公开(公告)号：US20190263883A1

公开(公告)日：2019-08-29

申请号：US16289081

申请日：2019-02-28

申请人： THE GENERAL HOSPITAL CORPORATION

发明人： Richard H. MASLAND

IPC分类号： C07K14/705 ,  A61K48/00 ,  A61K9/00 ,  A61K38/17 ,  C12N7/00

摘要： The invention provides compositions and methods of treating subjects afflicted with a photoreceptor disorder. Methods for treating a subject suffering from a disorder characterized by photoreceptor cell degeneration are provided, wherein a gene encoding a photosensitive protein is introduced into a retinal cell of a subject. In one aspect of the invention, the retinal cells which receive the photosensitive protein include non-photoreceptor cells such as horizontal cells, amacrine cells, bipolar cells, and ganglion cells.

公开(公告)号：US20160022841A1

公开(公告)日：2016-01-28

申请号：US14875174

申请日：2015-10-05

申请人： THE GENERAL HOSPITAL CORPORATION

发明人： Richard H. MASLAND

IPC分类号： A61K48/00 ,  C12N7/00 ,  A61K9/00 ,  A61K38/17

CPC分类号： C07K14/705 ,  A01K2217/05 ,  A01K2267/03 ,  A61K9/0048 ,  A61K38/177 ,  A61K48/00 ,  A61K48/0075 ,  C12N7/00 ,  C12N2710/10043 ,  C12N2710/10071 ,  C12N2710/16043 ,  C12N2710/16071 ,  C12N2710/16243 ,  C12N2710/16271 ,  C12N2710/22043 ,  C12N2710/22071 ,  C12N2710/24043 ,  C12N2710/24071 ,  C12N2740/10043 ,  C12N2740/10071 ,  C12N2740/15043 ,  C12N2740/15071 ,  C12N2799/021 ,  C12N2799/04 ,  C12N2830/008

摘要： The invention provides compositions and methods of treating subjects afflicted with a photoreceptor disorder. Methods for treating a subject suffering from a disorder characterized by photoreceptor cell degeneration are provided, wherein a gene encoding a photosensitive protein is introduced into a retinal cell of a subject. In one aspect of the invention, the retinal cells which receive the photosensitive protein include non-photoreceptor cells such as horizontal cells, amacrine cells, bipolar cells, and ganglion cells.

摘要翻译： 本发明提供了治疗患有感光细胞病症的受试者的组合物和方法。 提供了治疗患有感光细胞变性特征性疾病的受试者的方法，其中将编码感光性蛋白质的基因导入受试者的视网膜细胞。 在本发明的一个方面，接受感光性蛋白质的视网膜细胞包括非感光细胞，例如水平细胞，无长突细胞，双极细胞和神经节细胞。