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公开(公告)号：US06620617B2
公开(公告)日：2003-09-16
申请号：US09815807
申请日：2001-03-23
IPC分类号： C12N1500
CPC分类号： A61K9/1647 , A61K9/1272 , A61K9/2027 , A61K48/00
摘要： A means for obtaining efficient introduction of exogenous genes into a patient, with long term expression of the gene, is disclosed. The gene, under control of an appropriate promoter for expression in a particular cell type, is encapsulated or dispersed with a biocompatible, preferably biodegradable polymeric matrix, where the gene is able to diffuse out of the matrix over an extended period of time, for example, a period of three to twelve months or longer. The matrix is preferably in the form of a microparticle such as a microsphere (where the gene is dispersed throughout a solid polymeric matrix) or microcapsule (gene is stored in the core of a polymeric shell), a film, an implant, or a coating on a device such as a stent. The size and composition of the polymeric device is selected to result in favorable release kinetics in tissue. The size is also selected according to the method of delivery which is to be used, typically injection or administration of a suspension by aerosol into the nasal and/or pulmonary areas. The matrix composition can be selected to not only have favorable degradation rates, but to be formed of a material which is bioadhesive, to further increase the effectiveness of transfer when administered to a mucosal surface.
摘要翻译： 公开了一种用于获得有效引入外源基因到患者中的方法，其具有该基因的长期表达。 在特定细胞类型中用于表达的适当启动子的控制下的基因用生物相容的，优选可生物降解的聚合物基质包封或分散，其中基因能够在延长的时间段内扩散出基质，例如 ，期限为三至十二个月或更长。 基质优选为微粒，例如微球（其中基因分散在整个固体聚合物基质中）或微胶囊（基因存储在聚合物壳的核心中），膜，植入物或涂层 在诸如支架的装置上。 选择聚合物装置的尺寸和组成以在组织中产生有利的释放动力学。 还根据要使用的递送方法选择大小，通常通过气雾剂将悬浮液注射或施用于鼻和/或肺部区域。 可以选择基质组合物不仅具有良好的降解速率，而且由生物粘附的材料形成，以进一步提高施用于粘膜表面时转移的有效性。
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公开(公告)号：US06475779B2
公开(公告)日：2002-11-05
申请号：US09173205
申请日：1998-10-15
IPC分类号： C12N1563
CPC分类号： A61K9/1647 , A61K9/1272 , A61K9/2027 , A61K48/00
摘要： A means for obtaining efficient introduction of exogenous genes into a patient, with long term expression of the gene, is disclosed. The gene, under control of an appropriate promoter for expression in a particular cell type, is encapsulated or dispersed with a biocompatible, preferably biodegradable polymeric matrix, where the gene is able to diffuse out of the matrix over an extended period of time, for example, a period of three to twelve months or longer. The matrix is preferably in the form of a microparticle such as a microsphere (where the gene is dispersed throughout a solid polymeric matrix) or microcapsule (gene is stored in the core of a polymeric shell), a film, an implant, or a coating on a device such as a stent. The size and composition of the polymeric device is selected to result in favorable release kinetics in tissue. The size is also selected according to the method of delivery which is to be used, typically injection or administration of a suspension by aerosol into the nasal and/or pulmonary areas. The matrix composition can be selected to not only have favorable degradation rates, but to be formed of a material which is bioadhesive, to further increase the effectiveness of transfer when administered to a mucosal surface.
摘要翻译： 公开了一种用于获得有效引入外源基因到患者中的方法，其具有该基因的长期表达。 在特定细胞类型中用于表达的适当启动子的控制下的基因用生物相容的，优选可生物降解的聚合物基质包封或分散，其中基因能够在延长的时间段内扩散出基质，例如 ，期限为三至十二个月或更长。 基质优选为微粒，例如微球（其中基因分散在整个固体聚合物基质中）或微胶囊（基因存储在聚合物壳的核心中），膜，植入物或涂层 在诸如支架的装置上。 选择聚合物装置的尺寸和组成以在组织中产生有利的释放动力学。 还根据要使用的递送方法选择大小，通常通过气雾剂将悬浮液注射或施用于鼻和/或肺部区域。 可以选择基质组合物不仅具有良好的降解速率，而且由生物粘附的材料形成，以进一步提高施用于粘膜表面时转移的有效性。
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公开(公告)号：US06262034B1
公开(公告)日：2001-07-17
申请号：US08978522
申请日：1997-11-25
IPC分类号： A61K4800
CPC分类号： A61K9/1647 , A61K9/1272 , A61K9/2027 , A61K48/00
摘要： A means for obtaining efficient introduction of exogenous genes into a patient, with long term expression of the gene, is disclosed. The gene, under control of an appropriate promoter for expression in a particular cell type, is encapsulated or dispersed with a biocompatible, preferably biodegradable polymeric matrix, where the gene is able to diffuse out of the matrix over an extended period of time, for example, a period of three to twelve months or longer. The matrix is preferably in the form of a microparticle such as a microsphere (where the gene is dispersed throughout a solid polymeric matrix) or microcapsule (gene is stored in the core of a polymeric shell), a film, an implant, or a coating on a device such as a stent. The size and composition of the polymeric device is selected to result in favorable release kinetics in tissue. The size is also selected according to the method of delivery which is to be used, typically injection or administration of a suspension by aerosol into the nasal and/or pulmonary areas. The matrix composition can be selected to not only have favorable degradation rates, but to be formed of a material which is bioadhesive, to further increase the effectiveness of transfer when administered to a mucosal surface.
摘要翻译： 公开了一种用于获得有效引入外源基因到患者中的方法，其具有该基因的长期表达。 在特定细胞类型中用于表达的适当启动子的控制下的基因用生物相容的，优选可生物降解的聚合物基质包封或分散，其中基因能够在延长的时间段内扩散出基质，例如 ，期限为三至十二个月或更长。 基质优选为微粒，例如微球（其中基因分散在整个固体聚合物基质中）或微胶囊（基因存储在聚合物壳的核心中），膜，植入物或涂层 在诸如支架的装置上。 选择聚合物装置的尺寸和组成以在组织中产生有利的释放动力学。 还根据要使用的递送方法选择大小，通常通过气雾剂将悬浮液注射或施用于鼻和/或肺部区域。 可以选择基质组合物不仅具有良好的降解速率，而且由生物粘附的材料形成，以进一步提高施用于粘膜表面时转移的有效性。
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