MX379027B - 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
MX379027B
MX379027B MX2016007278A MX2016007278A MX379027B MX 379027 B MX379027 B MX 379027B MX 2016007278 A MX2016007278 A MX 2016007278A MX 2016007278 A MX2016007278 A MX 2016007278A MX 379027 B MX379027 B MX 379027B
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MX
Mexico
Prior art keywords
polynucleotide
interest
retinal pigment
subject
pigment epithelium
Prior art date
Application number
MX2016007278A
Other languages
English (en)
Other versions
MX2016007278A (es
Inventor
Christian Hamel
Marie Pequignot
Nicolas Cereso
Vasiliki Kalatzis
Original Assignee
Inst Nat Sante Rech Med
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 Inst Nat Sante Rech Med, Ct Hospitalier Universitaire Montpellier, Univ Montpellier filed Critical Inst Nat Sante Rech Med
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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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • 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
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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 MX2016007278A (es) 2016-09-16
MX379027B true 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
AU2017344059B2 (en) 2016-10-11 2021-12-23 Pharma Cinq, Llc Fusion protein between short form rod-derived cone viability factor and a hydrophilic peptide
CN110177579A (zh) * 2016-11-07 2019-08-27 星火治疗股份有限公司 Rab保护蛋白效价测定
TWI820034B (zh) 2017-07-31 2023-11-01 香港商映像生物有限公司 眼部疾病之細胞模式及用於眼部疾病的療法
WO2019079494A1 (en) * 2017-10-18 2019-04-25 Regenxbio, Inc. TREATMENT OF OCULAR DISEASES AND METASTATIC COLON CANCER WITH A VEGF TRAP WITH HUMAN POST-TRANSLATIONAL MODIFICATION
WO2019195727A1 (en) * 2018-04-05 2019-10-10 Oxford University Innovation Limited Compositions and methods for treating macular dystrophy
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
US20220143217A1 (en) * 2019-03-04 2022-05-12 The Trustees Of The University Of 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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Publication number Priority date Publication date Assignee Title
US8512981B2 (en) * 2006-06-21 2013-08-20 Amsterdam Molecular Therapeutics B.V. Vectors with modified initiation codon for the translation of AAV-Rep78 useful for production of AAV
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
US20120172419A1 (en) * 2009-09-15 2012-07-05 Medical College Of Wisconsin Research Foundation Inc. Reagents and methods for modulating cone photoreceptor activity
JP6208580B2 (ja) 2010-05-17 2017-10-04 サンガモ セラピューティクス， インコーポレイテッド 新規のｄｎａ結合タンパク質及びその使用
GB201103062D0 (en) * 2011-02-22 2011-04-06 Isis Innovation Method

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US20160310618A1 (en) 2016-10-27
JP6875856B2 (ja) 2021-05-26
KR102281881B1 (ko) 2021-07-27
EP3077520A1 (en) 2016-10-12
IL245989A0 (en) 2016-07-31
BR112016012716A2 (pt) 2020-11-24
AU2014359136A1 (en) 2016-06-16
MX2016007278A (es) 2016-09-16
CN105940109A (zh) 2016-09-14
WO2015082690A1 (en) 2015-06-11
KR20170009812A (ko) 2017-01-25
RU2723101C2 (ru) 2020-06-08
CA2932495A1 (en) 2015-06-11
MA39082B2 (fr) 2021-11-30
US10105452B2 (en) 2018-10-23
JP2017500060A (ja) 2017-01-05
JP2020023568A (ja) 2020-02-13
AU2014359136B2 (en) 2020-06-25
MA39082A1 (fr) 2017-10-31
TN2016000220A1 (en) 2017-10-06

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