TRANS-SPLICING MEDIATED PHOTODYNAMIC THERAPY
    1.
    发明申请
    TRANS-SPLICING MEDIATED PHOTODYNAMIC THERAPY 审中-公开
    转移介导的光电疗法

    公开(公告)号:US20090203143A1

    公开(公告)日:2009-08-13

    申请号:US12135247

    申请日:2008-06-09

    IPC分类号: C12N15/87 C12N15/11 C12N1/21

    摘要: The present invention provides methods and compositions for conferring selective death on cells expressing a specific target precursor messenger RNA (selective target pre-mRNA). The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) expressed within a cell and mediate a trans-splicing reaction resulting in the generation of a novel chimeric mRNA molecule (chimeric mRNA) capable of encoding a light producing protein or enzyme. Cell death is further mediated by the presence of a photosensitizer which upon photoactivation produces cytotoxicity.

    摘要翻译: 本发明提供了赋予表达特异性靶标前体信使RNA(选择性靶前体mRNA)的细胞上选择性死亡的方法和组合物。 本发明的组合物包括设计成与在细胞内表达的靶前体信使RNA分子(靶前体mRNA)相互作用的预转录分子(PTM),并介导反剪接反应,导致产生新的嵌合 能够编码产生光的蛋白质或酶的mRNA分子(嵌合mRNA)。 细胞死亡进一步由光敏剂的存在介导,光敏剂在光活化时产生细胞毒性。

    Trans-splicing mediated photodynamic therapy
    2.
    发明授权
    Trans-splicing mediated photodynamic therapy 失效
    转录介导的光动力疗法

    公开(公告)号:US07399753B2

    公开(公告)日:2008-07-15

    申请号:US10658617

    申请日:2003-09-09

    摘要: The present invention provides methods and compositions for conferring selective death on cells expressing a specific target precursor messenger RNA (selective target pre-mRNA). The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) expressed within a cell and mediate a trans-splicing reaction resulting in the generation of a novel chimeric mRNA molecule (chimeric mRNA) capable of encoding a light producing protein or enzyme. Cell death is further mediated by the presence of a photosensitizer which upon photoactivation produces cytotoxicity.

    摘要翻译: 本发明提供了赋予表达特异性靶标前体信使RNA(选择性靶前体mRNA)的细胞上选择性死亡的方法和组合物。 本发明的组合物包括设计成与在细胞内表达的靶前体信使RNA分子(靶前体mRNA)相互作用的预转录分子(PTM),并介导反剪接反应,导致产生新的嵌合 能够编码产生光的蛋白质或酶的mRNA分子(嵌合mRNA)。 细胞死亡进一步由光敏剂的存在介导,光敏剂在光活化时产生细胞毒性。

    Sequencing DNA; a modification of the polymerase chain reaction
    3.
    发明授权
    Sequencing DNA; a modification of the polymerase chain reaction 失效
    测序DNA; 聚合酶链反应的修饰

    公开(公告)号:US5817797A

    公开(公告)日:1998-10-06

    申请号:US200876

    申请日:1988-06-01

    摘要: The invention provides a process wherein a biotinylated oligonucleotide primer and an oligonucleotide primer which has not undergone biotinylation are used when amplifying a DNA sequence to facilitate separation of the DNA strands following the polymerase chain reaction process. The biotinylation/PCR product is then exposed to a support which will selectively bind the biotinylated strand to allow selective elution of the product.

    摘要翻译: 本发明提供一种方法,其中当扩增DNA序列以促进聚合酶链式反应方法后的DNA链分离时,使用未经历生物素化的生物素化寡核苷酸引物和寡核苷酸引物。 然后将生物素化/ PCR产物暴露于支持物,其将选择性结合生物素化的链,以允许产物的选择性洗脱。

    Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated RNA trans splicing
    4.
    发明授权
    Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated RNA trans splicing 有权
    使用剪接体介导的RNA反式剪接修正α-1-抗胰蛋白酶遗传缺陷

    公开(公告)号:US08053232B2

    公开(公告)日:2011-11-08

    申请号:US11040634

    申请日:2005-01-21

    摘要: 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 SERPINA1 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 SERPINA1 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 A1 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 SERPINA1 disorders such as AAT deficiency.

    摘要翻译: 本发明提供了通过靶向剪接体介导的RNA转接产生新型核酸分子的方法和组合物。 本发明的组合物包括设计为与SERPINA1靶前体信使RNA分子(靶前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SERPINA1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型SERPIN A1 mRNAs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SERPINA1病症如AAT缺陷。

    Methods and compositions for determining the purity of chemically synthesized nucleic acids
    8.
    发明授权
    Methods and compositions for determining the purity of chemically synthesized nucleic acids 有权
    用于测定化学合成核酸纯度的方法和组合物

    公开(公告)号:US07901892B2

    公开(公告)日:2011-03-08

    申请号:US10190795

    申请日:2002-07-08

    IPC分类号: G01N33/53

    摘要: This application describes an antibody that specifically binds to a synthetic oligomer (e.g., an oligonucleotide or oligopeptide) having a organic protecting group covalently bound thereto, which antibody does not bind to that synthetic oligomer when the organic protecting group is not covalently bound thereto. Methods of making and using such antibodies are also disclosed, along with cells for making such antibodies and articles carrying immobilized oligomers that can be used in assay procedures with such antibodies.

    摘要翻译: 本申请描述了与有机保护基团共价结合的合成寡聚物(例如寡核苷酸或寡肽)特异性结合的抗体,当有机保护基团不与其共价键合时,该抗体不与该合成低聚物结合。 还公开了制备和使用这些抗体的方法,以及用于制备此类抗体的细胞和携带固定化寡聚物的制品​​,其可用于具有此类抗体的测定程序。

    METHODS AND COMPOSITIONS FOR DETERMINING THE PURITY OF CHEMICALLY SYNTHESIZED NUCLEIC ACIDS
    9.
    发明申请
    METHODS AND COMPOSITIONS FOR DETERMINING THE PURITY OF CHEMICALLY SYNTHESIZED NUCLEIC ACIDS 有权
    用于测定化学合成核酸的纯度的方法和组合物

    公开(公告)号:US20120198575A1

    公开(公告)日:2012-08-02

    申请号:US13440613

    申请日:2012-04-05

    IPC分类号: C12P21/00 C40B40/06 C07H21/00

    摘要: This application describes an antibody that specifically binds to a synthetic oligomer (e.g., an oligonucleotide or oligopeptide) having a organic protecting group covalently bound thereto, which antibody does not bind to that synthetic oligomer when the organic protecting group is not covalently bound thereto. Methods of making and using such antibodies are also disclosed, along with cells for making such antibodies and articles carrying immobilized oligomers that can be used in assay procedures with such antibodies.

    摘要翻译: 本申请描述了与有机保护基团共价结合的合成寡聚物(例如寡核苷酸或寡肽)特异性结合的抗体,当有机保护基团不与其共价键合时,该抗体不与该合成低聚物结合。 还公开了制备和使用这些抗体的方法,以及用于制备此类抗体的细胞和携带固定化寡聚物的制品​​,其可用于具有此类抗体的测定程序。