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1. 发明公开

US20230310657A1 MODIFIED MGLUR6 PROMOTER AND METHODS OF USE 审中-公开
公开(公告)号：US20230310657A1
公开(公告)日：2023-10-05
申请号：US18152895
申请日：2023-01-11
申请人： Wayne State University
发明人： Zhuo-Hua Pan , Qi Lu , Tushar H. Ganjawala , JrGang Cheng
IPC分类号： C12N15/85 , A61K48/00 , C12N15/67 , C07K14/705 , A61K38/17 , A61K9/00 , C12N15/86
CPC分类号： A61K48/0058 , A61K9/0048 , A61K38/1787 , A61K48/0075 , C07K14/70571 , C12N15/67 , C12N15/85 , C12N15/86 , C12N2750/14143 , C12N2830/008 , C12N2830/42
摘要： The invention provides nucleic acids and nucleic acid expression vectors containing optimized mGluR6 promoters for expression of transgenes in the retina. The compositions and methods of the invention are useful for expression of gene products to preserve, improve, or restore phototransduction or vision.

2. 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。

3. 发明授权

US11583595B2 Modified mGluR6 promoter and methods of use 有权
公开(公告)号：US11583595B2
公开(公告)日：2023-02-21
申请号：US16395839
申请日：2019-04-26
申请人： Wayne State University
发明人： Zhuo-Hua Pan , Qi Lu , Tushar H. Ganjawala , JrGang Cheng
IPC分类号： A61K48/00 , C12N15/67 , C12N15/86 , C07K14/705 , C12N15/85 , A61K9/00 , A61K38/17
摘要： The invention provides nucleic acids and nucleic acid expression vectors containing optimized mGluR6 promoters for expression of transgenes in the retina. The compositions and methods of the invention are useful for expression of gene products to preserve, improve, or restore phototransduction or vision.

4. 发明授权

US09730888B2 Method of enhancing delivery of therapeutic compounds to the eye 有权
公开(公告)号：US09730888B2
公开(公告)日：2017-08-15
申请号：US14777420
申请日：2014-03-13
申请人： Wayne State University
发明人： Zhuo-Hua Pan , Elena Ivanova
IPC分类号： A61K48/00 , A61K9/00 , A61K38/36 , A61K31/7088 , A61K31/713 , A61K36/05 , A61K38/16 , A61K38/48
CPC分类号： A61K9/0048 , A61K31/7088 , A61K31/713 , A61K36/05 , A61K38/16 , A61K38/36 , A61K38/484 , A61K48/0008 , A61K48/0075 , C12Y304/21007
摘要： The invention provides methods for enhancing the delivery of therapeutic compounds to the eye of a subject by administering plasmin or derivatives thereof and the therapeutic compounds to the eye.

5. 发明申请

US20190241628A1 IDENTIFICATION OF MUTATIONS IN CHANNELOPSIN VARIANTS HAVING IMPROVED LIGHT SENSITIVITY AND METHODS OF USE THEREOF 审中-公开
公开(公告)号：US20190241628A1
公开(公告)日：2019-08-08
申请号：US16328916
申请日：2017-08-29
申请人： Wayne State University
发明人： Zhuo-Hua Pan
IPC分类号： C07K14/405 , A61P27/02
CPC分类号： C07K14/405 , A61K35/00 , A61K38/00 , A61P27/02
摘要： The invention provides compositions and kits including at least one nucleic acid or polypeptide molecule encoding for a mutant CoChop protein. Methods of the invention include administering a composition comprising a mutant CoChop to a subject to preserve, improve, or restore phototransduction. Preferably, the compositions and methods of the invention are provided to a subject having impaired vision, thereby restoring vision to normal levels.

6. 发明申请

US20220089660A1 IDENTIFICATION OF MUTATIONS IN CHANNELOPSIN VARIANTS HAVING IMPROVED LIGHT SENSITIVITY AND METHODS OF USE THEREOF 有权
公开(公告)号：US20220089660A1
公开(公告)日：2022-03-24
申请号：US17324501
申请日：2021-05-19
申请人： Wayne State University
发明人： Zhuo-Hua Pan
IPC分类号： C07K14/405 , A61P27/02
摘要： The invention provides compositions and kits including at least one nucleic acid or polypeptide molecule encoding for a mutant CoChop protein. Methods of the invention include administering a composition comprising a mutant CoChop to a subject to preserve, improve, or restore phototransduction. Preferably, the compositions and methods of the invention are provided to a subject having impaired vision, thereby restoring vision to normal levels.

