JP6942789B2 - Chrimsonを用いた光遺伝学的視覚回復 - Google Patents

Chrimsonを用いた光遺伝学的視覚回復 Download PDF

Info

Publication number
JP6942789B2
JP6942789B2 JP2019508309A JP2019508309A JP6942789B2 JP 6942789 B2 JP6942789 B2 JP 6942789B2 JP 2019508309 A JP2019508309 A JP 2019508309A JP 2019508309 A JP2019508309 A JP 2019508309A JP 6942789 B2 JP6942789 B2 JP 6942789B2
Authority
JP
Japan
Prior art keywords
protein
chrimson
tdt
cells
fused
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.)
Active
Application number
JP2019508309A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019518073A5 (https=
JP2019518073A (ja
Inventor
ディディエ プリュノー
ディディエ プリュノー
アンヌ ドゥアール
アンヌ ドゥアール
デニズ ダルカラ
デニズ ダルカラ
イェンス ドゥーベル
イェンス ドゥーベル
ロマン キャプレット
ロマン キャプレット
グレゴリー ゴーヴァン
グレゴリー ゴーヴァン
メリッサ デロシアーズ
メリッサ デロシアーズ
ホセ サヘル
ホセ サヘル
セルジュ ピコー
セルジュ ピコー
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Centre National de la Recherche Scientifique CNRS
Sorbonne Universite
Original Assignee
Centre National de la Recherche Scientifique CNRS
Sorbonne Universite
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Centre National de la Recherche Scientifique CNRS, Sorbonne Universite filed Critical Centre National de la Recherche Scientifique CNRS
Publication of JP2019518073A publication Critical patent/JP2019518073A/ja
Publication of JP2019518073A5 publication Critical patent/JP2019518073A5/ja
Application granted granted Critical
Publication of JP6942789B2 publication Critical patent/JP6942789B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K36/00Medicinal preparations of undetermined constitution containing material from algae, lichens, fungi or plants, or derivatives thereof, e.g. traditional herbal medicines
    • A61K36/06Fungi, e.g. yeasts
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/168Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from plants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1767Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from invertebrates
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K41/00Medicinal preparations obtained by treating materials with wave energy or particle radiation ; Therapies using these preparations
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/02Drugs for disorders of the nervous system for peripheral neuropathies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/405Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from algae
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62DNA sequences coding for fusion proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62DNA sequences coding for fusion proteins
    • C12N15/625DNA sequences coding for fusion proteins containing a sequence coding for a signal sequence
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645Adeno-associated virus
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/60Fusion polypeptide containing spectroscopic/fluorescent detection, e.g. green fluorescent protein [GFP]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • Botany (AREA)
  • Natural Medicines & Medicinal Plants (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Dermatology (AREA)
  • Marine Sciences & Fisheries (AREA)
JP2019508309A 2016-04-29 2017-04-28 Chrimsonを用いた光遺伝学的視覚回復 Active JP6942789B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Publications (3)

Publication Number Publication Date
JP2019518073A JP2019518073A (ja) 2019-06-27
JP2019518073A5 JP2019518073A5 (https=) 2020-05-07
JP6942789B2 true JP6942789B2 (ja) 2021-09-29

Family

ID=59253830

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2019508309A Active JP6942789B2 (ja) 2016-04-29 2017-04-28 Chrimsonを用いた光遺伝学的視覚回復

Country Status (8)

Country Link
US (2) US20190269755A1 (https=)
EP (1) EP3448411A1 (https=)
JP (1) JP6942789B2 (https=)
KR (1) KR102466887B1 (https=)
CN (1) CN110267673B (https=)
AU (2) AU2017256910B2 (https=)
CA (1) CA3025975A1 (https=)
WO (1) WO2017187272A1 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
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

Family Cites Families (12)

* 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
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
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
WO2015161308A1 (en) * 2014-04-18 2015-10-22 Massachusetts Institute Of Technology Mutant channelrhodopsins with altered ion selectivity
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

Also Published As

Publication number Publication date
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
CN110267673B (zh) 2023-05-16
CN110267673A (zh) 2019-09-20
EP3448411A1 (en) 2019-03-06
CA3025975A1 (en) 2017-11-02
KR102466887B1 (ko) 2022-11-11
JP2019518073A (ja) 2019-06-27
AU2022204884A1 (en) 2022-07-28
WO2017187272A1 (en) 2017-11-02

Similar Documents

Publication Publication Date Title
JP6942789B2 (ja) Chrimsonを用いた光遺伝学的視覚回復
AU2021245195B2 (en) Identification of Channelopsin-2 (Chop2) mutations and methods of use
RU2758211C2 (ru) Synp161, промотор для специфической экспрессии генов в палочковых фоторецепторах
US8754048B2 (en) Light-receiving channel rhodopsin having improved expression efficiency
ES2900486T3 (es) SynP160, un promotor para la expresión específica de genes en los fotorreceptores de los bastones
ES2909444T3 (es) Synp159, un promotor para la expresión específica de genes en los fotorreceptores de los bastones
ES2886664T3 (es) SynP162, un promotor para la expresión específica de genes en fotorreceptores de bastón
ES2881782T3 (es) Promotor para la expresión específica de genes en células endoteliales retinianas.
JP2016529256A (ja) 疼痛を制御するための組成物及び方法
KR20240132305A (ko) 감광성 Gq 접합 뉴롭신(옵신 5)을 사용한 광유전학 시각 회복
AU2017372351B2 (en) Optogenetic modulation by multi-characteristic opsins for vision restoration and other applications thereof
HK1205744B (en) Identification of channelrhodopsin-2 (chop2) mutations and methods of use
NZ727041B2 (en) Identification of channelrhodopsin-2 (chop2) mutations and methods of use

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200327

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20200327

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210413

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210713

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20210715

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20210831

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20210908

R150 Certificate of patent or registration of utility model

Ref document number: 6942789

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250