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11.发明授权公开(公告)号：US06280978B1
公开(公告)日：2001-08-28
申请号：US09158863
申请日：1998-09-23
IPC分类号： C07H2102
CPC分类号： C12N15/1027 , A61K38/00 , A61K48/00 , C07K14/34 , C07K14/4712 , C07K14/59 , C12N15/10 , C12N15/113 , C12N15/63 , C12N15/66 , C12N15/85 , C12N2310/111 , C12N2310/12 , C12N2840/44 , C12N2840/445
摘要： The molecules and methods of the present invention provide a means for in vivo production of a trans-spliced molecule in a selected subset of cells. The pre-trans-splicing molecules of the invention are substrates for a trans-splicing reaction between the pre-trans-splicing molecules and a pre-mRNA which is uniquely expressed in the specific target cells. The in vivo trans-splicing reaction provides a novel mRNA which is functional as mRNA or encodes a protein to be expressed in the target cells. The expression product of the mRNA is a protein of therapeutic value to the cell or host organism a toxin which causes killing of the specific cells or a novel protein not normally present in such cells. The invention further provides PTMs that have been genetically engineered for the identification of exon/intron boundaries of pre-mRNA molecules using an exon tagging method. The PTMs of the invention can also be designed to result in the production of chimeric RNA encoding for peptide affinity purification tags which can be used to purify and identify proteins expressed in a specific cell type.
摘要翻译： 本发明的分子和方法提供了在选定的细胞亚群中体内产生转录分子的方法。 本发明的预转录分子是用于前转录分子之间的转拼反应的底物和在特异性靶细胞中唯一表达的前mRNA。 体内转拼反应提供了新的mRNA，其作为mRNA起作用，或编码要在靶细胞中表达的蛋白质。 mRNA的表达产物是对细胞或宿主生物体具有治疗价值的蛋白质，其导致杀死特定细胞的毒素或这种细胞中通常不存在的新型蛋白质。 本发明进一步提供已使用外显子标记方法遗传工程化用于鉴定前mRNA分子的外显子/内含子边界的PTM。 本发明的PTM还可以被设计成导致编码肽亲和纯化标签的嵌合RNA的产生，其可以用于纯化和鉴定以特定细胞类型表达的蛋白质。
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公开(公告)号：US08735366B2
公开(公告)日：2014-05-27
申请号：US13056449
申请日：2009-07-30
申请人： Johann Bauer , Lloyd G. Mitchell
发明人： Johann Bauer , Lloyd G. Mitchell
CPC分类号： C12N15/85 , A61K48/005 , C07K14/78 , C12N2740/13043 , C12N2840/445
摘要： The present invention relates to specific and markedly improved pre-mRNA trans-splicing molecule (RTM) molecules which are designed to correct specific genes expressed within cells to be targeted, and which are associated with epidermolysis bullosa, cystic fibrosis, pachyonychia congenital, and psoriasis or neurodermitis, as well as cancers of the skin. In particular, the RTMs of the present invention are genetically engineered to interact with a specific target pre-mRNA expressed in cells to be targeted so as to result in correction of genetic defects or reprogramming of gene expression responsible for a variety of different skin disorders.
