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1. 发明公开

EP2561073A4 CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF 有权
标题翻译：对中枢神经系统靶向载体和使用方法
公开(公告)号：EP2561073A4
公开(公告)日：2014-01-08
申请号：EP11772784
申请日：2011-04-22
申请人： UNIV MASSACHUSETTS
发明人： GAO GUANGPING , ZHANG HONGWEI , WANG HONGYAN , XU ZUOSHANG
IPC分类号： C12N15/00 , A61K48/00 , C12N15/86
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.

2. 发明公开

EP1940862A4 ALLELE-SPECIFIC RNA INTERFERENCE 审中-公开
标题翻译： ALLELSPEZIFISCHE RNA-INTERFERENZ
公开(公告)号：EP1940862A4
公开(公告)日：2009-01-07
申请号：EP06836174
申请日：2006-10-02
申请人： UNIV MASSACHUSETTS
发明人： XU ZUOSHANG
IPC分类号： A61K38/00 , A61K48/00 , C12N5/02 , C12N15/11 , C12N15/113
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 (SODl), 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：由携带一个突变体和一个野生型基因拷贝的杂合子发生显性，功能获得性突变引起的人类疾病。由于野生型基因经常执行重要功能，而突变基因是有毒的，所以任何治疗策略都必须选择性抑制突变体，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保留共表达野生型等位基因的表达，所述治疗策略用于治疗与显性功能获得性基因突变相关的遗传疾病。本发明还包括选择性抑制引起遗传性肌萎缩性侧索硬化症（ALS）的铜锌超氧化物歧化酶（SOD1）的突变而非野生型表达的小干扰RNA（siRNA）和小发夹RNA（shRNA）。本发明进一步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。

3. 发明公开

EP2632952A4 ANTI-SOD1 ANTIBODIES AND USES THEREOF 审中-公开
标题翻译：抗体及其用途SOD1
公开(公告)号：EP2632952A4
公开(公告)日：2014-04-30
申请号：EP11836951
申请日：2011-10-25
申请人： UNIV MASSACHUSETTS
发明人： AMBROSINO DONNA , BABCOCK GREGORY , BROERING TERESA , XU ZUOSHANG
IPC分类号： C07K16/18 , A61K38/17 , A61K39/395 , A61P21/00 , C07K14/435 , C12N15/13
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

4. 发明公开

EP1567539A4 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：EP1567539A4
公开(公告)日：2006-07-26
申请号：EP03783121
申请日：2003-11-04
申请人： UNIV MASSACHUSETTS
发明人： XU ZUOSHANG , ZAMORE PHILLIP D
IPC分类号： A61K38/00 , A61K48/00 , C12N5/02 , C12N15/113 , C07H21/04 , C12N15/00 , C12N15/11
CPC分类号： C12N15/1137 , A61K38/00 , A61K48/00 , C12N2310/111 , C12N2310/14 , C12N2310/53 , 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 wildtype 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).

5. 发明申请

WO2005007875A3 ENHANCED PROMOTERS FOR SYNTHESIS OF SMALL HAIRPIN RNA 审中-公开
标题翻译：用于合成小毛发RNA的增强促进剂
公开(公告)号：WO2005007875A3
公开(公告)日：2008-04-10
申请号：PCT/US2004023215
申请日：2004-07-19
申请人： UNIV MASSACHUSETTS , XU ZUOSHANG , XIA XUGANG
发明人： XU ZUOSHANG , XIA XUGANG
IPC分类号： A61K31/70 , C07H21/02 , C07H21/04 , C12N5/00 , C12N9/22 , C12N15/11 , C12Q20060101 , C12Q1/68 , G01N33/48 , G01N33/50 , G06F19/00
CPC分类号： C12N15/111 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2330/30
摘要： The present invention provides compositions for RNA interference and methods of use thereof. In particular, the invention provides small hairpin RNAs (shRNAs) having modified promoters, including the Pol III U6 promoter, which may be used to increase the potency of shRNA by increasing the expression level. Modifications include constructs with a Pol II enhancer, such as the cytomegalovirus (CMV) enhancer, immediate-early promoter near the Pol III, e.g., U6 promoter, either upstream or downstream from the shRNA sequence and in either forward or backward orientation. Such constructs are useful for increasing the expression of the shRNA, thereby enhancing inhibition of a single nucleotide mismatched mutant allele. 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。特别地，本发明提供具有修饰的启动子的小发夹RNA（shRNA），其包括Pol III U6启动子，其可用于通过增加表达水平来增加shRNA的效力。修饰包括具有Pol II增强子的构建体，例如巨细胞病毒（CMV）增强子，在Pol III附近的即时早期启动子，例如U6启动子，位于shRNA序列的上游或下游以及向前或向后的方向。这种构建体可用于增加shRNA的表达，从而增强对单核苷酸错配突变体等位基因的抑制。功能和基因组和蛋白质组学方法。还介绍了治疗方法。

