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1. 发明申请

US20050255086A1 Nucleic acid silencing of Huntington's Disease gene 审中-公开
标题翻译：亨廷顿病基因的核酸沉默
公开(公告)号：US20050255086A1
公开(公告)日：2005-11-17
申请号：US11048627
申请日：2005-01-31
申请人： Beverly Davidson , Scott Harper
发明人： Beverly Davidson , Scott Harper
IPC分类号： A61K38/00 , A61K48/00 , C07H21/02 , C12N5/22 , C12N15/113 , C12N15/861
CPC分类号： C12N15/113 , A01K2217/05 , A61K38/00 , A61K48/00 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2799/021 , C12N2799/022 , Y02A50/465
摘要： The present invention is directed to small interfering RNA molecules (siRNA) targeted against a Huntington's Disease gene, and methods of using these siRNA molecules.
摘要翻译：本发明涉及针对亨廷顿疾病基因的小干扰RNA分子（siRNA）以及使用这些siRNA分子的方法。

2. 发明申请

US20060084620A1 RNA interference in respiratory epitheial cells 有权
标题翻译：呼吸道上皮细胞中的RNA干扰
公开(公告)号：US20060084620A1
公开(公告)日：2006-04-20
申请号：US11179848
申请日：2005-07-11
申请人： Paul McCray , Beverly Davidson , Anthony Fischer , Hong Jia , Maureen Donovan , Patrick Sinn , Mark Behlke
发明人： Paul McCray , Beverly Davidson , Anthony Fischer , Hong Jia , Maureen Donovan , Patrick Sinn , Mark Behlke
IPC分类号： A61K48/00 , C07H21/02
CPC分类号： C12N15/1131 , C12N15/111 , C12N15/1136 , C12N15/1137 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/31 , C12N2320/32
摘要： The present invention is directed to small interfering RNA molecules targeted against a gene of interest in respiratory epithelial cells, and methods of using these RNA molecules.
摘要翻译：本发明涉及针对呼吸上皮细胞中目的基因的小干扰RNA分子，以及使用这些RNA分子的方法。

3. 发明申请

US20050123517A1 Methods for producing and using in vivo pseudotyped retroviruses using envelope glycoproteins from lymphocytic choriomeningitis virus (LCMV) 有权
公开(公告)号：US20050123517A1
公开(公告)日：2005-06-09
申请号：US10993319
申请日：2004-11-19
申请人： Paul McCray , Beverly Davidson , Colleen Stein
发明人： Paul McCray , Beverly Davidson , Colleen Stein
IPC分类号： A61K48/00 , C07K14/08 , C12N5/10 , C12N15/867 , C07K14/005 , C12N7/00 , C12N15/86
CPC分类号： C12N15/86 , A61K48/00 , C07K14/005 , C12N2740/15043 , C12N2740/15045 , C12N2760/10022 , C12N2810/6072
摘要： The present invention provides novel pseudotyped retroviral vectors that can transduce human and other cells. Vectors are provided that are packaged efficiently in packaging cells and cell lines to generate high titer recombinant virus stocks expressing novel envelope glycoproteins. The present invention further relates to compositions for gene therapy.

