DK3368678T3 - Genetisk konstrukt - Google Patents

Genetisk konstrukt Download PDF

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Publication number
DK3368678T3
DK3368678T3 DK16788748.8T DK16788748T DK3368678T3 DK 3368678 T3 DK3368678 T3 DK 3368678T3 DK 16788748 T DK16788748 T DK 16788748T DK 3368678 T3 DK3368678 T3 DK 3368678T3
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genetic construct
genetic
construct
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DK16788748.8T
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Peter Widdowson
Keith Martin
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Quethera Ltd
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C07K14/475—Growth factors; Growth regulators
    • C07K14/48—Nerve growth factor [NGF]
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    • A61K38/18—Growth factors; Growth regulators
    • A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • A—HUMAN NECESSITIES
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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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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DK16788748.8T 2015-10-26 2016-10-25 Genetisk konstrukt DK3368678T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB1518911.1A GB2547179A (en) 2015-10-26 2015-10-26 Genetic construct
PCT/GB2016/053319 WO2017072498A1 (en) 2015-10-26 2016-10-25 Genetic construct

Publications (1)

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DK3368678T3 true DK3368678T3 (da) 2021-09-13

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US (2) US10342880B2 (da)
EP (2) EP3368678B1 (da)
JP (2) JP7073264B2 (da)
KR (1) KR20180081503A (da)
CN (1) CN108350467B (da)
AU (1) AU2016344508B2 (da)
CA (1) CA3001036A1 (da)
CY (1) CY1124588T1 (da)
DK (1) DK3368678T3 (da)
ES (1) ES2886009T3 (da)
GB (1) GB2547179A (da)
HK (1) HK1252168A1 (da)
HR (1) HRP20211407T1 (da)
HU (1) HUE056911T2 (da)
LT (1) LT3368678T (da)
MX (1) MX2018005286A (da)
PL (1) PL3368678T3 (da)
PT (1) PT3368678T (da)
RS (1) RS62460B1 (da)
RU (1) RU2734678C2 (da)
SI (1) SI3368678T1 (da)
WO (1) WO2017072498A1 (da)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2016332847B2 (en) 2015-10-01 2023-08-10 Goleini Inc. Targeted expression of chloride channels and methods of use thereof
GB201705484D0 (en) * 2017-04-05 2017-05-17 Quethera Ltd Genetic construct
KR20200083495A (ko) 2017-10-20 2020-07-08 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Nt-3 유전자 치료를 위한 방법 및 물질
US20200399656A1 (en) * 2017-12-05 2020-12-24 University Of Washington Compositions and methods for enhancing functional expression of therapeutic genes in photoreceptors
CN108250288A (zh) * 2018-01-17 2018-07-06 吉林省农业科学院 一种叶绿体表达的脑源性神经营养因子hBDNFb蛋白及其制备方法
CN109022487A (zh) * 2018-08-15 2018-12-18 上海市第人民医院 一种表达p65的基因载体及其用于治疗视网膜神经节细胞变性的基因治疗药物
CN113365603A (zh) * 2018-10-11 2021-09-07 分贝治疗公司 Aav1载体及其用于治疗耳部适应症的用途
US20220184231A1 (en) * 2019-03-06 2022-06-16 Generation Bio Co. Closed-ended dna (cedna) and immune modulating compounds
JP2022536692A (ja) 2019-06-11 2022-08-18 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 調節不全の血漿カリクレインによって媒介される疾患の治療のための抗体のアデノ随伴ウイルスベクター送達
US20230124994A1 (en) * 2019-07-18 2023-04-20 Lysogene Compositions and methods for the treatment of sanfilippo disease and other disorders
RU2719013C1 (ru) * 2019-09-25 2020-04-16 Федеральное государственное бюджетное образовательное учреждение высшего образования "Московский государственный университет имени М.В. Ломоносова" (МГУ) Генно-инженерная конструкция для стимуляции посттравматической регенерации периферических нервов
CN115176022A (zh) 2019-10-23 2022-10-11 夏尔人类遗传性治疗公司 用于遗传性血管性水肿的基于腺相关病毒载体的基因疗法
JP2023554066A (ja) 2020-12-16 2023-12-26 武田薬品工業株式会社 血漿カリクレインの制御不全によって媒介される疾患の処置のための抗体のアデノ随伴ウイルスベクター送達
EP4297800A1 (en) * 2021-02-26 2024-01-03 Icahn School of Medicine at Mount Sinai Method for decreasing degeneration of retinal ganglion cells

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5229500A (en) 1989-08-30 1993-07-20 Regeneron Pharmaceuticals, Inc. Brain derived neurotrophic factor
US6225282B1 (en) * 1996-01-05 2001-05-01 Genentech, Inc. Treatment of hearing impairments
WO1998016242A1 (en) * 1996-10-16 1998-04-23 Regeneron Pharmaceuticals, Inc. Method of enhancing the delivery of growth factors
WO1999053959A2 (en) * 1998-04-20 1999-10-28 Koninklijke Nederlandse Akademie Van Wetenschappen Use of viral vectors for treatment of the injured peripheral and central nervous system
EP1343532B1 (en) * 2000-12-19 2013-04-24 Research Development Foundation Lentiviral vector-mediated gene transfer and uses thereof
US7060429B2 (en) * 2001-02-22 2006-06-13 University Of Maryland, Baltimore Treatment of neurodegenerative diseases by altering levels of TrkB isoforms and/or TrkC isoforms
FR2906533B1 (fr) * 2006-09-28 2013-02-22 Pf Medicament Procede de generation d'anticorps actifs contre un antigene de resistance,anticorps obtenus par ledit procede et leurs utilisations
CN101980603A (zh) * 2007-10-11 2011-02-23 比奥根艾迪克Ma公司 LINGO-1和TrkB拮抗剂的用途
WO2009120978A2 (en) * 2008-03-27 2009-10-01 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of bdnf and compositions therfor
CN103614416B (zh) * 2013-09-30 2016-09-07 中国人民解放军第二军医大学东方肝胆外科医院 一种携带人穿膜肽p53与GM-CSF基因的重组溶瘤腺病毒及其用途

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JP2018535696A (ja) 2018-12-06
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EP3368678B1 (en) 2021-07-28
US10342880B2 (en) 2019-07-09
LT3368678T (lt) 2021-11-10
CN108350467B (zh) 2022-06-03
JP2022116040A (ja) 2022-08-09
US11471539B2 (en) 2022-10-18
GB2547179A (en) 2017-08-16
RU2018115760A (ru) 2019-11-28
HRP20211407T1 (hr) 2021-12-10
CA3001036A1 (en) 2017-05-04
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GB201518911D0 (en) 2015-12-09
EP3985119A1 (en) 2022-04-20
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EP3368678A1 (en) 2018-09-05
HK1252168A1 (zh) 2019-05-17
CN108350467A (zh) 2018-07-31
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ES2886009T3 (es) 2021-12-16
US20180353621A1 (en) 2018-12-13
RU2734678C2 (ru) 2020-10-21
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AU2016344508A1 (en) 2018-04-19
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JP7073264B2 (ja) 2022-05-23
KR20180081503A (ko) 2018-07-16
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