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公开(公告)号：US20060093589A1
公开(公告)日：2006-05-04
申请号：US10513348
申请日：2004-02-19
IPC分类号： A61K48/00 , C12N15/861
CPC分类号： C12N15/86 , A61K48/00 , C12N2750/14143 , C12N2750/14145 , C12N2810/50
摘要： Disclosed are improved VP2-modified recombinant adeno-associated viral (rAAV) vectors, expression systems, and rAAV virions that are fully virulent, yet lack functional VP2 protein expression. Also disclosed are pharmaceutical compositions, virus particles, host cells, and pharmaceutical formulations that comprise these modified vectors useful in the expression of therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes in the cells and tissues of selected mammals, including, for example, human tissues and host cells.
摘要翻译： 公开了改进的VP2修饰的重组腺相关病毒（rAAV）载体，表达系统和完全毒力但缺乏功能性VP2蛋白表达的rAAV病毒粒子。 还公开了包含这些修饰的载体的药物组合物，病毒颗粒，宿主细胞和药物制剂，其用于在所选哺乳动物的细胞和组织中表达治疗性蛋白质，多肽，肽，反义寡核苷酸和/或核酶，包括用于 例如，人体组织和宿主细胞。
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2.发明申请Raav expression systems for genetic modification of specific capsid proteins 有权用于特异性衣壳蛋白遗传修饰的Raav表达系统公开(公告)号：US20060088936A1
公开(公告)日：2006-04-27
申请号：US10513059
申请日：2003-05-01
IPC分类号： C12N15/861
CPC分类号： A61K48/005 , C07K14/005 , C12N15/86 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145 , C12N2810/50 , C12N2810/858
摘要： Disclosed are improved recombinant adeno-associated viral (rAAV) vectors having mutations in one or more capsid proteins. Exemplary vectors are provided that have altered affinity for heparin or heparin sulfate, as well as vectors, expression systems, and rAAV virions that lack functional VP2 protein expression, but are nevertheless, fully virulent. Also provided by the invention are rAAV vector-based compositions, virus particles, host cells, and pharmaceutical formulations that comprise them useful in the expression of selected therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes in selected mammals, including organs, tissues, and human host cells.
摘要翻译： 公开了在一种或多种衣壳蛋白中具有突变的改良的重组腺相关病毒（rAAV）载体。 提供了对肝素或硫酸肝素具有改变的亲和力的示例性载体，以及缺乏功能性VP2蛋白表达但仍然完全毒性的载体，表达系统和rAAV病毒体。 本发明还提供了基于rAAV载体的组合物，病毒颗粒，宿主细胞和药物制剂，其包含它们可用于选择的哺乳动物（包括器官）中选择的治疗性蛋白质，多肽，肽，反义寡核苷酸和/或核酶的表达， 组织和人宿主细胞。
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公开(公告)号：US6020192A
公开(公告)日：2000-02-01
申请号：US893327
申请日：1997-07-16
IPC分类号： G01N33/48 , C07K14/435 , C07K14/47 , C07K19/00 , C12N5/10 , C12N15/00 , C12N15/09 , C12N15/12 , C12N15/87 , C12P21/02 , C12Q1/02 , C12Q1/68 , C12R1/91 , C12N15/63 , C07H21/04
CPC分类号： C12N15/87 , C07K14/43595 , C07K2319/00 , C12N2799/025 , C12N2799/027
摘要： Disclosed are synthetic and "humanized" versions of green fluorescent protein (GFP) genes adapted for high level expression in mammalian cells, especially those of human origin. Base substitutions are made in various codons in order to change the codon usage to one more appropriate for expression in mammalian cells. Recombinant vectors carrying such humanized genes are also disclosed. In addition, various methods for using the efficient expression of humanized GFP in mammalian cells and in animals are described.
摘要翻译： 公开了适用于哺乳动物细胞，特别是人类来源的高水平表达的绿色荧光蛋白（GFP）基因的合成和“人源化”形式。 在各种密码子中进行碱基取代，以便将密码子使用改为更适于在哺乳动物细胞中表达的一种。 还公开了携带这种人源化基因的重组载体。 此外，描述了在哺乳动物细胞和动物中使用有效表达人源化GFP的各种方法。
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公开(公告)号：US5741683A
公开(公告)日：1998-04-21
申请号：US477538
申请日：1995-06-07
申请人： Xiaohuai Zhou , Nicholas Muzyczka , Sergei Zolotukhin , Tiehua Ni
发明人： Xiaohuai Zhou , Nicholas Muzyczka , Sergei Zolotukhin , Tiehua Ni
IPC分类号： A61K48/00 , C07K14/015 , C12N5/10 , C12N7/04 , C12N15/864 , C12Q1/70 , C12N7/00 , C12N15/00 , C12N15/85 , C12Q7/00 , C12Q15/00 , C12Q15/85
CPC分类号： C12N7/00 , C07K14/005 , C12N15/86 , A61K48/00 , C12N2750/14122 , C12N2750/14143 , C12N2750/14152
摘要： A method for in vitro packaging of adeno-associated viral particles is described. The procedure involves the preparation of cell-free extracts containing all the essential components for packaging. Homogeneous purified substrate DNA for packaging may be prepared separately. The in vitro packaged AAV particles are useful in transduction of mammalian cells and for gene therapy in animals. In one described method, the DNA packaged into AAV particles is not limited by the size constraints characteristic of in vivo packaged AAV particles.
