JP2022520232A5 - - Google Patents

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Publication number
JP2022520232A5
JP2022520232A5 JP2021546891A JP2021546891A JP2022520232A5 JP 2022520232 A5 JP2022520232 A5 JP 2022520232A5 JP 2021546891 A JP2021546891 A JP 2021546891A JP 2021546891 A JP2021546891 A JP 2021546891A JP 2022520232 A5 JP2022520232 A5 JP 2022520232A5
Authority
JP
Japan
Application number
JP2021546891A
Other languages
Japanese (ja)
Other versions
JP2022520232A (ja
JPWO2020167996A5 (https=
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/017987 external-priority patent/WO2020167996A1/en
Publication of JP2022520232A publication Critical patent/JP2022520232A/ja
Publication of JP2022520232A5 publication Critical patent/JP2022520232A5/ja
Publication of JPWO2020167996A5 publication Critical patent/JPWO2020167996A5/ja
Priority to JP2025007633A priority Critical patent/JP2025063228A/ja
Pending legal-status Critical Current

Links

JP2021546891A 2019-02-12 2020-02-12 ダノン病の治療のための遺伝子療法ベクター Pending JP2022520232A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025007633A JP2025063228A (ja) 2019-02-12 2025-01-20 ダノン病の治療のための遺伝子療法ベクター

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962804521P 2019-02-12 2019-02-12
US62/804,521 2019-02-12
US201962934928P 2019-11-13 2019-11-13
US62/934,928 2019-11-13
PCT/US2020/017987 WO2020167996A1 (en) 2019-02-12 2020-02-12 Gene therapy vectors for treatment of danon disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025007633A Division JP2025063228A (ja) 2019-02-12 2025-01-20 ダノン病の治療のための遺伝子療法ベクター

Publications (3)

Publication Number Publication Date
JP2022520232A JP2022520232A (ja) 2022-03-29
JP2022520232A5 true JP2022520232A5 (https=) 2023-02-21
JPWO2020167996A5 JPWO2020167996A5 (https=) 2023-02-21

Family

ID=72043957

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021546891A Pending JP2022520232A (ja) 2019-02-12 2020-02-12 ダノン病の治療のための遺伝子療法ベクター
JP2025007633A Pending JP2025063228A (ja) 2019-02-12 2025-01-20 ダノン病の治療のための遺伝子療法ベクター

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025007633A Pending JP2025063228A (ja) 2019-02-12 2025-01-20 ダノン病の治療のための遺伝子療法ベクター

Country Status (12)

Country Link
US (1) US20220143215A1 (https=)
EP (1) EP3924371A4 (https=)
JP (2) JP2022520232A (https=)
KR (1) KR20210125999A (https=)
CN (2) CN113508130A (https=)
AU (3) AU2020221842A1 (https=)
BR (1) BR112021015751A2 (https=)
CA (1) CA3128514A1 (https=)
IL (1) IL285238A (https=)
MX (1) MX2021009696A (https=)
SG (1) SG11202107744SA (https=)
WO (1) WO2020167996A1 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3021572A1 (en) 2016-04-20 2017-10-26 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas Compositions and methods for enhanced gene expression of pklr
EP3697452A4 (en) 2017-10-16 2021-11-24 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. LENTIVIRAL VECTORS FOR ADMINISTRATION OF PKLR TO TREAT PYRUVATE KINASE DEFECTS
BR112020020841A2 (pt) 2018-04-11 2021-01-19 Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus Composições e métodos para transplante de célulastronco
CA3096293A1 (en) 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration
WO2020014523A1 (en) * 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
JP2023538519A (ja) 2020-08-07 2023-09-08 スペースクラフト セブン リミテッド ライアビリティ カンパニー Aavベクターを使用したプラコフィリン-2(pkp2)遺伝子治療
CA3201247A1 (en) * 2020-12-07 2022-06-16 Gaurav Shah Treatment of danon disease
CN114874990A (zh) * 2021-02-05 2022-08-09 中国科学院苏州纳米技术与纳米仿生研究所 一种功能化外泌体及其制备方法和应用
JP7564368B2 (ja) 2021-09-23 2024-10-08 エルジー エナジー ソリューション リミテッド 高耐熱性コネクター及びこれを含むバッテリーモジュール、バッテリーパック、自動車
CN114457112A (zh) * 2022-02-07 2022-05-10 苏州市立医院 一种特异性神经靶向mr分子探针及其制备方法和应用
CN115975051B (zh) * 2022-11-17 2025-09-12 东南大学 靶向外泌体及制备方法、应用、药物和药物递送系统

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7985553B2 (en) * 2001-10-29 2011-07-26 Nathaniel Heintz Method for isolating cell type-specific mRNAs
US9217155B2 (en) * 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US9434928B2 (en) * 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
AU2013221212B2 (en) * 2012-02-17 2018-08-09 The Children's Hospital Of Philadelphia AAV vector compositions and methods for gene transfer to cells, organs and tissues
DK3405215T3 (da) * 2016-01-19 2022-09-19 Univ California Fremgangsmåder til behandling af danon-sygdom og andre autofagilidelser
WO2017136536A1 (en) * 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
US11713468B2 (en) * 2017-08-02 2023-08-01 Dna Twopointo Inc. DNA vectors and elements for sustained gene expression in eukaryotic cells
WO2020014523A1 (en) * 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
EP3833745A1 (en) * 2018-08-10 2021-06-16 REGENXBIO Inc. Scalable method for recombinant aav production

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