4. 发明授权

US06855549B1 Methods and compositions for increasing the infectivity of gene transfer vectors 失效
标题翻译：增加基因转移载体感染性的方法和组合物
公开(公告)号：US06855549B1
公开(公告)日：2005-02-15
申请号：US09448613
申请日：1999-11-22
申请人： Paul B. McCray, Jr. , Guoshun Wang , Beverly Davidson , Mordechai Bodner , Steven M. Herrmann , Douglas J. Jolly
发明人： Paul B. McCray, Jr. , Guoshun Wang , Beverly Davidson , Mordechai Bodner , Steven M. Herrmann , Douglas J. Jolly
IPC分类号： A01N43/04 , A61K9/00 , A61K47/18 , A61K48/00 , C07K14/47 , C12N15/86 , C12Q1/68
CPC分类号： A61K48/0008 , A61K9/0073 , A61K47/183 , A61K48/00 , C07K14/4712 , C12N2799/025 , C12N2799/027
摘要： The present invention involves methods and compositions for increasing the susceptibility of target cells to transduction by gene transfer vectors. Specifically, it is proposed that increasing intracellular permeability in epithelial tissue increases the percentage of input vector that will transduce that target tissue. Specific examples show that receptors for retrovirus are preferentially accessible on the basolateral surface of airway epithelia, and permeabilizing such tissues results in greater infection with retrovirus. This has important implications in gene therapy, for example, to treat cystic fibrosis with the CFTR gene.
摘要翻译：本发明涉及通过基因转移载体增加靶细胞对转导的敏感性的方法和组合物。具体来说，提出增加上皮组织中的细胞内通透性增加了将转导该靶组织的输入载体的百分比。具体实例表明，逆转录病毒的受体优先在气道上皮的基底外侧表面可及，并且透化这样的组织导致逆转录病毒感染更大。这在基因治疗中具有重要意义，例如用CFTR基因治疗囊性纤维化。

5. 发明授权

US06468524B1 AAV4 vector and uses thereof 有权
标题翻译： AAV4载体及其用途
公开(公告)号：US06468524B1
公开(公告)日：2002-10-22
申请号：US09532594
申请日：2000-03-22
申请人： John A. Chiorini , Robert M. Kotin , Beverly Davidson
发明人： John A. Chiorini , Robert M. Kotin , Beverly Davidson
IPC分类号： A61K4800
CPC分类号： C12N15/86 , A61K48/0058 , C12N2750/14143
摘要： The present invention relates to AAV4 vectors for methods of delivering nucleic acids to cells. Specifically, the present invention provides methods of delivering nucleic acids to specific regions and cells of the brain, particularly ependymal cells.
摘要翻译：本发明涉及用于将核酸递送至细胞的方法的AAV4载体。具体地，本发明提供了将核酸递送到脑的特定区域和细胞，特别是室管膜细胞的方法。

6. 发明申请

US20070059291A1 Methods for producing and using in vivo pseudotyped retroviruses using envelope glycoproteins from lymphocytic choriomeningitis virus (LCMV) 有权
公开(公告)号：US20070059291A1
公开(公告)日：2007-03-15
申请号：US11593963
申请日：2006-11-07
申请人： Paul McCray , Beverly Davidson , Colleen Stein
发明人： Paul McCray , Beverly Davidson , Colleen Stein
IPC分类号： A61K48/00 , C12N15/867
CPC分类号： C12N15/86 , A61K48/00 , C07K14/005 , C12N2740/15043 , C12N2740/15045 , C12N2760/10022 , C12N2810/6072
摘要： The present invention provides novel pseudotyped retroviral vectors that can transduce human and other cells. Vectors are provided that are packaged efficiently in packaging cells and cell lines to generate high titer recombinant virus stocks expressing novel envelope glycoproteins. The present invention further relates to compositions for gene therapy.

7. 发明申请

US20050100890A1 Methods for producing and using in vivo pseudotyped retroviruses 审中-公开
标题翻译：用于体内假型逆转录病毒的生产和使用方法
公开(公告)号：US20050100890A1
公开(公告)日：2005-05-12
申请号：US10964574
申请日：2004-10-13
申请人： Beverly Davidson , Paul McCray
发明人： Beverly Davidson , Paul McCray
IPC分类号： A61K48/00 , C07H21/04 , C07K14/005 , C07K14/11 , C07K14/15 , C12N7/00 , C12N15/867 , C12Q1/70
CPC分类号： C12N15/85 , A61K48/00 , C12N15/86 , C12N15/867 , C12N2015/8518 , C12N2710/14022 , C12N2740/15043 , C12N2740/15045 , C12N2800/107 , C12N2810/60
摘要： The present invention provides novel pseudotyped retroviral vectors that can transduce human and other cells. Vectors are provided that are packaged efficiently in packaging cells and cell lines to generate high titer recombinant virus stocks expressing novel envelope glycoproteins. The present invention further relates to compositions for gene therapy.
摘要翻译：本发明提供可以转导人和其他细胞的新型假型逆转录病毒载体。提供了在包装细胞和细胞系中有效包装以产生表达新的包膜糖蛋白的高效价重组病毒原种的载体。本发明还涉及用于基因治疗的组合物。

