CN115715327A - 用于治疗颗粒蛋白前体相关的神经退行性疾病或病症的腺伴随病毒(aav)系统 - Google Patents

用于治疗颗粒蛋白前体相关的神经退行性疾病或病症的腺伴随病毒(aav)系统 Download PDF

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Publication number
CN115715327A
CN115715327A CN202080089386.1A CN202080089386A CN115715327A CN 115715327 A CN115715327 A CN 115715327A CN 202080089386 A CN202080089386 A CN 202080089386A CN 115715327 A CN115715327 A CN 115715327A
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China
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aav
nucleic acid
seq
polynucleotide
promoter
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CN202080089386.1A
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English (en)
Chinese (zh)
Inventor
J·李
T·法里瓦
S·曼达帕迪
S·彭诺克
M·希尔曼
J·纽马克
A·蒂默斯
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Applied Genetic Technologies Corp
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Applied Genetic Technologies Corp
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Publication of CN115715327A publication Critical patent/CN115715327A/zh
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
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    • C12N2710/16011—Herpesviridae
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/00011—Details
    • 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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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202080089386.1A 2019-10-22 2020-10-22 用于治疗颗粒蛋白前体相关的神经退行性疾病或病症的腺伴随病毒(aav)系统 Pending CN115715327A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962924340P 2019-10-22 2019-10-22
US62/924340 2019-10-22
PCT/US2020/056860 WO2021081201A1 (en) 2019-10-22 2020-10-22 Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegeneative diseases or disorders

Publications (1)

Publication Number Publication Date
CN115715327A true CN115715327A (zh) 2023-02-24

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CN202080089386.1A Pending CN115715327A (zh) 2019-10-22 2020-10-22 用于治疗颗粒蛋白前体相关的神经退行性疾病或病症的腺伴随病毒(aav)系统

Country Status (10)

Country Link
US (2) US20210147872A1 (https=)
EP (1) EP4048799A4 (https=)
JP (1) JP2022553307A (https=)
KR (1) KR20230019402A (https=)
CN (1) CN115715327A (https=)
AU (1) AU2020371662A1 (https=)
CA (1) CA3158516A1 (https=)
IL (1) IL292382A (https=)
MX (1) MX2022004812A (https=)
WO (1) WO2021081201A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021533126A (ja) * 2018-07-31 2021-12-02 コーネル・ユニバーシティーCornell University 臓器機能を制御するための遺伝子治療方法
KR20230128001A (ko) * 2020-12-01 2023-09-01 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 엔젤만 증후군의 치료를 위한 조성물 및 이의 용도
US20240033332A1 (en) * 2020-12-17 2024-02-01 Kyoto University Agent for activating a neuron
WO2024035649A1 (en) * 2022-08-08 2024-02-15 Shape Therapeutics Inc. Compositions for progranulin expression and methods of use thereof
KR20250131772A (ko) * 2022-11-13 2025-09-03 알렉시온 파마 인터내셔널 오퍼레이션즈 리미티드 전두측두엽 치매에 대한 유전자 요법
WO2025251041A1 (en) * 2024-05-31 2025-12-04 Bluerock Therapeutics Lp METHODS AND COMPOSITIONS FOR TREATING PROGRANULIN DEFICIENCIES USING iPSC-DERIVED CELLS

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102006882A (zh) * 2008-01-16 2011-04-06 神经动力公司 利用颗粒体蛋白前体(pgrn)治疗神经变性疾病
WO2018136680A1 (en) * 2017-01-19 2018-07-26 New York University Progranulin and progranulin derivatives in treating impaired fracture healing
WO2019070894A1 (en) * 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMIAL DISORDERS

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP5879256B2 (ja) * 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
AU2013243948A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
WO2016125330A1 (ja) * 2015-02-02 2016-08-11 原　英彰 網膜再生促進薬
WO2017151884A1 (en) * 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
EP3692075A4 (en) * 2017-10-03 2022-02-09 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISEASES
US20220111005A1 (en) * 2019-02-01 2022-04-14 Avrobio, Inc. Compositions and methods for treating neurocognitive disorders
BR112021015817A2 (pt) * 2019-02-22 2021-10-13 The Trustees Of The University Of Pennsylvania Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102006882A (zh) * 2008-01-16 2011-04-06 神经动力公司 利用颗粒体蛋白前体(pgrn)治疗神经变性疾病
WO2018136680A1 (en) * 2017-01-19 2018-07-26 New York University Progranulin and progranulin derivatives in treating impaired fracture healing
WO2019070894A1 (en) * 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMIAL DISORDERS

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
HOLLER ET AL: "Intracellular Proteolysis of Progranulin Generates Stable, Lysosomal Granulins that Are Haploinsufficient in Patients with Frontotemporal Dementia Caused by GRN Mutations", ENEURO, vol. 4, no. 4, 9 August 2017 (2017-08-09), pages 1 - 22 *

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WO2021081201A1 (en) 2021-04-29
MX2022004812A (es) 2023-02-23
AU2020371662A1 (en) 2022-05-12
CA3158516A1 (en) 2021-04-29
EP4048799A4 (en) 2023-11-15
WO2021081201A8 (en) 2022-04-14
US20240318199A1 (en) 2024-09-26
US20210147872A1 (en) 2021-05-20
JP2022553307A (ja) 2022-12-22
IL292382A (en) 2022-06-01
EP4048799A1 (en) 2022-08-31
KR20230019402A (ko) 2023-02-08

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