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公开(公告)号:US20080152661A1
公开(公告)日:2008-06-26
申请号:US11840468
申请日:2007-08-17
申请人: David B. Rozema , Darren H. Wakefield , Jason Klein , So Wong , Jon A. Wolff , David L. Lewis , James E. Hagstrom , Andrei Blokhin , Vladimir Trubetskoy , Hans Herweijer
发明人: David B. Rozema , Darren H. Wakefield , Jason Klein , So Wong , Jon A. Wolff , David L. Lewis , James E. Hagstrom , Andrei Blokhin , Vladimir Trubetskoy , Hans Herweijer
IPC分类号: A61K31/70 , A61K39/395 , C12N5/00 , A61K38/00
CPC分类号: C12N15/87 , A01K2217/058 , A01K2267/0362 , A61K47/58 , A61K48/0041
摘要: The present invention is directed to compounds, compositions, and methods useful for delivering polynucleotides or other cell-impermeable molecules to mammalian cells. Described are polyconjugates systems that incorporate targeting, anti-opsonization, anti-aggregation, and transfection activities into small biocompatible in vivo delivery vehicles. The use of multiple reversible linkages connecting component parts provides for physiologically responsive activity modulation.
摘要翻译: 本发明涉及可用于将多核苷酸或其它细胞不可渗透分子递送至哺乳动物细胞的化合物,组合物和方法。 描述了将靶向,抗调理作用,抗聚集和转染活性结合到小的生物相容性体内递送载体中的多偶联体系。 连接组成部分的多个可逆连接的使用提供了生理响应活性调节。
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公开(公告)号:US07470539B2
公开(公告)日:2008-12-30
申请号:US11256322
申请日:2005-10-21
申请人: Darren H. Wakefield , David B. Rozema , Jon A. Wolff , Vladimir Trubetskoy , James E. Hagstrom , Vladimir G. Budker , Jason Klein , So Wong
发明人: Darren H. Wakefield , David B. Rozema , Jon A. Wolff , Vladimir Trubetskoy , James E. Hagstrom , Vladimir G. Budker , Jason Klein , So Wong
IPC分类号: C12N15/88
CPC分类号: C12N15/87 , A61K47/6455 , C12N15/88
摘要: Polyampholyte are able to condense nucleic acid to form small complexes which can be utilized in the delivery of nucleic acid to mammalian cells. The polyampholytes can be formed prior to interaction with nucleic acid or they can be formed in the presence of nucleic acid. Stabilized polycation/nucleic acid complexes can be modified to reduce the positive charge of the polycation and add targeting ligands without destabilizing the complex. The resultant particles retain their small size and are more effective in delivery of nucleic acid to cells in vivo.
摘要翻译: 聚阴离子能够冷凝核酸以形成可用于将核酸递送至哺乳动物细胞的小复合物。 聚阴离子可以在与核酸相互作用之前形成,或者可以在核酸存在下形成。 可以修饰稳定的聚阳离子/核酸复合物以减少聚阳离子的正电荷并添加靶向配体而不使复合物不稳定。 所得颗粒保留其小尺寸并且在体内将核酸递送至细胞更有效。
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公开(公告)号:US07094605B2
公开(公告)日:2006-08-22
申请号:US11046598
申请日:2005-01-28
申请人: Darren H. Wakefield , David B. Rozema , Jon A. Wolff , Vladimir Trubetskoy , James E. Hagstrom , Vladimir G. Budker , Jason Klein , So Wong
发明人: Darren H. Wakefield , David B. Rozema , Jon A. Wolff , Vladimir Trubetskoy , James E. Hagstrom , Vladimir G. Budker , Jason Klein , So Wong
IPC分类号: C12N15/88
CPC分类号: C12N15/87 , A61K47/6455 , C12N15/88
摘要: Polyampholyte are able to condense nucleic acid to form small complexes which can be utilized in the delivery of nucleic acid to mammalian cells. The polyampholytes can be formed prior to interaction with nucleic acid or they can be formed in the presence of nucleic acid. Stabilized polycation/nucleic acid complexes can be modified to reduce the positive charge of the polycation and add targeting ligands without destabilizing the complex. The resultant particles retain their small size and are more effective in delivery of nucleic acid to cells in vivo.
