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MX2016007278A - Metodos y composiciones farmaceuticas para la expresion de un polinucleotido de interes en el epitelio pigmentario retiniano de un sujeto. - Google Patents

Metodos y composiciones farmaceuticas para la expresion de un polinucleotido de interes en el epitelio pigmentario retiniano de un sujeto.

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Publication number
MX2016007278A
MX2016007278A MX2016007278A MX2016007278A MX2016007278A MX 2016007278 A MX2016007278 A MX 2016007278A MX 2016007278 A MX2016007278 A MX 2016007278A MX 2016007278 A MX2016007278 A MX 2016007278A MX 2016007278 A MX2016007278 A MX 2016007278A
Authority
MX
Mexico
Prior art keywords
polynucleotide
interest
retinal pigment
pigment epithelium
subject
Prior art date
Application number
MX2016007278A
Other languages
English (en)
Other versions
MX379027B (es
Inventor
hamel Christian
Kalatzis Vasiliki
Pequignot Marie
CERESO Nicolas
Original Assignee
Inserm (Institut Nat De La Santé Et De La Rech Médicale)
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 Nat De La Santé Et De La Rech Médicale) filed Critical Inserm (Institut Nat De La Santé Et De La Rech Médicale)
Publication of MX2016007278A publication Critical patent/MX2016007278A/es
Publication of MX379027B publication Critical patent/MX379027B/es

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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
    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • 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
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    • A—HUMAN NECESSITIES
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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
    • A—HUMAN NECESSITIES
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Abstract

La presente invención se refiere a métodos y a composiciones farmacéuticas para la expresión de un polinucleótido de interés en el epitelio pigmentario retiniano de un sujeto. En particular, la presente invención se refiere a un método para expresar selectivamente un polinucleótido de interés en el epitelio pigmentario retiniano en un ojo de un sujeto con necesidad del mismo que comprende la etapa de transducir el epitelio pigmentario retiniano con una cantidad de un vector rAAV2/5 que contiene el polinucleótido de interés.
MX2016007278A 2013-12-06 2014-12-05 Metodos y composiciones farmaceuticas para la expresion de un polinucleotido de interes en el epitelio pigmentario retiniano de un sujeto. MX379027B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP13306676 2013-12-06
PCT/EP2014/076740 WO2015082690A1 (en) 2013-12-06 2014-12-05 Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject

Publications (2)

Publication Number Publication Date
MX2016007278A true MX2016007278A (es) 2016-09-16
MX379027B MX379027B (es) 2025-03-10

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MX2016007278A MX379027B (es) 2013-12-06 2014-12-05 Metodos y composiciones farmaceuticas para la expresion de un polinucleotido de interes en el epitelio pigmentario retiniano de un sujeto.

Country Status (14)

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

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GB201604146D0 (en) * 2016-03-10 2016-04-27 Nightstarx Ltd 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 (en) 2016-10-11 2020-04-29 Wellstat Ophthalmics Corporation FUSION PROTEIN BETWEEN A SHORT-ROD-DERIVED CONE VIABILITY FACTOR AND A HYDROPHILIC PEPTIDE
EP3534967A4 (en) 2016-11-07 2020-07-22 Spark Therapeutics, Inc. POTENTIAL TEST FOR RAB ESCORT PROTEIN
CN111630170B (zh) * 2017-07-31 2025-03-07 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
CA3079565A1 (en) * 2017-10-18 2019-04-25 Regenxbio Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified vegf-trap
MX2020010477A (es) * 2018-04-05 2021-03-02 Univ Oxford Innovation Ltd Composiciones y metodos para el tratamiento de la distrofia macular.
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
SG11202109554XA (en) 2019-03-04 2021-09-29 Univ Pennsylvania Neuroprotective gene therapy targeting the akt pathway
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 (en) * 2022-05-02 2023-11-09 Fondazione Telethon Ets Gene therapy for gyrate atrophy of the choroid and retina

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DK3023500T3 (da) * 2006-06-21 2020-04-20 Uniqure Ip Bv Insektceller til fremstilling af AAV-vektorer
JP2011511090A (ja) * 2008-02-07 2011-04-07 セレジーン インコーポレイテッド 治療用タンパク質をコードする発現ベクターの硝子体内投与による光受容体のレスキュー方法
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
WO2011034947A2 (en) * 2009-09-15 2011-03-24 University Of Washington Reagents and methods for modulating cone photoreceptor activity
GB201103062D0 (en) * 2011-02-22 2011-04-06 Isis Innovation Method

Also Published As

Publication number Publication date
TN2016000220A1 (en) 2017-10-06
KR102281881B1 (ko) 2021-07-27
JP2017500060A (ja) 2017-01-05
MA39082B2 (fr) 2021-11-30
CN105940109A (zh) 2016-09-14
KR20170009812A (ko) 2017-01-25
JP6875856B2 (ja) 2021-05-26
CA2932495A1 (en) 2015-06-11
RU2723101C2 (ru) 2020-06-08
JP2020023568A (ja) 2020-02-13
IL245989A0 (en) 2016-07-31
US10105452B2 (en) 2018-10-23
BR112016012716A2 (pt) 2020-11-24
WO2015082690A1 (en) 2015-06-11
AU2014359136A1 (en) 2016-06-16
MA39082A1 (fr) 2017-10-31
MX379027B (es) 2025-03-10
US20160310618A1 (en) 2016-10-27
AU2014359136B2 (en) 2020-06-25
EP3077520A1 (en) 2016-10-12

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