摘要翻译： 描述了腺相关病毒颗粒的体外包装方法。 该方法涉及制备包含所有必需成分的无细胞提取物。 用于包装的均匀纯化的底物DNA可以单独制备。 体外包装的AAV颗粒可用于哺乳动物细胞的转导和动物基因治疗。 在一种描述的方法中，封装成AAV颗粒的DNA不受体内包装的AAV颗粒特征的尺寸约束的限制。
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公开(公告)号：US07098374B2
公开(公告)日：2006-08-29
申请号：US09780041
申请日：2001-02-09
申请人： Ronald Klein , Edwin Meyer , Nicholas Muzyczka , Mike King , Craig Meyers
发明人： Ronald Klein , Edwin Meyer , Nicholas Muzyczka , Mike King , Craig Meyers
IPC分类号： A01K67/00 , A01K67/033
CPC分类号： C12N15/8509 , A01K67/0278 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2227/105 , A01K2267/03 , A01K2267/0312 , A01K2267/0318 , C07K14/47 , C07K14/4711 , C12N15/86 , C12N2750/14143 , C12N2810/6018 , C12N2830/00 , C12N2830/15 , C12N2830/60 , C12N2830/90 , C12N2840/20 , C12N2840/203 , G01N33/5088
摘要： This invention provides a system for modeling neurodegenerative and other diseases through somatic gene transfer. In addition, methods of multiple gene transfer, disease analysis and drug testing are provided for.
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公开(公告)号：US06660514B1
公开(公告)日：2003-12-09
申请号：US09621475
申请日：2000-07-21
IPC分类号： C12N702
CPC分类号： C12N7/00 , C12N15/86 , C12N2750/14143 , C12N2750/14151 , Y10S435/803 , Y10S435/948 , Y10S530/826
摘要： Disclosed are methods for the isolation and purification of high-titer recombinant adeno-associated virus (rAAV) compositions. Also disclosed are methods for reducing or eliminating the concentration of helper adenovirus in rAAV samples. Methods are disclosed that provide highly-purified rAAV stocks having titers up to about 1013 particles/ml at particle-to-infectivity ratios of less than 100 in processes that are accomplished about 24 hours or less.
摘要翻译： 公开了分离和纯化高滴度重组腺相关病毒（rAAV）组合物的方法。 还公开了减少或消除rAAV样品中辅助性腺病毒浓度的方法。 公开了提供在约24小时或更短时间内完成的过程中以小于100的颗粒感染率比例高达约10 13个颗粒/毫升的高度纯化的rAAV原料的方法。
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公开(公告)号：US5874304A
公开(公告)日：1999-02-23
申请号：US588201
申请日：1996-01-18
IPC分类号： G01N33/48 , C07K14/435 , C07K14/47 , C07K19/00 , C12N5/10 , C12N15/00 , C12N15/09 , C12N15/12 , C12N15/87 , C12P21/02 , C12Q1/02 , C12Q1/68 , C12R1/91 , C07H21/04 , C12N15/63 , C12N15/85
CPC分类号： C07K14/43595 , C12N15/87 , C07K2319/00 , C12N2799/025 , C12N2799/027
摘要： Disclosed are synthetic and "humanized" versions of green fluorescent protein (GFP) genes adapted for high level expression in mammalian cells, especially those of human origin. Base substitutions are made in various codons in order to change the codon usage to one more appropriate for expression in mammalian cells. Recombinant vectors carrying such humanized genes are also disclosed. In addition, various methods for using the efficient expression of humanized GFP in mammalian cells and in animals are described.