8. 发明授权

US06262035B1 Gene replacement therapy for muscular dystrophy 有权
标题翻译：肌营养不良症的基因替代疗法
公开(公告)号：US06262035B1
公开(公告)日：2001-07-17
申请号：US09164664
申请日：1998-10-01
申请人： Kevin P. Campbell , Kathleen H. Holt , Franck Duclos , Leland E. Lim , Volker Straub , Beverly Davidson , Roger Williamson
发明人： Kevin P. Campbell , Kathleen H. Holt , Franck Duclos , Leland E. Lim , Volker Straub , Beverly Davidson , Roger Williamson
IPC分类号： A01N4304
CPC分类号： C12N15/8509 , A01K67/0276 , A01K2207/15 , A01K2217/00 , A01K2217/075 , A01K2227/105 , A01K2267/0306 , A61K38/1709 , A61K48/00
摘要： Disclosed is a method for treating a patient suffering from the disease sarcoglycan-deficient limb-girdle muscular dystrophy by gene replacement therapy. Sarcoglycan gene replacement therapy produces extensive long-term expression of the sarcoglycan species which restores the entire sarcoglycan complex, results in the stable association of alph&agr;-dystroglycan with the sarcolemma, and eliminates the morphological markers of limb-girdle muscular dystrophy. In another aspect, the invention relates to a method for determining a specific defective sarcoglycan species in the tissue of a patient. The method involves culture of muscle cells obtained from the patient, and the independent introduction of expression vectors encoding each of the sarcoglycan species, &agr;, &bgr;, &ggr;, and &dgr;, into the cultured cells with subsequent assaying for restoration of the dystrophin-glycoprotein complex. In another aspect, the invention relates to a mouse, and cells derived therefrom, homozygous for a disrupted &agr;-sarcoglycan gene. The disruption prevents the synthesis of functional &agr;-sarcoglycan in cells of the mouse and results in the mutant mouse having no detectable sarcospan, &bgr;-, &ggr;-, &dgr;-sarcoglycan, and reduced &agr;-dystroglycan in the sarcolemma of skeletal and cardiac muscles, and a reduction of dystrophin in skeletal muscle, when compared to tissue of a mouse lacking a disrupted &agr;-sarcoglycan gene. In another aspect, the invention relates to methods for screening for therapeutic agents useful in the treatment of sarcoglycan-deficient limb-girdle muscular dystrophy. The methods involve administering a candidate therapeutic agent to a mouse, or cells derived therefrom, and assaying for therapeutic effects on the mouse or cells, with the determination of therapeutic effects being a reduction or reversal in disease progression, or a restoration of the dystroglycan complex.
摘要翻译：本发明公开了一种通过基因替代疗法治疗患有疾病肌糖肌苷缺乏症的肢体肌营养不良症的患者的方法。 Sarcoglycan基因替代疗法产生广泛的长期表达的sarcoglycan物种，其恢复整个slegoglycan复合物，导致αphorpha-dystroglycan与肌膜的稳定关联，并消除肢体肌营养不良的形态学标志物。另一方面，本发明涉及一种用于确定患者组织中的特定缺陷型糖团聚物物种的方法。该方法包括从患者获得的肌肉细胞的培养，以及将编码每种肌糖原聚糖物质，α，β，γ和δ的表达载体独立引入培养的细胞中，随后测定肌营养不良蛋白 - 糖蛋白复合物的恢复。另一方面，本发明涉及一种小鼠及其衍生的细胞，用于破坏的α-丝裂霉素基因是纯合的。该破坏阻止了小鼠细胞中功能性α-sarcoglycan的合成，并导致突变小鼠在骨骼肌和心脏肌肉的肌膜中不具有可检测的sarcospan，β - ，γ-，δ-色氨酸聚糖和还原的α-二糖聚糖，和与缺乏α-sarcoglycan基因的小鼠的组织相比，骨骼肌中营养不良蛋白的减少。另一方面，本发明涉及筛选用于治疗肌糖原缺乏型肢体肌营养不良症的治疗剂的方法。所述方法包括将候选治疗剂施用于小鼠或由其衍生的细胞，以及测定对小鼠或细胞的治疗效果，其中治疗效果的确定是疾病进展中的减少或逆转，或者肌萎缩糖复合物的恢复。

