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    BIOMARKERS FOR CIRCULATING TUMOR CELLS
    1.
    发明申请
    BIOMARKERS FOR CIRCULATING TUMOR CELLS 审中-公开
    用于循环肿瘤细胞的生物标志物

    公开(公告)号：WO2011093927A1

    公开(公告)日：2011-08-04

    申请号：PCT/US2010/050223

    申请日：2010-09-24

    Applicant: DUKE UNIVERSITY ,  GARCIA-BLANCO, Mariano, A. ,  ARMSTRONG, Andrew, J. ,  GEORGE, Daniel, J. ,  OLTEAN, Sebastian

    Inventor： GARCIA-BLANCO, Mariano, A. ,  ARMSTRONG, Andrew, J. ,  GEORGE, Daniel, J. ,  OLTEAN, Sebastian

    IPC: G01N33/53

    CPC classification number: G01N33/6893 ,  G01N33/57484 ,  G01N2800/56

    Abstract: Provided are methods for detecting circulating tumor cells (CTCs) in a subject. The methods may include detecting the expression of at least one epithelial mesenchymal transition (EMT) biomarker. Further provided are kits for detecting CTCs. The kits may include antibodies to at least one EMT biomarker. Further provided are methods of predicting the responsiveness of a subject to a cancer drug, methods of targeting delivery of a cancer drug in a subject, methods of providing a cancer prognosis to a subject, and methods for following the progress of cancer in a subject.

    Abstract translation: 提供了用于检测受试者中循环肿瘤细胞（CTC）的方法。 所述方法可以包括检测至少一种上皮间质转化（EMT）生物标志物的表达。 还提供了用于检测四氯化碳的试剂盒。 试剂盒可以包括至少一种EMT生物标志物的抗体。 进一步提供了预测受试者对癌症药物的反应性的方法，靶向受试者中癌症药物递送的方法，对受试者提供癌症预后的方法，以及用于跟踪受试者的癌症进展的方法。

    TARGETED TRANS-SPLICING OF HIGHLY ABUNDANT TRANSCRIPTS FOR IN VIVO PRODUCTION OF RECOMBINANT PROTEINS
    2.
    发明申请
    TARGETED TRANS-SPLICING OF HIGHLY ABUNDANT TRANSCRIPTS FOR IN VIVO PRODUCTION OF RECOMBINANT PROTEINS 审中-公开
    用于生殖重组蛋白的高度突变转录物的目标转位分离

    公开(公告)号：WO2006042232A2

    公开(公告)日：2006-04-20

    申请号：PCT/US2005036424

    申请日：2005-10-07

    Applicant: INTRONN INC ,  MCGARRITY GERARD J ,  GARCIA-BLANCO MARIANO A

    Inventor： MCGARRITY GERARD J ,  GARCIA-BLANCO MARIANO A

    CPC classification number: C12P21/02 ,  C07K16/084 ,  C07K2317/622 ,  C12N15/1027

    Abstract: The present invention provides methods and compositions for generating novel nucleic acid molecules through RNA trans-splicing that target a highly expressed pre-mRNA and contain the coding sequence of a protein or polypeptide of interest. The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with the target precursor messenger RNA molecule (target pre-mRNA) that is abundantly expressed, and mediate a trans-splicing reaction resulting in the generation of novel chimeric RNA molecule (chimeric RNA) capable of encoding a protein or polypeptide of interest. The invention provides for the

    Abstract translation: 本发明提供了通过RNA转录产生新型核酸分子的方法和组合物，其靶向高度表达的前mRNA并含有感兴趣的蛋白质或多肽的编码序列。 本发明的组合物包括设计成与大量表达的靶前体信使RNA分子（靶前体mRNA）相互作用的转录前分子（PTM），并介导转拼反应，从而产生新的嵌合 能够编码目标蛋白质或多肽的RNA分子（嵌合RNA）。 本发明提供了

    METHODS AND COMPOSITIONS FOR USE IN SPLICEOSOME MEDIATED RNA TRANS-SPLICING
    3.
    发明申请
    METHODS AND COMPOSITIONS FOR USE IN SPLICEOSOME MEDIATED RNA TRANS-SPLICING 审中-公开
    用于SPLICEOSOME介导的RNA转移分离的方法和组合物