摘要翻译： 本发明涉及特异性和显着改进的mRNA前转录分子（RTM），其被设计用于校正在靶细胞内表达的特异性基因，并且其与大疱性表皮松解，囊性纤维化，先天性先天性和牛皮癣相关 或神经性皮炎，以及皮肤癌。 特别地，本发明的RTM被遗传工程改造以与待靶向的细胞中表达的特异性靶mRNA前体相互作用，从而导致基因缺陷的校正或负责各种不同皮肤病症的基因表达的重新编程。
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13.发明授权Methods and compositions for determining the purity of chemically synthesized nucleic acids 失效用于测定化学合成核酸纯度的方法和组合物公开(公告)号：US08697355B2
公开(公告)日：2014-04-15
申请号：US13670102
申请日：2012-11-06
IPC分类号： G01N33/53
CPC分类号： C07K16/44 , B01J2219/00274 , C07K16/18 , C12Q1/6804 , Y02P20/55
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。
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14.发明申请METHODS AND COMPOSITIONS FOR DETERMINING THE PURITY OF CHEMICALLY SYNTHESIZED NUCLEIC ACIDS 失效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：US20130071951A1
公开(公告)日：2013-03-21
申请号：US13670102
申请日：2012-11-06
IPC分类号： C12Q1/68
CPC分类号： C07K16/44 , B01J2219/00274 , C07K16/18 , C12Q1/6804 , Y02P20/55
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。
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公开(公告)号：US6013487A
公开(公告)日：2000-01-11
申请号：US766354
申请日：1996-12-13
申请人： Lloyd G. Mitchell
发明人： Lloyd G. Mitchell
IPC分类号： C12N15/09 , A01N43/04 , A61K31/70 , A61K31/7105 , A61K31/711 , A61K38/00 , A61K48/00 , A61P31/12 , A61P35/00 , A61P37/06 , A61P43/00 , C07H21/02 , C07H21/04 , C07K14/34 , C12N5/10 , C12N15/00 , C12N15/10 , C12N15/11 , C12N15/63 , C12N15/70 , C12N15/74 , C12N15/85 , C12P19/34
CPC分类号： A61K48/005 , A61K48/00 , C07K14/34 , C12N15/1027 , C12N15/111 , C12N15/63 , C12N15/85 , A61K38/00 , C12N2320/33 , C12N2840/44 , C12N2840/445
摘要： The molecules and methods of the present invention provide a means for in vivo production of a therapeutic molecule in a selected subset of cells. The pre-therapeutic molecules of the invention are substrates for a trans-splicing reaction between the pre-therapeutic molecules and a pre-mRNA which is uniquely expressed in the specific target cells. The in vivo trans-splicing reaction provides an active therapeutic RNA which is functional as RNA or encodes a protein to be expressed in the target cells. The expression product of the mRNA is a protein of therapeutic value to the cell or a toxin which causes killing of the specific cells.
摘要翻译： 本发明的分子和方法提供了在选定的细胞亚群中体内产生治疗分子的方法。 本发明的预处理分子是预处理分子与特异性靶细胞中唯一表达的前mRNA之间的剪接反应的底物。 体内转拼反应提供活性治疗RNA，其作为RNA起作用或编码要在靶细胞中表达的蛋白质。 mRNA的表达产物是对细胞或毒素具有治疗价值的蛋白质，其导致杀死特异性细胞。
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公开(公告)号：US20220062437A1
公开(公告)日：2022-03-03
申请号：US17230354
申请日：2021-04-14
摘要： A nucleic acid trans-splicing molecule is provided that can replace an exon in a targeted mammalian ocular gene carrying a defect or mutation causing an ocular disease with an exon having the naturally-occurring sequence without the defect or mutation. A method of treating an ocular disease, e.g., Stargardt's Disease, caused by a defect or mutation in a target gene, e.g., ABCA4 comprising: administering to the ocular cells of a subject having an ocular disease a composition comprising a recombinant AAV comprising a nucleic acid trans-splicing molecule as described above.
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公开(公告)号：US20180369412A1
公开(公告)日：2018-12-27
申请号：US15776663
申请日：2016-11-18
摘要： A nucleic acid trans-splicing molecule is provided that can replace an exon in a targeted mammalian ocular gene carrying a defect or mutation causing an ocular disease with an exon having the naturally-occurring sequence without the defect or mutation. A method of treating an ocular disease, e.g., Stargardt's Disease, caused by a defect or mutation in a target gene, e.g., ABCA4 comprising: administering to the ocular cells of a subject having an ocular disease a composition comprising a recombinant AAV comprising a nucleic acid trans-splicing molecule as described above.
