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1. 发明授权

US09109037B2 Anti-SOD1 antibodies and uses thereof 有权
标题翻译：抗SOD1抗体及其用途
公开(公告)号：US09109037B2
公开(公告)日：2015-08-18
申请号：US13882017
申请日：2011-10-25
申请人： Donna Ambrosino , Gregory Babcock , Teresa Broering , Zuoshang Xu
发明人： Donna Ambrosino , Gregory Babcock , Teresa Broering , Zuoshang Xu
IPC分类号： A61K39/395 , C07K16/40 , A61K45/06 , A61K39/00
CPC分类号： C07K16/40 , A61K31/428 , A61K39/3955 , A61K45/06 , A61K47/6871 , A61K2039/505 , C07K2317/21 , C07K2317/34 , C07K2317/565 , C07K2317/76 , C07K2317/92
摘要： The present invention features anti-SOD1 antibodies and methods of using the antibodies for the treatment of amyotrophic lateral sclerosis (ALS) or the amelioration of symptoms associated with ALS.
摘要翻译：本发明的特征在于抗SOD1抗体和使用该抗体治疗肌萎缩性侧索硬化（ALS）或改善与ALS相关的症状的方法。

2. 发明申请

US20140044722A1 ANTI-SOD1 ANTIBODIES AND USES THEREOF 有权
标题翻译：抗SOD1抗体及其用途
公开(公告)号：US20140044722A1
公开(公告)日：2014-02-13
申请号：US13882017
申请日：2011-10-25
申请人： Donna Ambrosino , Gregory Babcock , Teresa Broering , Zuoshang Xu
发明人： Donna Ambrosino , Gregory Babcock , Teresa Broering , Zuoshang Xu
IPC分类号： C07K16/40 , A61K45/06 , A61K39/395
CPC分类号： C07K16/40 , A61K31/428 , A61K39/3955 , A61K45/06 , A61K47/6871 , A61K2039/505 , C07K2317/21 , C07K2317/34 , C07K2317/565 , C07K2317/76 , C07K2317/92
摘要： The present invention features anti-SOD1 antibodies and methods of using the antibodies for the treatment of amyotrophic lateral sclerosis (ALS) or the amelioration of symptoms associated with ALS.
摘要翻译：本发明的特征在于抗SOD1抗体和使用该抗体治疗肌萎缩性侧索硬化（ALS）或改善与ALS相关的症状的方法。

3. 发明授权

US08309533B2 Allele-specific RNA interference 有权
标题翻译：等位基因特异性RNA干扰
公开(公告)号：US08309533B2
公开(公告)日：2012-11-13
申请号：US13031534
申请日：2011-02-21
申请人： Zuoshang Xu
发明人： Zuoshang Xu
IPC分类号： A61K31/70 , C07H21/02 , C07H21/04 , C12Q1/68
CPC分类号： C12N15/1137 , A61K38/00 , A61K48/00 , C12N15/111 , C12N2310/111 , C12N2310/14 , C12N2310/333 , C12N2310/336 , C12N2310/53 , C12N2320/51 , C12Y115/01001
摘要： Human diseases caused by dominant, gain-of-function mutations develop in heterozygotes bearing one mutant and one wild-type copy of a gene. Because the wild-type gene often performs important functions, whereas the mutant gene is toxic, any therapeutic strategy must selectively inhibit the mutant while retaining wild-type gene expression. The present invention includes methods of specifically inhibiting the expression of a mutant allele, while preserving the expression of a co-expressed wild-type allele using RNAi, a therapeutic strategy for treating genetic disorders associated with dominant, gain-of-function gene mutations. The invention also includes small interfering RNAs (siRNAs) and small hairpin RNAs (shRNAs) that selectively suppress mutant, but not wild-type, expression of copper zinc superoxide dismutase (SOD1), which causes inherited amyotrophic lateral sclerosis (ALS). The present invention further provides asymmetric siRNAs and shRNAs with enhanced efficacy and specificity and mediating RNAi.
摘要翻译：在具有一个基因的一个突变体和一个野生型拷贝的杂合子中，由显性增益功能突变引起的人类疾病发生。因为野生型基因通常具有重要的功能，而突变基因是有毒的，任何治疗策略必须选择性地抑制突变体，同时保留野生型基因表达。本发明包括特异性抑制突变等位基因表达的方法，同时使用RNAi保护共表达野生型等位基因的表达，用于治疗与显性功能性获得功能基因突变相关的遗传疾病的治疗策略。本发明还包括小干扰RNA（siRNA）和小发夹RNA（shRNA），其选择性地抑制引起遗传性肌萎缩性侧索硬化（ALS）的铜锌超氧化物歧化酶（SOD1）的突变体而不是野生型表达。本发明还提供了具有增强的功效和特异性并介导RNAi的不对称siRNA和shRNA。

