MA39082A1 - Méthodes et compositions pharmaceutiques pour exprimer un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien d'un sujet - Google Patents

Méthodes et compositions pharmaceutiques pour exprimer un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien d'un sujet

Info

Publication number
MA39082A1
MA39082A1 MA39082A MA39082A MA39082A1 MA 39082 A1 MA39082 A1 MA 39082A1 MA 39082 A MA39082 A MA 39082A MA 39082 A MA39082 A MA 39082A MA 39082 A1 MA39082 A1 MA 39082A1
Authority
MA
Morocco
Prior art keywords
polynucleotide
interest
retinal pigment
subject
pigment epithelium
Prior art date
Application number
MA39082A
Other languages
English (en)
Other versions
MA39082B2 (fr
Inventor
Christian Hamel
Vasiliki Kalatzis
Marie Pequignot
Nicolas Cereso
Original Assignee
Inserm (Institut National De La Santé Et De La Rech Médicale)
Ct Hospitalier Universitaire Montpellier
Univ Montpellier
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 Inserm (Institut National De La Santé Et De La Rech Médicale), Ct Hospitalier Universitaire Montpellier, Univ Montpellier filed Critical Inserm (Institut National De La Santé Et De La Rech Médicale)
Publication of MA39082A1 publication Critical patent/MA39082A1/fr
Publication of MA39082B2 publication Critical patent/MA39082B2/fr

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Dermatology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)

Abstract

L'invention concerne des méthodes et des compositions pharmaceutiques pour exprimer un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien d'un sujet. L'invention concerne, en particulier, une méthode pour exprimer sélectivement un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien dans l'œil d'un sujet qui en a besoin, comprenant l'étape consistant à transduire l'épithélium pigmentaire rétinien avec une quantité de vecteur raav2/5 contenant le polynucléotide d'intérêt.
MA39082A 2013-12-06 2014-12-05 Méthodes et compositions pharmaceutiques pour exprimer un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien d'un sujet MA39082B2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP13306676 2013-12-06
PCT/EP2014/076740 WO2015082690A1 (fr) 2013-12-06 2014-12-05 Méthodes et compositions pharmaceutiques pour exprimer un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien d'un sujet

Publications (2)

Publication Number Publication Date
MA39082A1 true MA39082A1 (fr) 2017-10-31
MA39082B2 MA39082B2 (fr) 2021-11-30

Family

ID=49885103

Family Applications (1)

Application Number Title Priority Date Filing Date
MA39082A MA39082B2 (fr) 2013-12-06 2014-12-05 Méthodes et compositions pharmaceutiques pour exprimer un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien d'un sujet

Country Status (14)

Country Link
US (1) US10105452B2 (fr)
EP (1) EP3077520A1 (fr)
JP (2) JP6875856B2 (fr)
KR (1) KR102281881B1 (fr)
CN (1) CN105940109A (fr)
AU (1) AU2014359136B2 (fr)
BR (1) BR112016012716A2 (fr)
CA (1) CA2932495A1 (fr)
IL (1) IL245989A0 (fr)
MA (1) MA39082B2 (fr)
MX (1) MX379027B (fr)
RU (1) RU2723101C2 (fr)
TN (1) TN2016000220A1 (fr)
WO (1) WO2015082690A1 (fr)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190017096A1 (en) * 2016-03-10 2019-01-17 Nightstarx Limited Prenylation assay
CN107287238B (zh) * 2016-04-11 2020-10-16 厦门继景生物技术有限责任公司 一种基因载体及其用于治疗雷柏氏先天性黑矇2型疾病的基因治疗药物
EP3526238A4 (fr) 2016-10-11 2020-04-29 Wellstat Ophthalmics Corporation Protéine de fusion entre un facteur de viabilité de cône dérivé d'une tige courte et un peptide hydrophile
WO2018085844A1 (fr) * 2016-11-07 2018-05-11 Spark Therapeutics, Inc. Dosage d'activité de protéine d'escorte de rab
CN120989074A (zh) 2017-07-31 2025-11-21 映像生物有限公司 眼部疾病的细胞模型及用于眼部疾病的疗法
WO2019079494A1 (fr) * 2017-10-18 2019-04-25 Regenxbio, Inc. Traitement de maladies oculaires et d'un cancer du côlon métastatique avec un piège à vegf avec modification post-traductionnelle humain
RU2020132890A (ru) * 2018-04-05 2022-05-06 Оксфорд Юниверсити Инновейшн Лимитед Композиции и способы лечения макулярной дистрофии
CN109136266B (zh) * 2018-08-10 2022-02-18 深圳泓熙生物科技发展有限公司 用于治疗或预防结晶样视网膜色素变性的基因载体及其用途
CN109112134B (zh) * 2018-09-28 2020-06-02 中国人民解放军陆军军医大学第一附属医院 一种视网膜变性疾病的best1新突变致病基因及其试剂盒
RU2732479C2 (ru) * 2018-12-21 2020-09-17 Селл энд Джин Терапи Лтд Генотерапевтический ДНК-вектор на основе генотерапевтического ДНК-вектора VTvaf17, несущий целевой ген, выбранный из группы генов BDNF, VEGFA, BFGF, NGF, GDNF, NT3, CNTF, IGF1, для повышения уровня экспрессии этих целевых генов, способ его получения и применения, штамм Escherichia coli SCS110-AF/VTvaf17-BDNF, или Escherichia coli SCS110-AF/VTvaf17-VEGFA, или Escherichia coli SCS110-AF/VTvaf17-BFGF, или Escherichia coli SCS110-AF/VTvaf17-NGF, или Escherichia coli SCS110-AF/VTvaf17-GDNF, или Escherichia coli SCS110-AF/VTvaf17-NT3, или Escherichia coli SCS110-AF/VTvaf17-CNTF, или Escherichia coli SCS110-AF/VTvaf17-IGF1, несущий генотерапевтический ДНК-вектор, способ его получения, способ производства в промышленных масштабах генотерапевтического ДНК-вектора
WO2020180886A1 (fr) * 2019-03-04 2020-09-10 The Trustees Of The University Of Pennsylvania Thérapie génique neuroprotectrice ciblant la voie akt
CN110455655B (zh) * 2019-08-23 2024-05-28 水利部杭州机械设计研究所 一种热喷涂涂层高通量检测装置及测试方法
CN110791502B (zh) * 2019-12-04 2022-07-08 陕西理工大学 人源rbp4启动子片段及其应用
CN113952472A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
WO2023213817A1 (fr) * 2022-05-02 2023-11-09 Fondazione Telethon Ets Thérapie génique pour l'atrophie gyrate de la choroïde et de la rétine