    公开(公告)号：WO2003069311A2

    公开(公告)日：2003-08-21

    申请号：PCT/US2003/004622

    申请日：2003-02-12

    Applicant: INTRONN, INC. ,  MITCHELL, Lloyd, G. ,  GARCIA-BLANCO, Mariano, A. ,  PUTTARAJU, Madaiah

    Inventor： MITCHELL, Lloyd, G. ,  GARCIA-BLANCO, Mariano, A. ,  PUTTARAJU, Madaiah

    IPC: G01N

    CPC classification number: C12N15/113 ,  A61K48/00

    Abstract: The present invention provides methods and compositions for delivery of synthetic pre- trans -splicing molecules (synthetic PTMs) into a target cell. The compositions of the invention include synthetic pre- trans -splicing molecules (PTMs) with enhanced stability against chemical and enzymatic degradation. The synthetic PTMs are designed to interact with a natural target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans- splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA).

    Abstract translation: 本发明提供了将合成的预转录分子（合成PTM）递送到靶细胞中的方法和组合物。 本发明的组合物包括具有增强的针对化学和酶降解的稳定性的合成的预转录分子（PTM）。 合成的PTM被设计为与天然靶标前体信使RNA分子（靶前mRNA）相互作用并介导导致产生新的嵌合RNA分子（嵌合RNA）的转拼反应。

    CORRECTION OF ALPHA-1-ANTITRYPSIN GENETIC DEFECTS USING SPLICEOSOME MEDIATED RNA TRANS SPLICING
    5.
    发明申请
    CORRECTION OF ALPHA-1-ANTITRYPSIN GENETIC DEFECTS USING SPLICEOSOME MEDIATED RNA TRANS SPLICING 审中-公开
    使用SPLICEOSOME介导的RNA转移分离的ALPHA-1-抗体基因缺陷的修正

    公开(公告)号：WO2005070948A1

    公开(公告)日：2005-08-04

    申请号：PCT/US2005/002549

    申请日：2005-01-21

    Applicant: INTRONN, INC. ,  PUTTARAJU, Madaiah ,  OTTO, Edward ,  GARCIA-BLANCO, Mariano, A. ,  MCGARRITY, Gerard, J. ,  TEMPLE, Gary, F. ,  COTE, Collette ,  MITCHELL, Lloyd, G. ,  MANSFIELD, Gary, S.

    Inventor： PUTTARAJU, Madaiah ,  OTTO, Edward ,  GARCIA-BLANCO, Mariano, A. ,  MCGARRITY, Gerard, J. ,  TEMPLE, Gary, F. ,  COTE, Collette ,  MITCHELL, Lloyd, G. ,  MANSFIELD, Gary, S.

    IPC: C07H21/02

    CPC classification number: C12N15/10 ,  C12N15/102 ,  C12P19/34

    Abstract: The present invention provides methods and compositions for generating novel nucleic acid molecules through targeted spliceosomal mediated RNA trans -splicing. The compositions of the invention include pre-trans -splicing molecules (PTMs) designed to interact with a SERPINAI target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans -splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA). In particular, the PTMs of the present invention include those genetically engineered to interact with SERPINA1 target pre-mRNA so as to result in correction of SERPINAI genetic defects responsible for AAT deficiency. The PTMs of the invention may also comprise sequences that are processed out of the PTM to yield duplex siRNA molecules directed specifically to mutant SERPIN Al mRNAs. Such duplexed siRNAs are designed to reduce the accumulation of toxic AAT protein in liver cells. The methods and compositions of the present invention can be used in gene therapy for correction of SERPINAI disorders such as AAT deficiency.