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公开(公告)号：US20130059901A1
公开(公告)日：2013-03-07
申请号：US13056449
申请日：2009-07-30
申请人： Johann Bauer , Lloyd G. Mitchell
发明人： Johann Bauer , Lloyd G. Mitchell
IPC分类号： A61K31/7088 , A61P37/00 , C12N5/10 , A61P17/00 , C12N15/113 , C12N15/85
CPC分类号： C12N15/85 , A61K48/005 , C07K14/78 , C12N2740/13043 , C12N2840/445
摘要： The present invention relates to specific and markedly improved pre-mRNA trans-splicing molecule (RTM) molecules which are designed to correct specific genes expressed within cells to be targeted, and which are associated with epidermolysis bullosa, cystic fibrosis, pachyonychia congenital, and psoriasis or neurodermitis, as well as cancers of the skin. In particular, the RTMs of the present invention are genetically engineered to interact with a specific target pre-mRNA expressed in cells to be targeted so as to result in correction of genetic defects or reprogramming of gene expression responsible for a variety of different skin disorders.
摘要翻译： 本发明涉及特异性和显着改进的mRNA前转录分子（RTM），其被设计用于校正在靶细胞内表达的特异性基因，并且其与大疱性表皮松解，囊性纤维化，先天性先天性和牛皮癣相关 或神经性皮炎，以及皮肤癌。 特别地，本发明的RTM被遗传工程改造以与待靶向的细胞中表达的特异性靶mRNA前体相互作用，从而导致基因缺陷的校正或负责各种不同皮肤病症的基因表达的重新编程。
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19.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：US08173377B2
公开(公告)日：2012-05-08
申请号：US13016409
申请日：2011-01-28
IPC分类号： G01N33/53
CPC分类号： C07K16/44 , B01J2219/00274 , C07K16/18 , C12Q1/6804 , Y02P20/55
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。
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20.发明授权Sample collection devices and methods using markers and the use of such markers as controls in sample validation, laboratory evaluation and/or accreditation 失效使用标记的样品收集装置和方法，以及在样品验证，实验室评估和/或认证中使用这些标记作为对照公开(公告)号：US06673621B1
公开(公告)日：2004-01-06
申请号：US09147698
申请日：1999-06-09
申请人： Lloyd G. Mitchell
发明人： Lloyd G. Mitchell
IPC分类号： G01N3700
CPC分类号： C12Q1/6816 , G01N2001/007 , Y10T436/13 , Y10T436/143333 , C12Q2563/185
摘要： The invention is directed to a device suitable for marking a collected sample, comprising collecting means for collecting the sample and at least one detectable marker which is associated with at least a portion of the collecting means, wherein the at least one detectable marker is contractable with the sample upon collection of the sample to mark the collected sample upon contact of the sample with the at least a portion of the collecting means having the at least one detectable marker associated therewith, and wherein the at least one detectable marker is other than a component which is present in the sample before collection and is inert to any component present in the sample before collection. Kits containing the device, methods for marking samples using the device, methods for determining the integrity of a marked sample, and use of the markers for the testing of laboratories and/or laboratory personnel for certification, proficiency testing or accreditation purposes are also provided.
摘要翻译： 本发明涉及一种适于标记收集的样品的装置，包括用于收集样品的收集装置和与至少一部分收集装置相关联的至少一个可检测标记，其中至少一个可检测标记可与 所述样品收集样品以在样品与收集装置的至少一部分接触时标记所收集的样品，所述至少一部分具有与其相关联的至少一个可检测标记，并且其中所述至少一种可检测标记物不是组分 其在收集之前存在于样品中，并且对于收集前样品中存在的任何成分是惰性的。 还提供了包含设备的套件，使用设备标记样品的方法，确定标记样品的完整性的方法，以及用于测试实验室和/或实验室人员的标记用于认证，水平测试或认证目的的使用。
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