KR20230019063A - C9orf72 연관 질환의 치료를 위한 삼중 기능 아데노-연관 바이러스 (aav) 벡터 - Google Patents

C9orf72 연관 질환의 치료를 위한 삼중 기능 아데노-연관 바이러스 (aav) 벡터 Download PDF

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KR20230019063A
KR20230019063A KR1020227017065A KR20227017065A KR20230019063A KR 20230019063 A KR20230019063 A KR 20230019063A KR 1020227017065 A KR1020227017065 A KR 1020227017065A KR 20227017065 A KR20227017065 A KR 20227017065A KR 20230019063 A KR20230019063 A KR 20230019063A
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c9orf72
nucleic acid
vector
sequence
aav
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페이신 주
시지아 왕
스티븐 페녹
마크 셔먼
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어플라이드 제네틱스 테크놀로지스 코퍼레이션
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    • C07—ORGANIC CHEMISTRY
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

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KR1020227017065A 2019-10-22 2020-10-22 C9orf72 연관 질환의 치료를 위한 삼중 기능 아데노-연관 바이러스 (aav) 벡터 Ceased KR20230019063A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962924351P 2019-10-22 2019-10-22
US62/924,351 2019-10-22
PCT/US2020/056905 WO2021081236A1 (en) 2019-10-22 2020-10-22 Triple function adeno-associated virus (aav) vectors for the treatment of c9orf72 associated diseases

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KR20230019063A true KR20230019063A (ko) 2023-02-07

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KR1020227017065A Ceased KR20230019063A (ko) 2019-10-22 2020-10-22 C9orf72 연관 질환의 치료를 위한 삼중 기능 아데노-연관 바이러스 (aav) 벡터

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US (2) US20210147873A1 (https=)
EP (1) EP4048794A4 (https=)
JP (1) JP2023501897A (https=)
KR (1) KR20230019063A (https=)
CN (1) CN116134134A (https=)
AU (1) AU2020370291A1 (https=)
CA (1) CA3158518A1 (https=)
IL (1) IL292384A (https=)
MX (1) MX2022004771A (https=)
WO (1) WO2021081236A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3947700A4 (en) 2019-04-01 2023-01-04 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
BR112023006305A2 (pt) 2020-10-09 2023-05-09 Tenaya Therapeutics Inc Métodos e composições de terapia gênica com placofilina-2
EP4347822A2 (en) * 2021-06-04 2024-04-10 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
EP4427049A4 (en) * 2021-11-01 2025-08-20 Univ Florida POLY-GA PROTEINS IN ALZHEIMER'S DISEASE
WO2023133574A1 (en) * 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful for treatment of c9orf72-mediated disorders
CN120035669A (zh) * 2022-09-28 2025-05-23 阿塔兰塔治疗公司 用于治疗神经系统病症的组合物和方法
CN121057823A (zh) * 2023-04-10 2025-12-02 耶鲁大学 体内修饰神经元以治疗和/或预防肌萎缩性侧索硬化(als)的方法
WO2025085713A1 (en) * 2023-10-20 2025-04-24 Spark Therapeutics, Inc. Inhibitory nucleic acid targeting alpha synuclein expression

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WO2012057363A1 (ja) * 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
US9096671B2 (en) * 2011-06-29 2015-08-04 Consejo Superior De Investigaciones Cientificas (Csic) LRP1 as key receptor for the transfer of sterified cholesterol from very-low-density lipoproteins (VLDL) to ischaemic cardiac muscle
US9896729B2 (en) * 2011-08-31 2018-02-20 The University Of Manchester Method for diagnosing a neurodegenerative disease
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
BR112020008033A2 (pt) * 2017-10-23 2020-10-27 Prevail Therapeutics, Inc. terapias gênicas para doença neurodegenerativa

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IL292384A (en) 2022-06-01
EP4048794A4 (en) 2024-04-17
WO2021081236A1 (en) 2021-04-29
MX2022004771A (es) 2022-10-07
JP2023501897A (ja) 2023-01-20
AU2020370291A1 (en) 2022-05-12
US20210147873A1 (en) 2021-05-20
EP4048794A1 (en) 2022-08-31
US20240067984A1 (en) 2024-02-29
CN116134134A (zh) 2023-05-16
CA3158518A1 (en) 2021-04-29

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