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1.
公开(公告)号:US6106827A
公开(公告)日:2000-08-22
申请号:US862438
申请日:1997-05-24
CPC分类号: C12N9/0071 , A61K48/0058 , C12N15/85 , C12N9/1211 , C12Y114/13041 , C12Y114/16002 , A61K48/00 , C12N2830/002 , C12N2830/008 , C12N2830/85
摘要: A genetically modified astrocyte for gene therapy is provided. The genetically modified astrocyte includes one or more stably introduced DNA sequences selected from DNA encoding a selectable marker, DNA encoding a poison pill, and DNA encoding a molecule useful for gene therapy. The genetically modified astrocyte may be produced utilizing plasmids and non-viral transfection methods, as are also provided by the subject invention. Methods for producing and utilizing the genetically modified astrocytes and regulating the engineered products, as well as kits thereof, are further provided.
摘要翻译: 提供用于基因治疗的转基因星形胶质细胞。 遗传修饰的星形胶质细胞包括选自编码选择性标记的DNA,编码毒药的DNA和编码可用于基因治疗的分子的DNA的一种或多种稳定导入的DNA序列。 基因修饰的星形胶质细胞可以利用本发明也提供的质粒和非病毒转染方法产生。 进一步提供生产和利用转基因星形胶质细胞并调节工程化产品的方法及其试剂盒。
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2.
公开(公告)号:US20040071678A1
公开(公告)日:2004-04-15
申请号:US10674598
申请日:2003-09-30
IPC分类号: A61K048/00 , C12N005/08
CPC分类号: C12N9/0071 , A61K48/00 , A61K48/0058 , C12N9/1211 , C12N15/85 , C12N2830/002 , C12N2830/008 , C12N2830/85 , C12Y114/13041 , C12Y114/16002
摘要: A genetically modified astrocyte for gene therapy is provided. The genetically modified astrocyte includes one or more stably introduced DNA sequences selected from DNA encoding a selectable marker, DNA encoding a poison pill, and DNA encoding a molecule useful for gene therapy. The genetically modified astrocyte may be produced utilizing plasmids and non-viral transfection methods, as are also provided by the subject invention. Methods for producing and utilizing the genetically modified astrocytes and regulating the engineered products, as well as kits thereof, are further provided.
摘要翻译: 提供用于基因治疗的转基因星形胶质细胞。 遗传修饰的星形胶质细胞包括选自编码选择性标记的DNA,编码毒药的DNA和编码可用于基因治疗的分子的DNA的一种或多种稳定导入的DNA序列。 基因修饰的星形胶质细胞可以利用本发明也提供的质粒和非病毒转染方法产生。 进一步提供生产和利用转基因星形胶质细胞并调节工程化产品的方法及其试剂盒。
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公开(公告)号:US5869463A
公开(公告)日:1999-02-09
申请号:US458384
申请日:1995-06-02
IPC分类号: C12N15/09 , A61K31/70 , A61K35/12 , A61K35/30 , A61K35/54 , A61K38/44 , A61K48/00 , A61P25/28 , C12N5/079 , C12N5/10 , C12N9/02 , C12N15/85
CPC分类号: C12N9/0071 , A61K35/30 , A61K35/54 , A61K48/00 , C12N15/85 , C12N5/0622 , C12Y114/13041 , C12Y114/16002 , A61K2035/126 , A61K38/00 , C12N2510/04 , Y10S435/948
摘要: Human fetal neuro-derived cell lines are implanted into host tissues. The methods allow for treatment of a variety of neurological disorders and other diseases. A preferred cell line is SVG.
摘要翻译: 将人胎儿神经衍生的细胞系植入宿主组织。 该方法允许治疗各种神经障碍和其他疾病。 优选的细胞系是SVG。
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公开(公告)号:US20030129170A1
公开(公告)日:2003-07-10
申请号:US10215647
申请日:2002-08-09
IPC分类号: G01N033/567 , C07H021/04 , C12N009/06 , A61K048/00 , C12N005/08
CPC分类号: C12N9/0073 , A01K2217/05 , A61K38/00 , A61K48/00 , C12N15/65 , C12N15/85 , C12N2830/00 , C12N2830/85 , C12Q1/6883 , C12Q2600/158 , C12Y114/13041
摘要: The present invention provides an isolated, purified and characterized human tyrosine hydroxylase (hTH) promoter nucleic acid sequence. The invention further provides a method of selecting TH positive (THnull) cells by preparing a construct comprising a hTH promoter operably linked to a heterologous nucleic acid sequence, for example, green fluorescent protein encoding sequence, and transfecting cells, particularly stem cells, with the construct. The invention also provides a hTH promoter, useful in gene therapeutic applications in driving therapeutic genes or other nucleic acid sequences operably linked to the hTH promoter. Additionally, the invention provides cell lines and transgenic animals expressing a transgene comprising the hTH promoter operably linked to a heterologous sequence, which cell lines and transgenic animals are useful for isolating THnull cells for transplantation or for screening of therapeutic agents that affect THnull function. Methods of producing cell lines and transgenic animals also provided.