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公开(公告)号:US08137695B2
公开(公告)日:2012-03-20
申请号:US11840468
申请日:2007-08-17
申请人: David B. Rozema , James E. Hagstrom , Jason Klein , David L. Lewis , Sean D. Monahan , Darren H. Wakefield , Jon A. Wolff , So Wong
发明人: David B. Rozema , James E. Hagstrom , Jason Klein , David L. Lewis , Sean D. Monahan , Darren H. Wakefield , Jon A. Wolff , So Wong
CPC分类号: C12N15/87 , A01K2217/058 , A01K2267/0362 , A61K47/58 , A61K48/0041
摘要: The present invention is directed to compounds, compositions, and methods useful for delivering polynucleotides or other cell-impermeable molecules to mammalian cells. Described are polyconjugates systems that incorporate targeting, anti-opsonization, anti-aggregation, and transfection activities into small biocompatible in vivo delivery vehicles. The use of multiple reversible linkages connecting component parts provides for physiologically responsive activity modulation.
摘要翻译: 本发明涉及可用于将多核苷酸或其它细胞不可渗透分子递送至哺乳动物细胞的化合物,组合物和方法。 描述了将靶向,抗调理作用,抗聚集和转染活性结合到小的生物相容性体内递送载体中的多偶联体系。 连接组成部分的多个可逆连接的使用提供了生理响应活性调节。
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公开(公告)号:US08658211B2
公开(公告)日:2014-02-25
申请号:US13368796
申请日:2012-02-08
申请人: David B. Rozema , Darren H. Wakefield , David L. Lewis , Jason Klein , So Wong , Jon A. Wolff , James E. Hagstrom
发明人: David B. Rozema , Darren H. Wakefield , David L. Lewis , Jason Klein , So Wong , Jon A. Wolff , James E. Hagstrom
IPC分类号: A61K9/14 , C07D207/444 , C07H21/02 , C12N15/00 , C12N15/11
CPC分类号: C12N15/87 , A01K2217/058 , A01K2267/0362 , A61K47/58 , A61K48/0041 , C12N15/111 , C12N2310/14 , C12N2310/351 , C12N2320/32 , C12N2800/95 , C12N2810/10
摘要: The present invention is directed to compounds, compositions, and methods useful for delivering polynucleotides or other cell-impermeable molecules to mammalian cells. Described are polyconjugates systems that incorporate targeting, anti-opsonization, anti-aggregation, and transfection activities into small biocompatible in vivo delivery vehicles. The use of multiple reversible or labile linkages connecting component parts provides for physiologically responsive activity modulation.
摘要翻译: 本发明涉及可用于将多核苷酸或其它细胞不可渗透分子递送至哺乳动物细胞的化合物,组合物和方法。 描述了将靶向,抗调理作用,抗聚集和转染活性结合到小的生物相容性体内递送载体中的多偶联体系。 使用连接组成部分的多个可逆或不稳定的连接提供了生理响应活性调节。
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公开(公告)号:US07501394B2
公开(公告)日:2009-03-10
申请号:US11337121
申请日:2006-01-20
申请人: Alexander V. Sokoloff , So Wong , Jon A. Wolff , Sean D. Monahan , James Ludtke , Lori Higgs , Darren H. Wakefield , Magdolna G. Sebestyén
发明人: Alexander V. Sokoloff , So Wong , Jon A. Wolff , Sean D. Monahan , James Ludtke , Lori Higgs , Darren H. Wakefield , Magdolna G. Sebestyén
IPC分类号: A61K38/00
CPC分类号: C07K14/005 , A61K41/00 , C12N2795/10222
摘要: We describe compounds that bind to and are internalized by hepatocytes. Association of these compounds to other molecules or complexes can be used to target the molecules or complexes to hepatocytes in vivo or in vitro.
摘要翻译: 我们描述与肝细胞结合并被内化的化合物。 这些化合物与其他分子或复合物的结合可用于在体内或体外靶向肝细胞的分子或复合物。
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公开(公告)号:US20050170505A1
公开(公告)日:2005-08-04
申请号:US11046598
申请日:2005-01-28
申请人: Darren Wakefield , David Rozema , Jon Wolff , Vladimir Trubetskoy , James Hagstrom , Vladimir Budker , Jason Klein , So Wong
发明人: Darren Wakefield , David Rozema , Jon Wolff , Vladimir Trubetskoy , James Hagstrom , Vladimir Budker , Jason Klein , So Wong
CPC分类号: C12N15/87 , A61K47/6455 , C12N15/88
摘要: Polyampholyte are able to condense nucleic acid to form small complexes which can be utilized in the delivery of nucleic acid to mammalian cells. The polyampholytes can be formed prior to interaction with nucleic acid or they can be formed in the presence of nucleic acid. Stabilized polycation/nucleic acid complexes can be modified to reduce the positive charge of the polycation and add targeting ligands without destabilizing the complex. The resultant particles retain their small size and are more effective in delivery of nucleic acid to cells in vivo.