4. 发明授权

US07498316B2 Methods and compositions for treating gain-of-function disorders using RNA interference 有权
标题翻译：使用RNA干扰治疗功能障碍功能障碍的方法和组合物
公开(公告)号：US07498316B2
公开(公告)日：2009-03-03
申请号：US11101162
申请日：2005-04-06
申请人： Zuoshang Xu , Xugang Xia
发明人： Zuoshang Xu , Xugang Xia
IPC分类号： A61K31/70 , C07H21/04 , C12Q1/68
CPC分类号： C12N15/1137 , C12N9/0089 , C12N15/111 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/31 , C12Y115/01001
摘要： The present invention relates to novel methods for treating dominant gain-of-function diseases. The invention provides methods for targeting regions of the copper zinc superoxide dismutase (SOD1), which causes inherited amyotrophic lateral sclerosis (ALS), with RNAi agent. The invention further provides RNAi resistant replacement genes containing mismatches with their respective RNAi agent s. The invention also provides for vectors that express RNAi agent and RNAi resistant replacement gene of the present invention.
摘要翻译：本发明涉及治疗主要功能障碍性疾病的新方法。本发明提供用RNAi试剂靶向导致遗传性肌萎缩性侧索硬化（ALS）的铜锌超氧化物歧化酶（SOD1）区域的方法。本发明还提供了RNAi抗性替代基因，其含有与它们各自的RNAi试剂的失配。本发明还提供了表达本发明的RNAi剂和RNAi抗性替代基因的载体。

5. 发明授权

US09102949B2 CNS targeting AAV vectors and methods of use thereof 有权
标题翻译：针对AAV载体的CNS及其使用方法
公开(公告)号：US09102949B2
公开(公告)日：2015-08-11
申请号：US13642719
申请日：2011-04-22
申请人： Guangping Gao , Hongwei Zhang , Hongyan Wang , Zuoshang Xu
发明人： Guangping Gao , Hongwei Zhang , Hongyan Wang , Zuoshang Xu
IPC分类号： C12N15/86 , C12N15/864 , C12N15/113 , A61K38/50 , A61K31/713 , C12N9/80 , C12N7/00 , A61K48/00 , C12N15/63
CPC分类号： C12N15/86 , A61K31/713 , A61K38/50 , A61K48/00 , A61K48/0058 , A61K48/0075 , C12N7/00 , C12N9/80 , C12N15/1137 , C12N15/635 , C12N15/8645 , C12N2310/141 , C12N2750/14133 , C12N2750/14141 , C12N2750/14143 , C12N2750/14145 , C12N2750/14162 , C12N2810/10 , C12N2840/007 , C12Y305/01015
摘要： The invention in some aspects relates to recombinant adeno-associated viruses useful for targeting transgenes to CNS tissue, and compositions comprising the same, and methods of use thereof. In some aspects, the invention provides methods and compositions for treating CNS-related disorders.
摘要翻译：本发明在一些方面涉及可用于靶向转基因到CNS组织的重组腺相关病毒，以及包含其的组合物及其使用方法。在一些方面，本发明提供了用于治疗CNS相关疾病的方法和组合物。

