公开(公告)号：US08728458B2

公开(公告)日：2014-05-20

申请号：US13266236

申请日：2010-04-29

申请人： Joseph Anderson ,  Gerhard Bauer ,  Jan A. Nolta

发明人： Joseph Anderson ,  Gerhard Bauer ,  Jan A. Nolta

IPC分类号： A61K48/00

CPC分类号： A61K48/0066 ,  A61K31/7105 ,  A61K38/53 ,  C07K14/7158 ,  C12N7/00 ,  C12N15/1138 ,  C12N15/86 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/531 ,  C12N2320/31 ,  C12N2320/32 ,  C12N2740/16011 ,  C12N2740/16043 ,  C12N2740/16051 ,  C12Y603/02

摘要： Recombinant lentiviral vectors containing at least: a lentiviral backbone comprising essential lentiviral sequences for integration into a target cell genome; a nucleic acid encoding a CCR5 RNAi; and an expression control element that regulates expression of the nucleic acid encoding the CCR5 RNAi element, are provided by this invention. In an alternative aspect, the vector also contains polynucleotides encoding TRIM5 alpha and HIV TAR decoy sequences along with gene expression regulation elements such as promoters operatively linked to the polynucleotides. The vectors are combined with packaging plasmid and envelope plasmids and optionally conjugated to cell-specific targeting antibodies. Diagnostic and therapeutic methods for using the compositions are further provided herein.

摘要翻译： 含有至少包含慢病毒骨架的重组慢病毒载体，其包含用于整合入靶细胞基因组的必需慢病毒序列; 编码CCR5 RNAi的核酸; 以及调节编码CCR5 RNAi元件的核酸表达的表达控制元件由本发明提供。 在另一方面，载体还含有编码TRIM5α和HIV TAR诱饵序列的多核苷酸以及基因表达调控元件，例如与多核苷酸可操作连接的启动子。 将载体与包装质粒和包膜质粒组合，并任选地与细胞特异性靶向抗体缀合。 本文进一步提供了使用组合物的诊断和治疗方法。

公开(公告)号：US20120114618A1

公开(公告)日：2012-05-10

申请号：US13260551

申请日：2010-03-25

申请人： Jan A. Nolta ,  Scott Olson ,  Louisa Wirthlin

发明人： Jan A. Nolta ,  Scott Olson ,  Louisa Wirthlin

IPC分类号： A61K35/12 ,  C12N5/10 ,  A61P25/14 ,  C12N5/0775

CPC分类号： A61K35/28 ,  A61K35/12 ,  A61K2035/124 ,  C12N5/0662 ,  C12N15/111 ,  C12N15/87 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2320/32 ,  C12N2330/51

摘要： Compositions and methods for delivering a siRNA, dsRNA, or miRNA polynucleotide into a target cell comprising contacting the target cell with a mesenchymal stem cell, which mesenchymal stem cell comprises an exogenous DNA sequence expressing the siRNA or dsRNA polynucleotide, thereby delivering the siRNA, dsRNA, or miRNA polynucleotide to the target cell through a cellular protrusion or a microvesicle.

摘要翻译： 将siRNA，dsRNA或miRNA多核苷酸递送至靶细胞的组合物和方法，包括使所述靶细胞与间充质干细胞接触，所述间充质干细胞包含表达siRNA或dsRNA多核苷酸的外源DNA序列，从而递送siRNA，dsRNA ，或miRNA多核苷酸通过细胞突起或微囊进入靶细胞。

公开(公告)号：US20120076763A1

公开(公告)日：2012-03-29

申请号：US13266236

申请日：2010-04-29

申请人： Joseph Anderson ,  Gerhard Bauer ,  Jan A. Nolta

发明人： Joseph Anderson ,  Gerhard Bauer ,  Jan A. Nolta

IPC分类号： A61K48/00 ,  C12N5/071 ,  A61P31/18 ,  C12N5/10 ,  C12Q1/70 ,  C12N15/63 ,  C12N7/01

CPC分类号： A61K48/0066 ,  A61K31/7105 ,  A61K38/53 ,  C07K14/7158 ,  C12N7/00 ,  C12N15/1138 ,  C12N15/86 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/531 ,  C12N2320/31 ,  C12N2320/32 ,  C12N2740/16011 ,  C12N2740/16043 ,  C12N2740/16051 ,  C12Y603/02

摘要： Recombinant lentiviral vectors containing at least: a lentiviral backbone comprising essential lentiviral sequences for integration into a target cell genome; a nucleic acid encoding a CCR5 RNAi; and an expression control element that regulates expression of the nucleic acid encoding the CCR5 RNAi element, are provided by this invention. In an alternative aspect, the vector also contains polynucleotides encoding TRIMS alpha and HIV TAR decoy sequences along with gene expression regulation elements such as promoters operatively linked to the polynucleotides. The vectors are combined with packaging plasmid and envelope plasmids and optionally conjugated to cell-specific targeting antibodies. Diagnostic and therapeutic methods for using the compositions are further provided herein.

摘要翻译： 含有至少包含慢病毒骨架的重组慢病毒载体，其包含用于整合入靶细胞基因组的必需慢病毒序列; 编码CCR5 RNAi的核酸; 和调节编码CCR5 RNAi元件的核酸表达的表达控制元件由本发明提供。 在替代方面，载体还含有编码TRIMSα和HIV TAR诱饵序列的多核苷酸以及基因表达调控元件，例如与多核苷酸可操作连接的启动子。 将载体与包装质粒和包膜质粒组合，并任选地与细胞特异性靶向抗体缀合。 本文进一步提供了使用组合物的诊断和治疗方法。