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2921594A1 (fr) * 2006-06-21 2007-12-27 Uniqure Ip B.V. Vecteurs aav dotes de sequences de codage rep destines a la production de cellules d'insecte
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
US20120172419A1 (en) * 2009-09-15 2012-07-05 Medical College Of Wisconsin Research Foundation Inc. Reagents and methods for modulating cone photoreceptor activity
WO2011146121A1 (fr) 2010-05-17 2011-11-24 Sangamo Biosciences, Inc. Nouvelles protéines se liant à l'adn et leurs utilisations
GB201103062D0 (en) * 2011-02-22 2011-04-06 Isis Innovation Method

Also Published As

Publication number Publication date
EP3077520A1 (fr) 2016-10-12
CN105940109A (zh) 2016-09-14
WO2015082690A1 (fr) 2015-06-11
MX2016007278A (es) 2016-09-16
AU2014359136B2 (en) 2020-06-25
JP6875856B2 (ja) 2021-05-26
IL245989A0 (en) 2016-07-31
BR112016012716A2 (pt) 2020-11-24
KR20170009812A (ko) 2017-01-25
AU2014359136A1 (en) 2016-06-16
KR102281881B1 (ko) 2021-07-27
JP2020023568A (ja) 2020-02-13
US10105452B2 (en) 2018-10-23
TN2016000220A1 (en) 2017-10-06
MA39082B2 (fr) 2021-11-30
CA2932495A1 (fr) 2015-06-11
MX379027B (es) 2025-03-10
JP2017500060A (ja) 2017-01-05
US20160310618A1 (en) 2016-10-27
RU2723101C2 (ru) 2020-06-08

Similar Documents

Publication Publication Date Title
MA39082A1 (fr) Méthodes et compositions pharmaceutiques pour exprimer un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien d'un sujet
EP4393495A3 (fr) Compositions d'arni de ligand 1 de mort cellulaire programmée 1 (pd-l1) et leurs procédés d'utilisation
EP3897747A4 (fr) Molécules bi-fonctionnelles pour le ciblage des lysosomes, compositions et méthodes associées
PH12019550076A1 (en) SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP3177201C0 (fr) Systèmes et méthodes pour evaluer une activité neuronale dans l'oeil
SG10201903823QA (en) Methods and compositions relating to microbial treatment and diagnosis of disorders
EP3523419A4 (fr) Compositions et méthodes pour déterminer une viabilité cellulaire
MA38814B2 (fr) Agoniste gitr et antagoniste pd-1 pour leurs utilisations dans le traitement de troubles proliferatifs
WO2017019660A8 (fr) Compositions d'arni de xanthine déshydrogénase et leurs méthodes d'utilisation
WO2017044857A3 (fr) Méthodes et compositions pour le traitement du glaucome
MA38261A1 (fr) Composes comprenant un noyau polycyclique destines a etre utilises dans le traitement de l'hepatite c (vhc)
WO2015179724A8 (fr) Compositions d'arni produisant un effet sur l'angiotensinogène (agt) et leurs procédés d'utilisation
WO2014190157A8 (fr) Compositions de tmprss6 et méthodes d'utilisation de ces compositions
EP3490366A4 (fr) Procédés et compositions pour l'expression de gènes dans des plantes
MA38174A1 (fr) Souche de bifidobacterium animalis ssp. Animalis
EP3393436C0 (fr) Composition comprenant une alkylcellulose, des huiles non compatibles siliconées et hydrocarbonées et une méthode pour l'utiliser.
IL251866A0 (en) Identifying an individual's abilities, skills and interests through game data analysis
MX2018002102A (es) Concentrados en suspension aceitosa con baja separacion gravimetrica y baja viscosidad.
EP3641752A4 (fr) Méthodes de traitement du cancer au moyen de compositions comprenant de l'amlexanox et des immunomodulateurs
EP4218790A8 (fr) Modulateurs de l'activité du complément
EP3413981A4 (fr) Compositions contenant une capsule dérivée de cellulose avec un agent actif d'écran solaire
MA39347A1 (fr) Protéines de fusion d'uti
EP3445406A4 (fr) Compositions et méthodes permettant d'obtenir une expression améliorée du gène de pklr
MX2017009103A (es) Metodo y aparato para evaluar la eficacia del tratamiento de la sensibilidad dental con un producto para el cuidado bucal.
MA38142A1 (fr) Composition comprenant un agent de lutte biologique et un fongicide