9. 发明申请

US20070148666A1 Diagnosis and Treatment of Brain Cancer 审中-公开
标题翻译：脑癌的诊断与治疗
公开(公告)号：US20070148666A1
公开(公告)日：2007-06-28
申请号：US11532496
申请日：2006-09-15
申请人： Rajeev Vibhakar , Anup Madam , Beverly Davidson
发明人： Rajeev Vibhakar , Anup Madam , Beverly Davidson
IPC分类号： C12Q1/68 , G01N33/574
CPC分类号： G01N33/57407 , C12Q1/6886 , C12Q2600/106 , C12Q2600/118 , C12Q2600/158
摘要： Certain genes are silenced in medulloblastoma. These genes include WIF1, sFRP1 and Dkk1. By detecting the expression of at least one of these genes, it is possible to diagnose cancer or to determine whether the cancer is recurring after treatment. Diagnostic methods, methods of treatment, and kits are provided.
摘要翻译：某些基因在成神经管细胞瘤中沉默。这些基因包括WIF1，sFRP1和Dkk1。通过检测这些基因中的至少一个的表达，可以诊断癌症或确定癌症是否在治疗后复发。提供诊断方法，治疗方法和试剂盒。

10. 发明申请

US20050112098A1 Pseudotyped viruses and methods for their use 审中-公开
标题翻译：伪型病毒及其使用方法
公开(公告)号：US20050112098A1
公开(公告)日：2005-05-26
申请号：US10811353
申请日：2004-03-26
申请人： Paul McCray , David Sanders , Scott Jeffers , Beverly Davidson , Patrick Sinn
发明人： Paul McCray , David Sanders , Scott Jeffers , Beverly Davidson , Patrick Sinn
IPC分类号： A61K38/16 , A61K48/00
CPC分类号： A61K38/1709 , A61K48/0008 , C12N2740/13045 , C12N2740/15045 , C12N2760/14222 , C12N2810/6072 , C12N2810/6081 , C12N2810/609
摘要： The invention provides a method to introduce a selected nucleic acid sequence into an airway epithelial cell that involves contacting the airway epithelial cell with a pseudotyped retrovirus that includes a glycoprotein in which a portion of an O-glycosylation region within the glycoprotein has been deleted and a retroviral capsid that includes the selected nucleic acid sequence. The invention also provides a method to reduce or eliminate the symptoms of cystic fibrosis in a mammal that involves contacting an airway epithelial cell of the mammal with a pseudotyped retrovirus that includes a glycoprotein in which a portion of an O-glycosylation region within the glycoprotein has been deleted and a retroviral capsid that includes a nucleic acid sequence that encodes a cystic fibrosis transmembrane regulator protein.
摘要翻译：本发明提供了将选择的核酸序列引入呼吸道上皮细胞的方法，所述方法包括将气道上皮细胞与假型逆转录病毒接触，所述假型逆转录病毒包括其中糖蛋白内的O-糖基化区域的一部分已被缺失的糖蛋白，包含选择的核酸序列的逆转录病毒衣壳。本发明还提供了一种减少或消除哺乳动物囊肿性纤维化症状的方法，该方法包括使哺乳动物的气道上皮细胞与假型逆转录病毒接触，所述假型逆转录病毒包括糖蛋白中的一部分O-糖基化区域内的糖蛋白以及包含编码囊性纤维化跨膜调节蛋白的核酸序列的逆转录病毒衣壳。

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