METHODS AND PHARMACEUTICAL COMPOSITIONS FOR EXPRESSING A POLYNUCLEOTIDE OF INTEREST IN THE RETINAL PIGMENT EPITHELIUM OF A SUBJECT
摘要
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.
申请公布号
WO2015082690(A1)
申请公布日期
2015.06.11
申请号
WO2014EP76740
申请日期
2014.12.05
申请人
INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE);CENTRE HOSPITALIER UNIVERSITAIRE DE MONTPELLIER;UNIVERSITE DE MONTPELLIER
发明人
HAMEL, CHRISTIAN;KALATZIS, VASILIKI;PEQUIGNOT, MARIE;CERESO, NICOLAS