    Abstract translation: 本发明提供了通过靶向剪接体介导的RNA转接产生新的核酸分子的方法和组合物。 本发明的组合物包括设计为与SERPINAI靶前体信使RNA分子（靶前体mRNA）相互作用的转录前分子（PTM），并介导导致产生新型嵌合RNA分子的反式剪接反应（ 嵌合RNA）。 特别地，本发明的PTM包括经遗传工程改造以与SERPINA1靶前体mRNA相互作用，从而导致对负责AAT缺陷的SERPINAI遗传缺陷的校正。 本发明的PTM还可以包含从PTM中加工得到的特异性引导突变体SERPIN A1 mRNA的双链体siRNA分子的序列。 这样的双链体siRNA被设计为减少肝细胞中有毒的AAT蛋白的积累。 本发明的方法和组合物可用于基因治疗以纠正SERPINAI病症如AAT缺陷。

    EXPRESSION OF APOA-1 AND VARIANTS THEREOF USING SPLICEOSOME MEDIATED RNA TRANS-SPLICING
    6.
    发明申请
    EXPRESSION OF APOA-1 AND VARIANTS THEREOF USING SPLICEOSOME MEDIATED RNA TRANS-SPLICING 审中-公开
    APOA-1的表达及其使用SPLICEOSOME介导的RNA转移分离的变体

    公开(公告)号：WO2005070023A2

    公开(公告)日：2005-08-04

    申请号：PCT/US2005002392

    申请日：2005-01-21

    Applicant: INTRONN INC ,  PUTTARAJU MADAIAH ,  OTTO EDWARD ,  GARCIA-BLANCO MARIANO A ,  MCGARRITY GERARD J ,  TEMPLE GARY F ,  MITCHELL LLOYD G

    Inventor： PUTTARAJU MADAIAH ,  OTTO EDWARD ,  GARCIA-BLANCO MARIANO A ,  MCGARRITY GERARD J ,  TEMPLE GARY F ,  MITCHELL LLOYD G

    IPC: C07K14/775 ,  C12N15/11 ,  C12N15/113

    CPC classification number: C07K14/775 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/3519 ,  C12N2320/33

    Abstract: The present invention provides methods and compositions for generating novel nucleic acid molecules through targeted spliceosome mediated RNA trans-splicing that result in expression of an apoA-1 variant, the preferred embodiment referred to herein as the apoA-1 Milano variant. The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans­-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA) capable of encoding the apoA-1 Milano variant. The expression of this variant protein results in protection against vascular disorders resulting from plaque build up, i.e., strokes and heart attacks. In particular, the PTMs of the presént invention include those genetically engineered to interact with the apoA-1 target pre­mRNA so as to result in expression of the apoA-1 Milano variant. In addition, the PTMs of the invention include those genetically engineered to interact with the apoB or albumin or other specific target pre-mRNAs so as to result in expression of an apoB/apoA-1 and/or alb/apoA-1 wild type or Milano fusion protein thereby reducing apoB expression and simultaneously produce ApoA-1 function.

    Abstract translation: 本发明提供了通过靶向剪接体介导的RNA转接产生新的核酸分子的方法和组合物，其导致apoA-1变体（本文中称为apoA-1 Milano变体）的优选实施方案的表达。 本发明的组合物包括被设计成与靶前体信使RNA分子（靶前mRNA）相互作用并且介导导致产生新的嵌合RNA分子（嵌合体）的反式剪接反应的预转录分子（PTM） RNA）能够编码apoA-1 Milano变体。 该变体蛋白质的表达导致保护免受斑块积聚引起的血管疾病，即中风和心脏病发作。 特别地，预发明的PTM包括经遗传工程改造以与apoA-1靶preRNA相互作用以产生载脂蛋白-A 1米兰变体的表达的那些。 此外，本发明的PTM包括经遗传工程改造以与载脂蛋白B或白蛋白或其它特异性靶前体mRNA相互作用以产生apoB / apoA-1和/或alb / apoA-1野生型或 米诺融合蛋白从而降低apoB表达并同时产生ApoA-1功能。

    SPLICEOSOME MEDIATED RNA TRANS-SPLICING AND CORRECTION OF FACTOR VIII GENETIC DEFECTS USING SPLICEOSOME MEDIATED RNA TRANS SPLING
    7.
    发明申请
    SPLICEOSOME MEDIATED RNA TRANS-SPLICING AND CORRECTION OF FACTOR VIII GENETIC DEFECTS USING SPLICEOSOME MEDIATED RNA TRANS SPLING 审中-公开
    SPLICEOSOME介导的RNA转移分离和因子VIII的遗传缺陷的修正使用SPLICEOSOME介导的RNA转移分离

    公开(公告)号：WO2003104412A2

    公开(公告)日：2003-12-18

    申请号：PCT/US2003/017913

    申请日：2003-06-05

    Applicant: INTRONN, INC. ,  MITCHELL, Lloyd, G. ,  MANSFIELD, S. Gary ,  PUTTARAJU, Madaiah ,  CLARK, Rebecca ,  GARCIA-BLANCO, Mariano, A.