摘要翻译: 聚阴离子能够冷凝核酸以形成可用于将核酸递送至哺乳动物细胞的小复合物。 聚阴离子可以在与核酸相互作用之前形成,或者可以在核酸存在下形成。 可以修饰稳定的聚阳离子/核酸复合物以减少聚阳离子的正电荷并添加靶向配体而不使复合物不稳定。 所得颗粒保留其小尺寸并且在体内将核酸递送至细胞更有效。
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公开(公告)号:US20060063257A1
公开(公告)日:2006-03-23
申请号:US11256322
申请日:2005-10-21
申请人: Darren Wakefield , David Rozema , Jon Wolff , Vladimir Trubetskoy , James Hagstrom , Vladimir Budker , Jason Klein , So Wong
发明人: Darren Wakefield , David Rozema , Jon Wolff , Vladimir Trubetskoy , James Hagstrom , Vladimir Budker , Jason Klein , So Wong
IPC分类号: C12N15/87
CPC分类号: C12N15/87 , A61K47/6455 , C12N15/88
摘要: Polyampholyte are able to condense nucleic acid to form small complexes which can be utilized in the delivery of nucleic acid to mammalian cells. The polyampholytes can be formed prior to interaction with nucleic acid or they can be formed in the presence of nucleic acid. Stabilized polycation/nucleic acid complexes can be modified to reduce the positive charge of the polycation and add targeting ligands without destabilizing the complex. The resultant particles retain their small size and are more effective in delivery of nucleic acid to cells in vivo.
摘要翻译: 聚阴离子能够冷凝核酸以形成可用于将核酸递送至哺乳动物细胞的小复合物。 聚阴离子可以在与核酸相互作用之前形成,或者可以在核酸存在下形成。 可以修饰稳定的聚阳离子/核酸复合物以减少聚阳离子的正电荷并添加靶向配体而不使复合物失稳。 所得颗粒保留其小尺寸并且在体内将核酸递送至细胞更有效。
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公开(公告)号:US07071163B2
公开(公告)日:2006-07-04
申请号:US10633808
申请日:2003-08-04
申请人: Alexander V. Sokoloff , So Wong , Jon A. Wolff , Sean D. Monahan , James Ludtke , Lori Higgs , Darren Wakefield , Magdolna G. Sebestyén
发明人: Alexander V. Sokoloff , So Wong , Jon A. Wolff , Sean D. Monahan , James Ludtke , Lori Higgs , Darren Wakefield , Magdolna G. Sebestyén
IPC分类号: A61K38/00
CPC分类号: C07K14/005 , A61K41/00 , C12N2795/10222
摘要: We describe compounds that bind to and are internalized by hepatocytes. Association of these compounds to other molecules or complexes can be used to target the molecules or complexes to hepatocytes in vivo or in vitro.
摘要翻译: 我们描述与肝细胞结合并被内化的化合物。 这些化合物与其他分子或复合物的结合可用于在体内或体外靶向肝细胞的分子或复合物。
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公开(公告)号:US20060154867A1
公开(公告)日:2006-07-13
申请号:US11337121
申请日:2006-01-20
申请人: Alexander Sokoloff , So Wong , Jon Wolff , Sean Monahan , James Ludtke , Lori Higgs , Darren Wakefield , Magdolna Sebestyen
发明人: Alexander Sokoloff , So Wong , Jon Wolff , Sean Monahan , James Ludtke , Lori Higgs , Darren Wakefield , Magdolna Sebestyen
IPC分类号: A61K48/00 , A61K38/21 , A61K31/4188 , A61K9/127
CPC分类号: C07K14/005 , A61K41/00 , C12N2795/10222
摘要: We describe compounds that bind to and are internalized by hepatocytes. Association of these compounds to other molecules or complexes can be used to target the molecules or complexes to hepatocytes in vivo or in vitro.
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