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公开(公告)号：US20060292117A1
公开(公告)日：2006-12-28
申请号：US10511914
申请日：2003-04-17
申请人： Scott Loiler , Terence Flotte , Nicholas Muzyczka , Mark Atkinson
发明人： Scott Loiler , Terence Flotte , Nicholas Muzyczka , Mark Atkinson
IPC分类号： A61K48/00 , A61K9/127 , C12N15/861
CPC分类号： C12N15/86 , A61K48/00 , C07K14/005 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145 , C12N2810/858
摘要： Disclosed are methods for the use of therapeutic polypeptide-encoding polynucleotides in the creation of transformed host cells and transgenic animals. In particular, the use of recombinant adeno-associated viral (rAAV) vector compositions that specifically target mammalian cells, such as pancreatic islets cells, that express low-density lipoprotein receptors on their cell surface. The disclosed vectors comprise one or more polynucleotide sequences that express one or more mammalian polypeptides having therapeutic efficacy in the amelioration, treatment and/or prevention of AAT- or cytokine polypeptide deficiencies, such as for example in diabetes and related diseases, as well as a variety of autoimmune disorders including, for example, lupus and rheumatoid arthritis.
摘要翻译： 公开了在产生转化的宿主细胞和转基因动物中使用治疗性多肽编码多核苷酸的方法。 特别地，使用特异性靶向哺乳动物细胞的重组腺相关病毒（rAAV）载体组合物，所述哺乳动物细胞例如胰岛细胞在其细胞表面上表达低密度脂蛋白受体。 所公开的载体包含一种或多种多核苷酸序列，其表达一种或多种在改善，治疗和/或预防AAT或细胞因子多肽缺陷（例如糖尿病和相关疾病）中具有治疗功效的哺乳动物多肽，以及 多种自身免疫性疾病，包括例如狼疮和类风湿性关节炎。
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公开(公告)号：US07094604B2
公开(公告)日：2006-08-22
申请号：US10798192
申请日：2004-03-11
申请人： Richard O. Snyder , Sergei Zolotukhin , Yoshihisa Sakai , Barry J. Byrne , Mark R. Potter , Irine Zolotukhin , Scott Loiler , Vince A. Chiodo , Nicholas Muzyczka , William W. Hauswirth , Terence R. Flotte , Corinna Burger , Edgardo Rodriguez , Kevin R. Nash , Thomas J. Fraites
发明人： Richard O. Snyder , Sergei Zolotukhin , Yoshihisa Sakai , Barry J. Byrne , Mark R. Potter , Irine Zolotukhin , Scott Loiler , Vince A. Chiodo , Nicholas Muzyczka , William W. Hauswirth , Terence R. Flotte , Corinna Burger , Edgardo Rodriguez , Kevin R. Nash , Thomas J. Fraites
IPC分类号： C12N5/10 , C12N15/864 , C12N15/35
CPC分类号： C12N15/86 , C12N2710/10322 , C12N2750/14143 , C12N2840/20
摘要： Vectors that encode Adeno-Associated Virus (AAV) Rep and Cap proteins of different serotypes and Adenovirus transcription products that provide helper functions were used to produce pseudotyped recombinant AAV (rAAV) virions. Purification methods generated pseudotyped rAAV virion stocks that were 99% pure with titers of 1×1012–1×1013 vector genomes/ml.
摘要翻译： 使用编码提供辅助功能的不同血清型和腺病毒转录产物的腺相关病毒（AAV）Rep和Cap蛋白的载体产生假型重组AAV（rAAV）病毒粒子。 纯化方法产生99％纯度的假型分离的rAAV病毒颗粒，其滴度为1×10 12×10 13个载体基因组/ ml。
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10.发明申请Raav expression systems and methods for enhancing transduction of mammalian neural cells 审中-公开用于增强哺乳动物神经细胞转导的Raav表达系统和方法公开(公告)号：US20060127358A1
公开(公告)日：2006-06-15
申请号：US10513064
申请日：2003-05-01
IPC分类号： A61K48/00 , C12N15/861
CPC分类号： C12N15/86 , A61K48/00 , C12N2750/14143
摘要： Disclosed are serotype-specific recombinant adeno-associated viral (rAAV) vectors, as well as viral particles and compositions comprising them, useful in the expression of neurotherapeutic agents (including neurotherapeutic peptides and polypeptides) in selected mammalian neural cells, as well as tissues and organ systems comprising them. In particular embodiments, rAAV serotype 1 and serotype 5 vectors are disclosed useful for the delivery of therapeutic agents to neural cells of affected mammals.
摘要翻译： 所公开的是血清型特异性重组腺相关病毒（rAAV）载体，以及包含它们的病毒颗粒和组合物，其可用于选择的哺乳动物神经细胞中神经治疗剂（包括神经治疗肽和多肽）的表达，以及组织和 包括它们的器官系统。 在具体实施方案中，公开了rAAV血清型1和血清型5载体，其用于将治疗剂递送至受影响的哺乳动物的神经细胞。
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