公开(公告)号：US20050048041A1

公开(公告)日：2005-03-03

申请号：US10867628

申请日：2004-06-15

申请人： Mahendra Rao ,  Mario Capecchi

发明人： Mahendra Rao ,  Mario Capecchi

IPC分类号： C12N5/0797 ,  C12N15/90 ,  A61K48/00 ,  C12N5/08 ,  C12N9/10 ,  C12N15/85

CPC分类号： C12N5/0623 ,  C12N15/902 ,  C12N2510/04 ,  C12N2800/108 ,  C12N2800/30 ,  C12N2800/60 ,  C12N2830/42 ,  C12N2840/203 ,  C12N2840/44

摘要： A method of obtaining and the resulting isolated progenitor or stem cell population of proliferating cells persistently expressing a candidate molecule. Further, novel methods of ex vivo gene product (e.g., protein) production and treating symptoms of neurological or neurodegenerative disorders are also provided.

摘要翻译： 获得和产生的增殖细胞的分离祖细胞或干细胞群的方法持续地表达候选分子。 此外，还提供了离体基因产物（例如蛋白质）生产和治疗神经或神经退行性疾病症状的新方法。

公开(公告)号：US20080295192A1

公开(公告)日：2008-11-27

申请号：US11759574

申请日：2007-06-07

申请人： Kirk R. Thomas ,  Kenneth E. Bernstein ,  Michaeline Bunting ,  Joy Greer ,  Mario Capecchi

发明人： Kirk R. Thomas ,  Kenneth E. Bernstein ,  Michaeline Bunting ,  Joy Greer ,  Mario Capecchi

IPC分类号： A01K67/027 ,  C07H21/04 ,  C12N15/63

CPC分类号： C12N15/63 ,  A01K2217/075 ,  A61K48/00 ,  C12N15/67

摘要： The present invention is directed to a method for deleting DNA sequences in a tissue specific manner. In one embodiment, DNA sequences are specifically deleted in germline tissue. In a second embodiment, DNA sequences are specifically deleted in desired somatic tissue. The present invention is further directed to a nucleic acid molecule for use in the method. More specifically, a nucleic acid molecule is provide by the present invention which comprises (a) a recombinase site, (b) a tissue-specific promoter, (c) a recombinase gene, (d) a foreign DNA, and (e) a recombinase site. The nucleic acid molecule may further comprise a gene which is desired to be incorporated into and expressed in a transgenic organism. The method can be used in both plants and animals, and has many applications as described herein.

摘要翻译： 本发明涉及以组织特异性方式缺失DNA序列的方法。 在一个实施方案中，在序列组织中特异性缺失DNA序列。 在第二个实施方案中，DNA序列在期望的体细胞组织中被特异性缺失。 本发明还涉及用于该方法的核酸分子。 更具体地说，本发明提供了一种核酸分子，其包含（a）重组酶位点，（b）组织特异性启动子，（c）重组酶基因，（d）外源DNA，和（e） 重组酶位点。 核酸分子可以进一步包含希望并入并在转基因生物体中表达的基因。 该方法可以在植物和动物中使用，并且具有如本文所述的许多应用。

公开(公告)号：US20050149998A1

公开(公告)日：2005-07-07

申请号：US10746207

申请日：2003-12-23

申请人： Mario Capecchi ,  Kirk Thomas

发明人： Mario Capecchi ,  Kirk Thomas

IPC分类号： A61K48/00 ,  C07K14/47 ,  C07K14/50 ,  C07K14/755 ,  C12N9/10 ,  C12N15/82 ,  C12N15/90 ,  A01K67/027 ,  C12N15/85

CPC分类号： C12N15/8209 ,  A01K2217/05 ,  A61K48/00 ,  C07K14/47 ,  C07K14/50 ,  C07K14/755 ,  C12N9/1077 ,  C12N15/8213 ,  C12N15/907

摘要： Positive-negative selector (PNS) vectors are provided for modifying a target DNA sequence contained in the genome of a target cell capable of homologous recombination. The vector comprises a first DNA sequence which contains at least one sequence portion which is substantially homologous to a portion of a first region of a target DNA sequence. The vector also includes a second DNA sequence containing at least one sequence portion which is substantially homologous to another portion of a second region of a target DNA sequence. A third DNA sequence is positioned between the first and second DNA sequences and encodes a positive selection marker which when expressed is functional in the target cell in which the vector is used. A fourth DNA sequence encoding a negative selection marker, also functional in the target cell, is positioned 5′ to the first or 3′ to the second DNA sequence and is substantially incapable of homologous recombination with the target DNA sequence. The invention also includes transformed cells containing at least one predetermined modification of a target DNA sequence contained in the genome of the cell. In addition, the invention includes organisms such as non-human transgenic animals and plants which contain cells having predetermined modifications of a target DNA sequence in the genome of the organism.

摘要翻译： 提供阳性 - 阴性选择（PNS）载体用于修饰能够进行同源重组的靶细胞的基因组中包含的靶DNA序列。 载体包含第一DNA序列，其含有与目标DNA序列的第一区域的一部分基本同源的至少一个序列部分。 载体还包括含有至少一个与目标DNA序列的第二区域的另一部分基本同源的序列部分的第二DNA序列。 第三个DNA序列位于第一个和第二个DNA序列之间，并编码一个阳性选择标记，当其表达在其中使用该载体的靶细胞中是有功能的。 编码靶细胞功能的阴性选择标记的第四个DNA序列位于第一个或第三个DNA序列的5'处，并且基本上不能与靶DNA序列同源重组。 本发明还包括含有细胞基因组中包含的靶DNA序列的至少一个预定修饰的转化细胞。 此外，本发明包括诸如非人转基因动物和植物的生物体，其含有具有生物体基因组中靶DNA序列的预定修饰的细胞。