| 发明名称 | Widespread gene delivery to the retina using systemic administration of AAV vectors | ||
| 摘要 | The present invention relates to compositions and methods, in particular to methods based on systemic administration of scAAV, for delivering genes to cells of the retina of mammals, and in particular to photoreceptor cells, ganglion cells, glial cells, inner nuclear layer cells or cells of the retinal pigmented epithelium. | ||
| 申请公布号 | US9114161(B2) | 申请公布日期 | 2015.08.25 |
| 申请号 | US201013388083 | 申请日期 | 2010.07.30 |
| 申请人 | ASSOCIATION INSTITUT DE MYOLOGIE;GENETHON;CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE;INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE);UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6) | 发明人 | Barkats Martine |
| 分类号 | A61K48/00;C12N15/861;A61P27/02 | 主分类号 | A61K48/00 |
| 代理机构 | Saliwanchik, Lloyd & Eisenschenk | 代理人 | Saliwanchik, Lloyd & Eisenschenk |
| 主权项 | 1. A method of treating a disorder of the eye, the method comprising the systemic administration of a double stranded self-complementary AAV (scAAV) vector comprising a therapeutic gene to a subject having the disorder of the eye, and wherein said method comprises delivery of the therapeutic gene to photoreceptor cells, glial cells, cells of the retinal pigmented epithelium (RPE) or cells of the ciliary body. | ||
| 地址 | Paris FR | ||