METHODS AND PHARMACEUTICAL COMPOSITIONS FOR EXPRESSING A POLYNUCLEOTIDE OF INTEREST IN THE RETINAL PIGMENT EPITHELIUM OF A SUBJECT

The present invention relates to methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject. In particular, the present invention relates to a method for selectively expressing a polynucleotide of interest in the retinal pigment...

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Hauptverfasser: Nicolas CERESO, Christian HAMEL, Vasiliki KALATZIS, Marie PEQUIGNOT
Format: Patent
Sprache:eng ; spa
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Zusammenfassung:The present invention relates to methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject. In particular, the present invention relates to a method for selectively expressing a polynucleotide of interest in the retinal pigment epithelium in an eye of a subject in need thereof comprising the step of transducing the retinal pigment epithelium with an amount of a rAAV2/5 vector containing the polynucleotide of interest. 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.