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1. 发明申请

US20140121265A1 Restoration of Visual Responses by In Vivo Delivery of Rhodopsin Nucleic Acids 有权
标题翻译：通过视紫红质核酸的体内递送恢复视觉反应
公开(公告)号：US20140121265A1
公开(公告)日：2014-05-01
申请号：US13899198
申请日：2013-05-21
申请人： Wayne State University , Salus University
发明人： Zhuo-Hua Pan , Alexander M. Dizhoor
IPC分类号： A61K38/17
CPC分类号： A61K38/177 , A61K38/00 , A61K48/005 , C07K14/705 , C12N15/86 , C12N2750/14143 , C12N2830/002 , C12N2830/008
摘要： Nucleic acid vectors encoding light-gated cation-selective membrane channels, in particular channelrhodopsin-2 (Chop2), converted inner retinal neurons to photosensitive cells in photoreceptor-degenerated retina in an animal model. Such treatment restored visual perception and various aspects of vision. A method of restoring light sensitivity to a retina of a subject suffering from vision loss due to photoreceptor degeneration, as in retinitis pigmentosa or macular degeneration, is provided. The method comprises delivering to the subject by intravitreal or subretinal injection, the above nucleic acid vector which comprises an open reading frame encoding a rhodopsin, to which is operatively linked a promoter and transcriptional regulatory sequences, so that the nucleic acid is expressed in inner retinal neurons. These cells, normally light-insensitive, are converted to a light-sensitive state and transmit visual information to the brain, compensating for the loss, and leading to restoration of various visual capabilities.
摘要翻译：在动物模型中，编码光门控阳离子选择性膜通道的核酸载体，特别是通道视紫红质-2（Chop2），将内视网膜神经元转化为光感受器退化的视网膜中的感光细胞。这种治疗恢复了视觉感知和视觉的各个方面。提供了一种恢复对由于光感受器变性导致的视力丧失的受试者的视网膜的光敏性的方法，如视网膜色素变性或黄斑变性。所述方法包括通过玻璃体内或视网膜下注射向受试者递送上述核酸载体，其包含编码视紫红质的开放阅读框，可操作地连接启动子和转录调节序列，使得核酸在内视网膜中表达神经元。这些细胞通常不敏感，被转换为光敏感状态，并将视觉信息传输给大脑，补偿损失，并导致恢复各种视觉功能。

2. 发明授权

US11883463B2 Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids 有权
公开(公告)号：US11883463B2
公开(公告)日：2024-01-30
申请号：US16700201
申请日：2019-12-02
申请人： Wayne State University , Salus University
发明人： Zhuo-Hua Pan , Alexander M. Dizhoor
IPC分类号： A61K48/00 , A61K38/17 , C07K14/705 , C12N15/86 , A61P27/02 , A61K38/00
CPC分类号： A61K38/177 , A61K48/005 , A61P27/02 , C07K14/705 , C12N15/86 , A61K38/00 , C12N2750/14143 , C12N2830/002 , C12N2830/008
摘要： Nucleic acid vectors encoding light-gated cation-selective membrane channels, in particular channelrhodopsin-2 (Chop2), converted inner retinal neurons to photosensitive cells in photoreceptor-degenerated retina in an animal model. Such treatment restored visual perception and various aspects of vision. A method of restoring light sensitivity to a retina of a subject suffering from vision loss due to photoreceptor degeneration, as in retinitis pigmentosa or macular degeneration, is provided. The method comprises delivering to the subject by intravitreal or subretinal injection, the above nucleic acid vector which comprises an open reading frame encoding a rhodopsin, to which is operatively linked a promoter and transcriptional regulatory sequences, so that the nucleic acid is expressed in inner retinal neurons. These cells, normally light-insensitive, are converted to a light-sensitive state and transmit visual information to the brain, compensating for the loss, and leading to restoration of various visual capabilities.

3. 发明申请

US20210100875A1 RESTORATION OF VISUAL RESPONSES BY IN VIVO DELIVERY OF RHODOPSIN NUCLEIC ACIDS 有权
公开(公告)号：US20210100875A1
公开(公告)日：2021-04-08
申请号：US16700201
申请日：2019-12-02
申请人： Wayne State University , Salus University
发明人： Zhuo-Hua PAN , Alexander M. Dizhoor
IPC分类号： A61K38/17 , A61K48/00 , C07K14/705 , C12N15/86
摘要： Nucleic acid vectors encoding light-gated cation-selective membrane channels, in particular channelrhodopsin-2 (Chop2), converted inner retinal neurons to photosensitive cells in photoreceptor-degenerated retina in an animal model. Such treatment restored visual perception and various aspects of vision. A method of restoring light sensitivity to a retina of a subject suffering from vision loss due to photoreceptor degeneration, as in retinitis pigmentosa or macular degeneration, is provided. The method comprises delivering to the subject by intravitreal or subretinal injection, the above nucleic acid vector which comprises an open reading frame encoding a rhodopsin, to which is operatively linked a promoter and transcriptional regulatory sequences, so that the nucleic acid is expressed in inner retinal neurons. These cells, normally light-insensitive, are converted to a light-sensitive state and transmit visual information to the brain, compensating for the loss, and leading to restoration of various visual capabilities.

4. 发明授权

US09730981B2 Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids 有权
公开(公告)号：US09730981B2
公开(公告)日：2017-08-15
申请号：US13899198
申请日：2013-05-21
申请人： Wayne State University , Salus University
发明人： Zhuo-Hua Pan , Alexander M. Dizhoor
IPC分类号： A61K48/00 , A61K38/17 , C07K14/705 , C12N15/86 , A61K38/00
CPC分类号： A61K38/177 , A61K38/00 , A61K48/005 , C07K14/705 , C12N15/86 , C12N2750/14143 , C12N2830/002 , C12N2830/008
摘要： Nucleic acid vectors encoding light-gated cation-selective membrane channels, in particular channelrhodopsin-2 (Chop2), converted inner retinal neurons to photosensitive cells in photoreceptor-degenerated retina in an animal model. Such treatment restored visual perception and various aspects of vision. A method of restoring light sensitivity to a retina of a subject suffering from vision loss due to photoreceptor degeneration, as in retinitis pigmentosa or macular degeneration, is provided. The method comprises delivering to the subject by intravitreal or subretinal injection, the above nucleic acid vector which comprises an open reading frame encoding a rhodopsin, to which is operatively linked a promoter and transcriptional regulatory sequences, so that the nucleic acid is expressed in inner retinal neurons. These cells, normally light-insensitive, are converted to a light-sensitive state and transmit visual information to the brain, compensating for the loss, and leading to restoration of various visual capabilities.

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