JP2023065516A5 - - Google Patents

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Publication number
JP2023065516A5
JP2023065516A5 JP2023026870A JP2023026870A JP2023065516A5 JP 2023065516 A5 JP2023065516 A5 JP 2023065516A5 JP 2023026870 A JP2023026870 A JP 2023026870A JP 2023026870 A JP2023026870 A JP 2023026870A JP 2023065516 A5 JP2023065516 A5 JP 2023065516A5
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JP
Japan
Prior art keywords
promoter
nucleic acid
acid molecule
tuberin
seq
Prior art date
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Application number
JP2023026870A
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English (en)
Japanese (ja)
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JP2023065516A (ja
JP7725514B2 (ja
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Priority claimed from PCT/US2018/033247 external-priority patent/WO2018213618A1/en
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Publication of JP2023065516A publication Critical patent/JP2023065516A/ja
Publication of JP2023065516A5 publication Critical patent/JP2023065516A5/ja
Application granted granted Critical
Publication of JP7725514B2 publication Critical patent/JP7725514B2/ja
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JP2023026870A 2017-05-17 2023-02-24 結節性硬化症の遺伝子治療 Active JP7725514B2 (ja)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762507358P 2017-05-17 2017-05-17
US62/507,358 2017-05-17
PCT/US2018/033247 WO2018213618A1 (en) 2017-05-17 2018-05-17 Gene therapy for tuberous sclerosis
JP2019558745A JP7235676B2 (ja) 2017-05-17 2018-05-17 結節性硬化症の遺伝子治療

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2019558745A Division JP7235676B2 (ja) 2017-05-17 2018-05-17 結節性硬化症の遺伝子治療

Publications (3)

Publication Number Publication Date
JP2023065516A JP2023065516A (ja) 2023-05-12
JP2023065516A5 true JP2023065516A5 (https=) 2023-10-23
JP7725514B2 JP7725514B2 (ja) 2025-08-19

Family

ID=64274693

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019558745A Active JP7235676B2 (ja) 2017-05-17 2018-05-17 結節性硬化症の遺伝子治療
JP2023026870A Active JP7725514B2 (ja) 2017-05-17 2023-02-24 結節性硬化症の遺伝子治療

Family Applications Before (1)

Application Number Title Priority Date Filing Date
JP2019558745A Active JP7235676B2 (ja) 2017-05-17 2018-05-17 結節性硬化症の遺伝子治療

Country Status (6)

Country Link
US (2) US11958887B2 (https=)
EP (2) EP3624856B1 (https=)
JP (2) JP7235676B2 (https=)
CA (1) CA3061656A1 (https=)
ES (1) ES2978297T3 (https=)
WO (1) WO2018213618A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7235676B2 (ja) * 2017-05-17 2023-03-08 ザ ジェネラル ホスピタル コーポレイション 結節性硬化症の遺伝子治療
CN117715928A (zh) * 2021-06-14 2024-03-15 桥生物基因疗法研究有限责任公司 用于结节性硬化的基因疗法
AU2024272186A1 (en) * 2023-05-17 2025-11-20 Ohio State Innovation Foundation Protein engineering micro-tuberin gene therapy candidates for tuberous sclerosis complex type 2

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US6884419B1 (en) * 1996-12-23 2005-04-26 Kyowa Hakko Kogyo, Co., Ltd. hG-CSF fusion polypeptide having c-mpl activity, DNA coding for same and methods of treating anemia using same
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
AU2003259803B2 (en) 2002-08-12 2007-08-02 The Regents Of The University Of Michigan Diagnosis and treatment of tuberous sclerosis
DK2311848T3 (da) 2002-12-23 2013-10-14 Vical Inc Kodon-optimerede polynukleotidbaserede vacciner mod human cytomegalovirus-infektion
CN1897977A (zh) * 2003-10-20 2007-01-17 Ns基因公司 帕金森氏病的体内基因治疗
US20090143288A1 (en) * 2007-03-13 2009-06-04 Roche Palo Alto Llc Peptide-complement conjugates
US7561973B1 (en) 2008-07-31 2009-07-14 Dna Twopointo, Inc. Methods for determining properties that affect an expression property value of polynucleotides in an expression system
US7561972B1 (en) 2008-06-06 2009-07-14 Dna Twopointo, Inc. Synthetic nucleic acids for expression of encoded proteins
US20120252877A1 (en) * 2009-08-14 2012-10-04 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex
US9249425B2 (en) 2011-05-16 2016-02-02 The Trustees Of The University Of Pennslyvania Proviral plasmids and production of recombinant adeno-associated virus
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
US11230589B2 (en) 2012-11-05 2022-01-25 Foundation Medicine, Inc. Fusion molecules and uses thereof
JP7235676B2 (ja) 2017-05-17 2023-03-08 ザ ジェネラル ホスピタル コーポレイション 結節性硬化症の遺伝子治療

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