摘要翻译: 本发明提供分离的,纯化的和表征的人酪氨酸羟化酶(hTH)启动子核酸序列。 本发明还提供了通过制备包含可操作地连接到异源核酸序列例如绿色荧光蛋白编码序列的hTH启动子,并转染细胞,特别是干细胞的构建体来选择TH阳性(TH +)细胞的方法,其中 构造。 本发明还提供了一种hTH启动子,其可用于驱动治疗性基因或与hTH启动子可操作地连接的其它核酸序列的基因治疗应用。 另外,本发明提供了表达包含与异源序列可操作地连接的hTH启动子的转基因的细胞系和转基因动物,该细胞系和转基因动物可用于分离用于移植的TH +细胞或用于筛选影响TH +功能的治疗剂。 还提供了产生细胞系和转基因动物的方法。
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5.
公开(公告)号:US20020110546A1
公开(公告)日:2002-08-15
申请号:US09973198
申请日:2001-10-10
申请人: Pro-Virus, Inc.
IPC分类号: A61K048/00
CPC分类号: C12N9/0071 , A61K35/30 , A61K35/54 , A61K38/00 , A61K48/00 , A61K2035/126 , C12N5/0622 , C12N15/85 , C12N2510/04 , C12Y114/13041 , C12Y114/16002 , Y10S435/948
摘要: Human fetal neuro-derived cell lines are implanted into host tissues. The methods allow for treatment of a variety of neurological disorders and other diseases. A preferred cell line is SVG.
摘要翻译: 将人胎儿神经衍生的细胞系植入宿主组织。 该方法允许治疗各种神经障碍和其他疾病。 优选的细胞系是SVG。
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公开(公告)号:US07195910B2
公开(公告)日:2007-03-27
申请号:US10215647
申请日:2002-08-09
CPC分类号: C12N9/0073 , A01K2217/05 , A61K38/00 , A61K48/00 , C12N15/65 , C12N15/85 , C12N2830/00 , C12N2830/85 , C12Q1/6883 , C12Q2600/158 , C12Y114/13041
摘要: The present invention provides an isolated, purified and characterized human tyrosine hydroxylase (hTH) promoter nucleic acid sequence. The invention further provides a method of selecting TH positive (TH+) cells by preparing a construct comprising a hTH promoter operably linked to a heterologous nucleic acid sequence, for example, green fluorescent protein encoding sequence, and transfecting cells, particularly stem cells, with the construct. The invention also provides a hTH promoter, useful in gene therapeutic applications in driving therapeutic genes or other nucleic acid sequences operably linked to the hTH promoter. Additionally, the invention provides cell lines and transgenic animals expressing a transgene comprising the hTH promoter operably linked to a heterologous sequence, which cell lines and transgenic animals are useful for isolating TH+ cells for transplantation or for screening of therapeutic agents that affect TH+ function. Methods of producing cell lines and transgenic animals also provided.
摘要翻译: 本发明提供分离的,纯化的和表征的人酪氨酸羟化酶(hTH)启动子核酸序列。 本发明还提供了通过制备包含可操作地连接到异源核酸序列例如绿色荧光蛋白编码序列的hTH启动子,并转染细胞,特别是干细胞的构建体来选择TH阳性(TH +)细胞的方法,其中 构造。 本发明还提供了一种hTH启动子,其可用于驱动治疗性基因或与hTH启动子可操作地连接的其它核酸序列的基因治疗应用。 另外,本发明提供了表达包含与异源序列可操作地连接的hTH启动子的转基因的细胞系和转基因动物,该细胞系和转基因动物可用于分离用于移植的TH +细胞或用于筛选影响TH +功能的治疗剂。 还提供了产生细胞系和转基因动物的方法。
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公开(公告)号:US6087171A
公开(公告)日:2000-07-11
申请号:US362495
申请日:1996-11-18
申请人: Toomas Neuman , Kikuo Suda , Howard O. Nornes
发明人: Toomas Neuman , Kikuo Suda , Howard O. Nornes
IPC分类号: C07K14/075 , C07K14/47 , C07K14/48 , C12N9/02 , C12N15/85 , A61K48/00 , C12N15/87 , C12N15/88 , C12N15/90
CPC分类号: C07K14/005 , C07K14/4738 , C07K14/48 , C12N15/85 , C12N9/0071 , C12Y114/13041 , C12Y114/16002 , C12N2710/10322
摘要: A method is provided for inducing DNA synthesis in differentiated neurons. According to certain embodiments of the invention, a method for inducing DNA synthesis in a differentiated neuron is provided that includes obtaining a vector comprising nucleic acid encoding an E2F regulator and/or an E1A regulator, wherein the vector can be used to express the nucleic acid in a differentiated neuron, and transfecting a differentiated neuron with the vector. According to certain embodiments of the invention, a method for integrating DNA encoding a desired protein in a differentiated neuron is provided that includes obtaining a vector comprising nucleic acid encoding an E2F regulator and/or an E1A regulator, wherein the vector can be used to express the nucleic acid in a neuron, obtaining DNA encoding a desired protein, and cotransfecting a differentiated neuron with the vector and the DNA encoding the desired protein such that the DNA encoding the desired protein is integrated in the differentiated neuron and the desired protein is produced.
