよびその使用方法
EP4127172A4 (en) 2020-03-25 2025-06-04 President and Fellows of Harvard College Methods and compositions for restoring STMN2 levels

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090253622A1 (en) * 2006-02-03 2009-10-08 Nederlandse Organisatie Voor Toegepast - Natuurwetenschappelijk Onderzoek Tno Use of tlr3 agonists for the treatment of neurodegenerative disorders
WO2013169793A2 (en) * 2012-05-09 2013-11-14 Ipierian, Inc. Methods and compositions for tdp-43 proteinopathies
US20160291037A1 (en) * 2015-03-31 2016-10-06 Arizona Board Of Regents On Behalf Of Arizona State University Methods for detecting traumatic brain injury
WO2018154337A1 (en) * 2017-02-27 2018-08-30 St George's Hospital Medical School Biomarkers

Non-Patent Citations (2)

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Title
GUO-LIN CHEN等: "Modulation of nuclear REST by alternative splicing: a potential therapeutic target for Huntington’s disease", 《J. CELL. MOL. MED.》 *
KLIM, J: "TDP-43 - The Key to Unlocking ALS Through Pluripotent Stem Cell Disease Modeling", 《STEM CELL AND REGENERATIVE MEDICINE CENTER, UNIVERSITY OF WISCONSIN - MADISON》 *

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US12496327B2 (en) 2025-12-16
WO2020150290A2 (en) 2020-07-23
US20220133848A1 (en) 2022-05-05
WO2020150290A3 (en) 2020-09-10
EP3911411A4 (en) 2023-04-12
JP2022517117A (ja) 2022-03-04
EP3911411A2 (en) 2021-11-24
CA3126918A1 (en) 2020-07-23

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