6. 发明申请

US20060128650A1 Allele-specific RNA interference 有权
标题翻译：等位基因特异性RNA干扰
公开(公告)号：US20060128650A1
公开(公告)日：2006-06-15
申请号：US11241873
申请日：2005-09-30
申请人： Zuoshang Xu
发明人： Zuoshang Xu
IPC分类号： A61K48/00 , C07H21/02 , C07H21/04
CPC分类号： C12N15/1137 , A61K38/00 , A61K48/00 , C12N15/111 , C12N2310/111 , C12N2310/14 , C12N2310/333 , C12N2310/336 , C12N2310/53 , C12N2320/51 , C12Y115/01001
摘要： Human diseases caused by dominant, gain-of-function mutations develop in heterozygotes bearing one mutant and one wild-type copy of a gene. Because the wild-type gene often performs important functions, whereas the mutant gene is toxic, any therapeutic strategy must selectively inhibit the mutant while retaining wild-type gene expression. The present invention includes methods of specifically inhibiting the expression of a mutant allele, while preserving the expression of a co-expressed wild-type allele using RNAi, a therapeutic strategy for treating genetic disorders associated with dominant, gain-of-function gene mutations. The invention also includes small interfering RNAs (siRNAs) and small hairpin RNAs (shRNAs) that selectively suppress mutant, but not wild-type, expression of copper zinc superoxide dismutase (SOD1), which causes inherited amyotrophic lateral sclerosis (ALS). The present invention further provides asysmmetric siRNAs and shRNAs with enhanced efficacy and specificity and mediating RNAi.
摘要翻译：在具有一个基因的一个突变体和一个野生型拷贝的杂合子中，由显性增益功能突变引起的人类疾病发生。因为野生型基因通常具有重要的功能，而突变基因是有毒的，任何治疗策略必须选择性地抑制突变体，同时保留野生型基因表达。本发明包括特异性抑制突变等位基因表达的方法，同时使用RNAi保护共表达野生型等位基因的表达，用于治疗与显性功能性获得功能基因突变相关的遗传疾病的治疗策略。本发明还包括小干扰RNA（siRNA）和小发夹RNA（shRNA），其选择性地抑制引起遗传性肌萎缩性侧索硬化（ALS）的铜锌超氧化物歧化酶（SOD1）的突变体而不是野生型表达。本发明还提供了具有增强的功效和特异性并介导RNAi的不对称siRNA和shRNA。

7. 发明申请

US20050130919A1 Regulatable promoters for synthesis of small hairpin RNA 审中-公开
标题翻译：用于合成小发夹RNA的可调节启动子
公开(公告)号：US20050130919A1
公开(公告)日：2005-06-16
申请号：US10894949
申请日：2004-07-19
申请人： Zuoshang Xu , Xugang Xia
发明人： Zuoshang Xu , Xugang Xia
IPC分类号： A01K67/027 , A61K48/00 , C07H21/02 , C07H21/04 , C07K14/47 , C12N7/00 , C12N15/09 , C12N15/11 , C12N15/63 , C12N15/85 , C12N15/86 , C12P19/34 , C12Q20060101
CPC分类号： C12N15/111 , A01K2217/058 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/50 , C12N2330/30 , C12N2800/30 , C12N2830/003
摘要： The present invention provides compositions for RNA interference and methods of use thereof. The present invention is based on the development of promoters that can be used to regulate shRNA expression spatially (in specific cells) and temporally (at specific times) in cells or transgenic animals that express a recombinase. The compositions and methods of the present invention feature regulatable promoters that allow for inhibition of the expression of target alleles in a spatially and temporally regulatable manner. Thus, the compositions of the present invention are useful for investigating gene functions, both physiologic and pathologic, in specific cell groups and in specific ages, in normal and disease pathways. Functional and genomic and proteomic methods are featured. Therapeutic methods are also featured.
摘要翻译：本发明提供了用于RNA干扰的组合物及其使用方法。本发明是基于开发可以用于在表达重组酶的细胞或转基因动物中空间（特定细胞）和时间（特定时间）调节shRNA表达的启动子的开发。本发明的组合物和方法具有可调节的启动子，其允许以空间和时间上可调节的方式抑制靶基因的表达。因此，本发明的组合物可用于在正常和疾病途径中研究特定细胞组和特定年龄的生理和病理学基因功能。功能和基因组和蛋白质组学方法。还介绍了治疗方法。