7. 发明授权

US11041004B2 Identification of mutations in channelopsin variants having improved light sensitivity and methods of use thereof 有权
公开(公告)号：US11041004B2
公开(公告)日：2021-06-22
申请号：US16328916
申请日：2017-08-29
申请人： Wayne State University
发明人： Zhuo-Hua Pan
IPC分类号： C07K14/405 , A61P27/02 , A61K38/00 , A61K35/00
摘要： The invention provides compositions and kits including at least one nucleic acid or polypeptide molecule encoding for a mutant CoChop protein. Methods of the invention include administering a composition comprising a mutant CoChop to a subject to preserve, improve, or restore phototransduction. Preferably, the compositions and methods of the invention are provided to a subject having impaired vision, thereby restoring vision to normal levels.

8. 发明申请

US20200093938A1 MODIFIED MGLUR6 PROMOTER AND METHODS OF USE 审中-公开
公开(公告)号：US20200093938A1
公开(公告)日：2020-03-26
申请号：US16395839
申请日：2019-04-26
申请人： Wayne State University
发明人： Zhuo-Hua Pan , Qi Lu , Tushar H. Ganjawala , JrGang Cheng
IPC分类号： A61K48/00 , C12N15/67 , C07K14/705 , C12N15/85 , C12N15/86 , A61K9/00 , A61K38/17
摘要： The invention provides nucleic acids and nucleic acid expression vectors containing optimized mGluR6 promoters for expression of transgenes in the retina. The compositions and methods of the invention are useful for expression of gene products to preserve, improve, or restore phototransduction or vision.

9. 发明授权

US09968689B2 AAV-mediated subcellular targeting of heterologous rhodopsins in retinal ganglion cells 有权
公开(公告)号：US09968689B2
公开(公告)日：2018-05-15
申请号：US15236152
申请日：2016-08-12
申请人： Wayne State University
发明人： Zhuo-Hua Pan
IPC分类号： A61K48/00 , C07K14/47 , C07K14/705 , C12N15/861 , A61K38/00
CPC分类号： A61K48/005 , A61K38/00 , C07K14/4702 , C07K14/705 , C07K2319/01 , C12N15/8616 , C12N2750/14143
摘要： Microbial type rhodopsins, such as the light-gated cation-selective membrane channel, channelrhodopsin-2 (Chop2/ChR2) or the ion pump halorhodopsin (HaloR) are expressed in retinal ganglion cells upon transduction using recombinant AAV vectors. Selective targeting of these transgenes for expression in discrete subcellular regions or sites is achieved by including a sorting motif in the vector that can target either the central area or surround (off-center) area of these cells. Nucleic acid molecules comprising nucleotide sequences encoding such rhodopsins and sorting motifs and their use in methods of differential expression of the transgene are disclosed. These compositions and methods provide significant improvements for restoring visual perception and various aspects of vision, particular in patients with retinal disease.

10. 发明申请

US20170021038A1 A MODIFIED MGLUR6 PROMOTER AND METHODS OF USE 审中-公开
标题翻译：改良MGLUR6促进剂及其使用方法
公开(公告)号：US20170021038A1
公开(公告)日：2017-01-26
申请号：US15125066
申请日：2015-03-11
申请人： Wayne State University
发明人： Zhuo-Hua Pan , Qi Lu , Tushar H. Ganjawala , JrGang Cheng
IPC分类号： A61K48/00 , C12N15/86 , A61K38/17 , A61K9/00 , C07K14/705
CPC分类号： A61K48/0058 , A61K9/0048 , A61K38/1787 , A61K48/0075 , C07K14/70571 , C12N15/67 , C12N15/85 , C12N15/86 , C12N2750/14143 , C12N2830/008 , C12N2830/42
摘要： The invention provides nucleic acids and nucleic acid expression vectors containing optimized mGluR6 promoters for expression of transgenes in the retina. The compositions and methods of the invention are useful for expression of gene products to preserve, improve, or restore phototransduction or vision.
摘要翻译：本发明提供含有优化的mGluR6启动子的核酸和核酸表达载体，用于在视网膜中表达转基因。本发明的组合物和方法可用于表达基因产物以保存，改善或恢复光转导或视力。

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