摘要翻译: PCT No.PCT / US94 / 14614 Sec。 371日期:1996年11月18日 102(e)1996年11月18日PCT PCT 1994年12月19日PCT公布。 公开号WO95 / 16774 日期:1995年6月22日提供了用于在分化的神经元中诱导DNA合成的方法。 根据本发明的某些实施方案,提供了在分化神经元中诱导DNA合成的方法,其包括获得包含编码E2F调节子和/或E1A调节子的核酸的载体,其中所述载体可用于表达所述核酸 在分化神经元中,并用载体转染分化的神经元。 根据本发明的某些实施方案,提供了一种用于整合在分化神经元中编码所需蛋白质的DNA的方法,其包括获得包含编码E2F调节剂和/或E1A调节剂的核酸的载体,其中所述载体可用于表达 神经元中的核酸,获得编码期望蛋白质的DNA,并用载体和编码所需蛋白质的DNA共转染分化的神经元,使得编码期望的蛋白质的DNA整合在分化的神经元和所需的蛋白质中。
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公开(公告)号:US5690927A
公开(公告)日:1997-11-25
申请号:US459136
申请日:1995-06-02
IPC分类号: C12N15/09 , A61K31/70 , A61K35/12 , A61K35/30 , A61K35/54 , A61K38/44 , A61K48/00 , A61P25/28 , C12N5/079 , C12N5/10 , C12N9/02 , C12N15/85
CPC分类号: C12N9/0071 , A61K35/30 , A61K35/54 , A61K48/00 , C12N15/85 , C12N5/0622 , C12Y114/13041 , C12Y114/16002 , A61K2035/126 , A61K38/00 , C12N2510/04 , Y10S435/948
摘要: Human fetal neuro-derived cell lines are implanted into host tissues. The methods allow for treatment of a variety of neurological disorders and other diseases. A preferred cell line is SVG.
摘要翻译: 将人胎儿神经衍生的细胞系植入宿主组织。 该方法允许治疗各种神经障碍和其他疾病。 优选的细胞系是SVG。
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公开(公告)号:US20030027779A1
公开(公告)日:2003-02-06
申请号:US10057777
申请日:2002-01-25
申请人: Spinal Cord Society
发明人: Toomas Neuman , Kikuo Suda , Howard O. Nornes
IPC分类号: A61K048/00 , A61K009/127 , C12N015/88
CPC分类号: C07K14/005 , C07K14/4738 , C07K14/48 , C12N9/0071 , C12N15/85 , C12N2710/10322 , C12Y114/13041 , C12Y114/16002
摘要: A method is provided for inducing DNA synthesis in differentiated neurons. According to certain embodiments of the invention, a method for inducing DNA synthesis in a differentiated neuron is provided that includes obtaining a vector comprising nucleic acid encoding an E2F regulator and/or an E1A regulator, wherein the vector can be used to express the nucleic acid in a differentiated neuron, and transfecting a differentiated neuron with the vector. According to certain embodiments of the invention, a method for integrating DNA encoding a desired protein in a differentiated neuron is provided that includes obtaining a vector comprising nucleic acid encoding an E2F regulator and/or an E1A regulator, wherein the vector can be used to express the nucleic acid in a neuron, obtaining DNA encoding a desired protein, and cotransfecting a differentiated neuron with the vector and the DNA encoding the desired protein such that the DNA encoding the desired protein is integrated in the differentiated neuron and the desired protein is produced.
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公开(公告)号:US20020106794A1
公开(公告)日:2002-08-08
申请号:US09942325
申请日:2001-08-29
IPC分类号: A61K048/00 , C12N005/08 , C07H021/04 , C12N015/87
CPC分类号: C12N9/0073 , A01K2217/05 , A61K38/00 , A61K48/00 , C12N15/65 , C12N15/85 , C12N2830/00 , C12N2830/85 , C12Q1/6883 , C12Q2600/158 , C12Y114/13041
摘要: The differentiated cells of the adult mammalian central nervous system (CNS) have little or no ability to generate new nerve cells. This inability to produce new nerve cells is a distinct disadvantage when the need to replace lost neurons arises due to injury or disease. The present invention provides the sequence of 10.828 kB of the human tyrosine hydroxylase promoter. This sequence is used to purify dopaminergic cells, thus providing treatment for neurological diseases or disorders, such as Parkinson's disease, wherein a biologically active tyrosine hydroxylase is limiting or absent.
摘要翻译: 成年哺乳动物中枢神经系统(CNS)的分化细胞几乎没有或没有产生新神经细胞的能力。 当由于损伤或疾病而需要替换丢失的神经元时,这种不能产生新的神经细胞是一个明显的缺点。 本发明提供10.828 kB的人酪氨酸羟化酶启动子的序列。 该序列用于纯化多巴胺能细胞,从而提供治疗神经疾病或病症,例如帕金森病,其中生物活性酪氨酸羟化酶是有限制的或不存在的。
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