8. 发明授权

US08008271B2 Methods and compositions for treating gain-of-function disorders using RNA interference 有权
标题翻译：使用RNA干扰治疗功能障碍功能障碍的方法和组合物
公开(公告)号：US08008271B2
公开(公告)日：2011-08-30
申请号：US12175369
申请日：2008-07-17
申请人： Zuoshang Xu , Xugang Xia
发明人： Zuoshang Xu , Xugang Xia
IPC分类号： A61K31/70 , C07H21/04
CPC分类号： C12N15/1137 , C12N9/0089 , C12N15/111 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/31 , C12Y115/01001
摘要： The present invention relates to novel methods for treating dominant gain-of-function diseases. The invention provides methods for targeting regions of the copper zinc superoxide dismutase (SOD1), which causes inherited amyotrophic lateral sclerosis (ALS), with RNAi agent. The invention further provides RNAi resistant replacement genes containing mismatches with their respective RNAi agents. The invention also provides for vectors that express RNAi agent and RNAi resistant replacement gene of the present invention.
摘要翻译：本发明涉及治疗主要功能障碍性疾病的新方法。本发明提供用RNAi试剂靶向导致遗传性肌萎缩性侧索硬化（ALS）的铜锌超氧化物歧化酶（SOD1）区域的方法。本发明还提供了含有与其各自的RNAi试剂不匹配的RNAi抗性替代基因。本发明还提供了表达本发明的RNAi剂和RNAi抗性替代基因的载体。

9. 发明授权

US07951784B2 RNA interference agents for therapeutic use 有权
标题翻译：用于治疗用途的RNA干扰剂
公开(公告)号：US07951784B2
公开(公告)日：2011-05-31
申请号：US11698785
申请日：2007-01-26
申请人： Tariq M. Rana , Zuoshang Xu
发明人： Tariq M. Rana , Zuoshang Xu
IPC分类号： A61K31/70 , C07H21/04
CPC分类号： C12N15/87 , A61K9/0019 , A61K9/5146 , A61K47/34 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2320/32 , Y02A50/465
摘要： The invention features chemically modified small interfering RNAs (siRNAs) that are stable in vivo and retain the ability to form an A-form helix when in association with a target RNA. The features siRNA are effective therapeutics, particularly for targeting SOD1.
摘要翻译：本发明的特征在于在体内稳定的化学修饰的小干扰RNA（siRNA），并且当与靶RNA结合时保留形成A-型螺旋的能力。特征siRNA是有效的治疗剂，特别是用于靶向SOD1。

10. 发明授权

US07892793B2 Allele-specific RNA interference 有权
标题翻译：等位基因特异性RNA干扰
公开(公告)号：US07892793B2
公开(公告)日：2011-02-22
申请号：US11241873
申请日：2005-09-30
申请人： Zuoshang Xu
发明人： Zuoshang Xu
IPC分类号： C07H21/02 , C07H21/04 , A61K31/70 , C12Q1/68
CPC分类号： C12N15/1137 , A61K38/00 , A61K48/00 , C12N15/111 , C12N2310/111 , C12N2310/14 , C12N2310/333 , C12N2310/336 , C12N2310/53 , C12N2320/51 , C12Y115/01001
摘要： Human diseases caused by dominant, gain-of-function mutations develop in heterozygotes bearing one mutant and one wild-type copy of a gene. Because the wild-type gene often performs important functions, whereas the mutant gene is toxic, any therapeutic strategy must selectively inhibit the mutant while retaining wild-type gene expression. The present invention includes methods of specifically inhibiting the expression of a mutant allele, while preserving the expression of a co-expressed wild-type allele using RNAi, a therapeutic strategy for treating genetic disorders associated with dominant, gain-of-function gene mutations. The invention also includes small interfering RNAs (siRNAs) and small hairpin RNAs (shRNAs) that selectively suppress mutant, but not wild-type, expression of copper zinc superoxide dismutase (SOD1), which causes inherited amyotrophic lateral sclerosis (ALS). The present invention further provides asysmmetric siRNAs and shRNAs with enhanced efficacy and specificity and mediating RNAi.
摘要翻译：在具有一个基因的一个突变体和一个野生型拷贝的杂合子中，由显性增益功能突变引起的人类疾病发生。因为野生型基因通常具有重要的功能，而突变基因是有毒的，任何治疗策略必须选择性地抑制突变体，同时保留野生型基因表达。本发明包括特异性抑制突变等位基因表达的方法，同时使用RNAi保护共表达野生型等位基因的表达，用于治疗与显性功能性获得功能基因突变相关的遗传疾病的治疗策略。本发明还包括小干扰RNA（siRNA）和小发夹RNA（shRNA），其选择性地抑制引起遗传性肌萎缩性侧索硬化（ALS）的铜锌超氧化物歧化酶（SOD1）的突变体而不是野生型表达。本发明还提供了具有增强的功效和特异性并介导RNAi的不对称siRNA和shRNA。

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