CN110267673B - 利用chrimson来进行光遗传视觉恢复 - Google Patents

利用chrimson来进行光遗传视觉恢复 Download PDF

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CN110267673B
CN110267673B CN201780041321.8A CN201780041321A CN110267673B CN 110267673 B CN110267673 B CN 110267673B CN 201780041321 A CN201780041321 A CN 201780041321A CN 110267673 B CN110267673 B CN 110267673B
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light
protein
ion channel
cells
vector
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CN110267673A (zh
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迪迪尔·普陆瑙
安妮·杜瓦
丹尼兹·达尔卡拉
简斯·度贝尔
罗梅因·卡普莱特
格利高里·高文
梅莉萨·德罗歇尔斯
何塞·萨赫勒
塞尔吉·皮科
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Cherish Biological Co ltd
Centre National de la Recherche Scientifique CNRS
Institut National de la Sante et de la Recherche Medicale INSERM
Sorbonne Universite
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Cherish Biological Co ltd
Centre National de la Recherche Scientifique CNRS
Institut National de la Sante et de la Recherche Medicale INSERM
Sorbonne Universite
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CN201780041321.8A 2016-04-29 2017-04-28 利用chrimson来进行光遗传视觉恢复 Active CN110267673B (zh)

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US201662329692P 2016-04-29 2016-04-29
US62/329,692 2016-04-29
PCT/IB2017/000663 WO2017187272A1 (en) 2016-04-29 2017-04-28 Optogenetic visual restoration using chrimson

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CN110267673A CN110267673A (zh) 2019-09-20
CN110267673B true CN110267673B (zh) 2023-05-16

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US (2) US20190269755A1 (https=)
EP (1) EP3448411A1 (https=)
JP (1) JP6942789B2 (https=)
KR (1) KR102466887B1 (https=)
CN (1) CN110267673B (https=)
AU (2) AU2017256910B2 (https=)
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Families Citing this family (9)

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Publication number Priority date Publication date Assignee Title
WO2016094850A1 (en) 2014-12-12 2016-06-16 The Trustees Of The University Of Pennsylvania Methods of Promoting Nervous System Regeneration
EP3442409A4 (en) 2016-04-14 2019-11-13 The Trustees of The University of Pennsylvania IMPLANTABLE LIVING ELECTRODES AND METHOD FOR USE THEREOF
EP3693061A1 (en) 2019-02-05 2020-08-12 Gensight Biologics Method for controlling an optogenetic device using filtering and associated devices
EP3693060A1 (en) 2019-02-05 2020-08-12 Gensight Biologics Method for controlling an optogenetic device using a command law for the radiant power of a light source and associated devices
EP3733139A1 (en) 2019-05-02 2020-11-04 Gensight Biologics Viewing apparatus and method for projecting a light signal
WO2021132903A1 (ko) * 2019-12-26 2021-07-01 고려대학교 산학협력단 광유전학 기법을 이용한 알츠하이머 질환 뇌에서의 뇌파 손상 회복 방법
US20250235585A1 (en) * 2022-02-18 2025-07-24 The Trustees Of The University Of Pennsylvania Tissue Engineered Spinal Tracts For Functional Regeneration After Spinal Cord Injury
EP4568710A1 (en) 2022-08-11 2025-06-18 Institute Of Molecular And Clinical Ophthalmology Basel (Iob) Human cone photoreceptor optogenetic constructs
CN119827471B (zh) * 2025-02-14 2025-10-03 中山大学中山眼科中心 一种体内追踪外段蛋白运输的方法

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101484005A (zh) * 2006-05-04 2009-07-15 韦恩州立大学 通过向体内递送视紫红质核酸恢复视觉响应
WO2015161308A1 (en) * 2014-04-18 2015-10-22 Massachusetts Institute Of Technology Mutant channelrhodopsins with altered ion selectivity

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10216005A1 (de) 2002-04-11 2003-10-30 Max Planck Gesellschaft Verwendung von biologischen Photorezeptoren als direkt lichtgesteuerte Ionenkanäle
EP1919497B1 (en) 2005-07-22 2020-02-12 The Board of Trustees of the Leland Stanford Junior University Light-activated cation channel and uses thereof
EP1891976A1 (en) 2006-08-23 2008-02-27 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute for Biomedical Research Use of light sensitive genes
CN101980668A (zh) * 2008-02-07 2011-02-23 赛拉基因公司 通过玻璃体内施用编码治疗用蛋白质的表达载体救援光感器
MX2010011467A (es) 2008-04-18 2011-02-15 Novartis Forschungsstiftung Novedosas herramientas y metodos terapeuticos para tratar la ceguera.
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
WO2013071231A1 (en) 2011-11-12 2013-05-16 Massachusetts Institute Of Technology Channelrhodopsins for optical control of cells
US10882892B2 (en) * 2014-08-05 2021-01-05 Massachusetts Institute Of Technology Channelrhodopsin variants and uses thereof
EP3371572B1 (en) * 2015-11-05 2021-05-05 Inscopix, Inc. System for optogenetic imaging
WO2017093566A1 (en) * 2015-12-04 2017-06-08 Universite Pierre Et Marie Curie (Paris 6) Promoters and uses thereof

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101484005A (zh) * 2006-05-04 2009-07-15 韦恩州立大学 通过向体内递送视紫红质核酸恢复视觉响应
WO2015161308A1 (en) * 2014-04-18 2015-10-22 Massachusetts Institute Of Technology Mutant channelrhodopsins with altered ion selectivity

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Optogenetics;JENS DUEBEL;《CURRENT OPINION IN OPHTHALMOLOGY》;20150501;第26卷(第3期);第226-232页 *

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AU2017256910B2 (en) 2022-04-07
US20190269755A1 (en) 2019-09-05
US20240165198A1 (en) 2024-05-23
KR20190058383A (ko) 2019-05-29
WO2017187272A8 (en) 2018-12-13
AU2017256910A1 (en) 2018-11-22
CN110267673A (zh) 2019-09-20
EP3448411A1 (en) 2019-03-06
CA3025975A1 (en) 2017-11-02
JP6942789B2 (ja) 2021-09-29
KR102466887B1 (ko) 2022-11-11
JP2019518073A (ja) 2019-06-27
AU2022204884A1 (en) 2022-07-28
WO2017187272A1 (en) 2017-11-02

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