    Inventor： MITCHELL, Lloyd, G. ,  MANSFIELD, S. Gary ,  PUTTARAJU, Madaiah ,  CLARK, Rebecca ,  GARCIA-BLANCO, Mariano, A.

    IPC: C12N

    CPC classification number: C07K14/755 ,  C12N15/10

    Abstract: The compositions of the invention include pre- trans -splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans -splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA). In particular, the PTMs of the present invention are genetically engineered to interact with factor VIII (FVIII) target pre-mRNA so as to result in correction of clotting FVIII genetic defects responsible for hemophilia A. The compositions of the invention further include recombinant vector systems capable of expressing the PTMs of the invention and cells expressing said PTMs. The methods of the invention encompass contacting the PTMs of the invention with a FVIII target pre-mRNA under conditions in which a portion of the PTM is trans -spliced to a portion of the target pre-mRNA to form a RNA molecule wherein the genetic defect in the FVIII gene has been corrected. The methods and compositions of the present invention can be used in gene therapy for correction of FVIII disorders such as hemophilia A.

    Abstract translation: 本发明的组合物包括被设计为与靶前体信使RNA分子（靶前mRNA）相互作用并且介导导致产生新的嵌合RNA分子（嵌合体）的反式剪接反应的预转录分子（PTM） RNA）。 特别地，本发明的PTM被遗传工程化以与因子VIII（FVIII）靶前mRNA相互作用，从而导致血友病A引起的凝血FVIII遗传缺陷的校正。本发明的组合物还包括重组载体系统 能够表达本发明的PTM和表达所述PTM的细胞。 本发明的方法包括将本发明的PTM与FVIII靶前体mRNA接触，其中将部分PTM转染到靶前mRNA的一部分以形成RNA分子的条件下，其中遗传缺陷 在FVIII基因中已被纠正。 本发明的方法和组合物可用于基因治疗中用于校正FVIII病症如血友病A.

    SPLICEOSOME MEDIATED RNA TRANS-SPLICING AND CORRECTION OF FACTOR VIII GENETIC DEFECTS
    9.
    发明申请
    SPLICEOSOME MEDIATED RNA TRANS-SPLICING AND CORRECTION OF FACTOR VIII GENETIC DEFECTS 审中-公开

    公开(公告)号：WO2003104412A3

    公开(公告)日：2003-12-18

    申请号：PCT/US2003/017913

    申请日：2003-06-05

    Applicant: INTRONN, INC. ,  MITCHELL, Lloyd, G. ,  MANSFIELD, S. Gary ,  PUTTARAJU, Madaiah ,  CLARK, Rebecca ,  GARCIA-BLANCO, Mariano, A.

    Inventor： MITCHELL, Lloyd, G. ,  MANSFIELD, S. Gary ,  PUTTARAJU, Madaiah ,  CLARK, Rebecca ,  GARCIA-BLANCO, Mariano, A.

    IPC: C07H21/02

    Abstract: The compositions of the invention include pre- trans -splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans -splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA). In particular, the PTMs of the present invention are genetically engineered to interact with factor VIII (FVIII) target pre-mRNA so as to result in correction of clotting FVIII genetic defects responsible for hemophilia A. The compositions of the invention further include recombinant vector systems capable of expressing the PTMs of the invention and cells expressing said PTMs. The methods of the invention encompass contacting the PTMs of the invention with a FVIII target pre-mRNA under conditions in which a portion of the PTM is trans -spliced to a portion of the target pre-mRNA to form a RNA molecule wherein the genetic defect in the FVIII gene has been corrected. The methods and compositions of the present invention can be used in gene therapy for correction of FVIII disorders such as hemophilia A.

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