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1.发明授权Methods of making and using a cell penetrating peptide for enhanced delivery of nucleic acids, proteins, drugs, and adenovirus to tissues and cells, and compositions and kits 有权制备和使用细胞穿透肽以增强核酸，蛋白质，药物和腺病毒对组织和细胞的传递的方法以及组合物和试剂盒公开(公告)号：US08778886B2
公开(公告)日：2014-07-15
申请号：US12714106
申请日：2010-02-26
IPC分类号： A61K38/00 , A61K47/00 , A61K38/16 , A61K38/10 , A61K38/08 , A61K39/385 , A61K38/04 , A61M31/00 , A61P9/10 , A61P27/02 , C07K5/00 , C07K7/00 , C07K16/00 , C07K17/00
CPC分类号： A61K47/6929 , A61K47/60 , A61K47/645
摘要： A peptide-POD with ability to penetrate and deliver fluorophores, siRNA, DNA and quantum dots to cells in culture and retinal and ocular tissues in vivo is provided herein. POD couples to adenovirus vectors, enhancing tropism for certain cells, potentially providing a safer and more efficacious method to deliver molecules to ocular and other tissues in vivo. POD constructs are therapeutic delivery vehicles for treating cells and tissues, including ocular cells and tissues suffering from retinal degeneration.
摘要翻译： 本文提供了能够在体内将荧光团，siRNA，DNA和量子点传递到培养物和视网膜和眼组织中的细胞的能力的肽-POD。 POD与腺病毒载体耦合，增强某些细胞的嗜性，潜在地提供了一种更安全，更有效的方法来将分子递送到眼睛和其他组织体内。 POD构建体是用于治疗细胞和组织的治疗性递送载体，包括眼细胞和患有视网膜变性的组织。
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2.发明申请Compositions and methods for retinal transduction and photoreceptor specific transgene expression 审中-公开视网膜转导和感光细胞特异性转基因表达的组合物和方法公开(公告)号：US20100184838A1
公开(公告)日：2010-07-22
申请号：US12595651
申请日：2008-04-11
IPC分类号： A61K31/711 , C12N15/74 , A61P27/02
CPC分类号： A61K48/0075 , C07K14/47 , C07K14/4716 , C12N7/00 , C12N15/85 , C12N15/86 , C12N2710/10343 , C12N2710/10345 , C12N2800/24 , C12N2810/405 , C12N2830/008 , C12N2830/85
摘要： Adenovirus (Ad) vectors are here provided for treatment of ocular tissues as are suitable methods to transduce photoreceptor (PR) cells, the tissue associated with degeneration. Expression from CMV or chicken beta actin (CBA) promoters in neural retina were compared, and CBA was found to be 173-fold more potent than CMV. Further, the RGD domain in Ad penton was found to play a key role in RPE tropism. Deletion of the RGD domain coupled with the CBA promoter permitted transgene expression in neural retina approximately 667-fold more efficiently than with prior Ad5 vectors. Use of Ad vectors in combination with a 4.7 kb rhodopsin promoter enabled transgene expression exclusively in photoreceptor cells in vivo.
摘要翻译： 这里提供腺病毒（Ad）载体用于治疗眼组织，如转导感光器（PR）细胞，与变性相关的组织的合适方法。 比较CMV或鸡β肌动蛋白（CBA）启动子在神经视网膜中的表达，发现CBA比CMV更有效173倍。 此外，Ad penton的RGD域被发现在RPE向性中起关键作用。 与CBA启动子结合的RGD结构域的删除允许神经视网膜中的转基因表达比先前的Ad5载体高约667倍。 使用Ad载体与4.7 kb视紫红质启动子组合使得能够在体内在感光细胞中独家表达转基因表达。
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3.发明申请Methods of making and using a cell penetrating peptide for enhanced delivery of nucleic acids, proteins, drugs, and adenovirus to tissues and cells, and compositions and kits 有权制备和使用细胞穿透肽以增强核酸，蛋白质，药物和腺病毒对组织和细胞的传递的方法以及组合物和试剂盒公开(公告)号：US20100209447A1
公开(公告)日：2010-08-19
申请号：US12714106
申请日：2010-02-26
IPC分类号： A61K39/385 , A61K31/7088 , A61K31/713 , A61K38/02 , A61K31/70 , A61K38/16 , C12N5/00 , A61P27/02
CPC分类号： A61K47/6929 , A61K47/60 , A61K47/645
摘要： A peptide-POD with ability to penetrate and deliver fluorophores, siRNA, DNA and quantum dots to cells in culture and retinal and ocular tissues in vivo is provided herein. POD couples to adenovirus vectors, enhancing tropism for certain cells, potentially providing a safer and more efficacious method to deliver molecules to ocular and other tissues in vivo. POD constructs are therapeutic delivery vehicles for treating cells and tissues, including ocular cells and tissues suffering from retinal degeneration.
摘要翻译： 本文提供了能够在体内将荧光团，siRNA，DNA和量子点传递到培养物和视网膜和眼组织中的细胞的能力的肽-POD。 POD与腺病毒载体耦合，增强某些细胞的嗜性，潜在地提供了一种更安全，更有效的方法来将分子递送到眼睛和其他组织体内。 POD构建体是用于治疗细胞和组织的治疗性递送载体，包括眼细胞和患有视网膜变性的组织。
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4.发明授权Humanized model of membrane attack complex (MAC) formation on murine retina and compositions, kits and methods for treatment of macular degeneration 有权鼠视网膜上的膜攻击复合物（MAC）形成的人源化模型及其治疗黄斑变性的组合物，试剂盒和方法公开(公告)号：US08324182B2
公开(公告)日：2012-12-04
申请号：US12867566
申请日：2009-02-13
CPC分类号： A61K48/005 , A61K48/0075 , C12N2799/022 , G01N33/564 , G01N2333/70596 , G01N2800/164
摘要： Methods and compositions for treating a subject having age-related macular degeneration (AMD), methods of assaying human macular degeneration (MD), and methods and kits for assaying potential therapeutic agents for treatment of human MD are provided herein.
摘要翻译： 本文提供了用于治疗患有年龄相关性黄斑变性（AMD）的受试者的方法和组合物，测定人黄斑变性（MD）的方法，以及用于测定治疗人MD的潜在治疗剂的方法和试剂盒。
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5.发明申请humanized model of membrane attack complex (MAC) formation on murine retina and compositions, kits and methods for treatment of macular degeneration 有权鼠视网膜上的膜攻击复合物（MAC）形成的人源化模型和用于治疗黄斑变性的组合物，试剂盒和方法公开(公告)号：US20110015136A1
公开(公告)日：2011-01-20
申请号：US12867566
申请日：2009-02-13
IPC分类号： A61K38/16 , C12Q1/68 , G01N33/53 , G01N33/567 , A61K31/7088 , A61P27/02
CPC分类号： A61K48/005 , A61K48/0075 , C12N2799/022 , G01N33/564 , G01N2333/70596 , G01N2800/164
摘要： Methods and compositions for treating a subject having age-related macular degeneration (AMD), methods of assaying human macular degeneration (MD), and methods and kits for assaying potential therapeutic agents for treatment of human MD are provided herein.
摘要翻译： 本文提供了用于治疗患有年龄相关性黄斑变性（AMD）的受试者的方法和组合物，测定人黄斑变性（MD）的方法，以及用于测定治疗人MD的潜在治疗剂的方法和试剂盒。
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