JP2022553307A5 - - Google Patents

Info

Publication number
JP2022553307A5
JP2022553307A5 JP2022523408A JP2022523408A JP2022553307A5 JP 2022553307 A5 JP2022553307 A5 JP 2022553307A5 JP 2022523408 A JP2022523408 A JP 2022523408A JP 2022523408 A JP2022523408 A JP 2022523408A JP 2022553307 A5 JP2022553307 A5 JP 2022553307A5
Authority
JP
Japan
Prior art keywords
aav
seq
functionally linked
nucleic acid
polynucleotide
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2022523408A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022553307A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/056860 external-priority patent/WO2021081201A1/en
Publication of JP2022553307A publication Critical patent/JP2022553307A/ja
Publication of JP2022553307A5 publication Critical patent/JP2022553307A5/ja
Pending legal-status Critical Current

Links

JP2022523408A 2019-10-22 2020-10-22 プログラニュリン関連神経変性疾患または障害の治療のためのアデノ随伴ウイルス(aav)システム Pending JP2022553307A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962924340P 2019-10-22 2019-10-22
US62/924,340 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 (2)

Publication Number Publication Date
JP2022553307A JP2022553307A (ja) 2022-12-22
JP2022553307A5 true JP2022553307A5 (enExample) 2023-10-30

Family

ID=75620215

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022523408A Pending JP2022553307A (ja) 2019-10-22 2020-10-22 プログラニュリン関連神経変性疾患または障害の治療のためのアデノ随伴ウイルス(aav)システム

Country Status (10)

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

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20210052450A (ko) * 2018-07-31 2021-05-10 코넬 유니버시티 기관 기능을 제어하기 위한 유전자 요법 방법
IL303239A (en) * 2020-12-01 2023-07-01 Univ Pennsylvania Compositions and their uses for the treatment of Engelmann syndrome
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
AU2023378899A1 (en) * 2022-11-13 2025-06-26 Alexion Pharma International Operations Limited Gene therapy for frontotemporal dementia
WO2025251041A1 (en) * 2024-05-31 2025-12-04 Bluerock Therapeutics Lp METHODS AND COMPOSITIONS FOR TREATING PROGRANULIN DEFICIENCIES USING iPSC-DERIVED CELLS

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009089635A1 (en) * 2008-01-16 2009-07-23 Neurodyn, Inc. Treating neurodegenerative diseases with progranulin (pgrn)
HRP20250897T1 (hr) * 2009-05-02 2025-09-26 Genzyme Corporation Genska terapija za neurodegenerativne poremećaje
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics 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
US20190328906A1 (en) * 2016-03-02 2019-10-31 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
US20190388508A1 (en) * 2017-01-19 2019-12-26 New York University Progranulin and progranulin derivatives in treating impaired fracture healing
AU2018346104B2 (en) * 2017-10-03 2023-07-27 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
BR112020006661A2 (pt) * 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
WO2020160458A1 (en) * 2019-02-01 2020-08-06 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

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