Invention Grant
US08420619B2 Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein
失效
通过玻璃体内施用编码治疗性蛋白质的表达载体拯救光感受器
- Patent Title: Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein
- Patent Title (中): 通过玻璃体内施用编码治疗性蛋白质的表达载体拯救光感受器
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Application No.: US13212078Application Date: 2011-08-17
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Publication No.: US08420619B2Publication Date: 2013-04-16
- Inventor: Raymond T. Bartus , Kathie M. Bishop , Mehdi Gasmi
- Applicant: Raymond T. Bartus , Kathie M. Bishop , Mehdi Gasmi
- Applicant Address: US CA San Diego
- Assignee: Ceregene, Inc.
- Current Assignee: Ceregene, Inc.
- Current Assignee Address: US CA San Diego
- Agency: DLA Piper LLP (US)
- Main IPC: A61K48/00
- IPC: A61K48/00
Abstract:
The invention provides methods for treating ocular diseases using a recombinant vehicle to express a protein useful in the treatment of ocular disease, with particular preference for use of neurotrophin-4 (NT4) for targeting subpopulations of cells in the retina. A genetically engineered gene transfer vector containing sequences encoding a growth factor such as neurotrophin-4 (NT4) is used to transduce cells of the retinal ganglion cell (RGC) layer, in situ, via administration of the vector intravitreally. Accordingly, methods are disclosed for treating subjects in need thereof by therapeutic protein delivery via a recombinant expression vector, including rescue of photoreceptors by targeting the RGC layer subpopulation of retinal cells.
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