6. 发明专利

ES2605305T3 Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos 未知
公开(公告)号：ES2605305T3
公开(公告)日：2017-03-13
申请号：ES11772784
申请日：2011-04-22
申请人： UNIV MASSACHUSETTS
发明人： GAO GUANGPING , ZHANG HONGWEI , WANG HONGYAN , XU ZUOSHANG
IPC分类号： C12N15/00 , A61K48/00 , C12N15/86
摘要： Un rAAV que comprende (i) una proteína de la cápside que comprende la secuencia como se expone en SEQ ID NO: 9 y (ii) un ácido nucleico que comprende un promotor operativamente unido con un transgén para su uso en el tratamiento de esclerosis lateral amiotrófica (ELA); en el que el rAAV va a administrarse a tejido del sistema nervioso central (SNC) de un sujeto por administración intratecal, y opcionalmente también se administra por administración intracerebral.

7. 发明专利

DK2561073T3 AAV-VEKTORER MÅLRETTET MOD CENTRALNERVESYSTEMET OG FREMGANGSMÅDER TIL ANVENDELSE DERAF 未知
公开(公告)号：DK2561073T3
公开(公告)日：2016-12-12
申请号：DK11772784
申请日：2011-04-22
申请人： UNIV MASSACHUSETTS
发明人： GAO GUANGPING , WANG HONGYAN , XU ZUOSHANG , ZHANG HONGWEI
IPC分类号： C12N15/00 , A61K48/00 , C12N15/86

8. 发明专利

CA3035810A1 DETECTION OF GENE LOCI WITH CRISPR ARRAYED REPEATS AND/OR POLYCHROMATIC SINGLE GUIDE RIBONUCLEIC ACIDS 未知
公开(公告)号：CA3035810A1
公开(公告)日：2017-03-09
申请号：CA3035810
申请日：2016-09-01
申请人： UNIV MASSACHUSETTS
发明人： PEDERSON THORU , MA HANHUI , LI-CHUNG TU , NASERI ARDALAN , XU ZUOSHANG , HUISMAN MAXIMILAAN , YANG CHUNXING , ZHANG SHAOJIE
IPC分类号： A61K48/00 , C07H21/02 , C12N5/074 , C12N15/63 , C12N15/86 , C12Q1/68
摘要： A C9orf72 DNA repeat expansion can be detected using a CRISPR Arrayed Repeat Detection System (CARDS). Based upon the compositions and methods supporting this platform primary cell cultures and/or blood cell smears can be tested under conventional clinical diagnostic laboratory conditions to diagnose genetically-based diseases having DNA repeat expansions, including but not limited to ALS. dCas9 constructs are also contemplated as having fluorescent proteins bound to any or all stem loop sequences, wherein detection of a plurality of dCas9 constructs having different colored fluorescent proteins can simultaneously detect at least six (6) different gene target loci.

9. 发明专利

CA2819117A1 ANTI-SOD1 ANTIBODIES AND USES THEREOF 未知
公开(公告)号：CA2819117A1
公开(公告)日：2012-05-03
申请号：CA2819117
申请日：2011-10-25
申请人： UNIV MASSACHUSETTS
发明人： AMBROSINO DONNA , BABCOCK GREGORY , BROERING TERESA , XU ZUOSHANG
IPC分类号： C07K16/18 , A61K38/17 , A61K39/395 , A61P21/00 , C07K14/435 , C12N15/13
摘要： The present invention features anti-SODl antibodies and methods of using the antibodies for the treatment of amyotrophic lateral sclerosis (ALS) or the amelioration of symptoms associated with ALS.

10. 发明专利

CA2624673A1 ALLELE-SPECIFIC RNA INTERFERENCE 未知
公开(公告)号：CA2624673A1
公开(公告)日：2007-04-19
申请号：CA2624673
申请日：2006-10-02
申请人： UNIV MASSACHUSETTS
发明人： XU ZUOSHANG
IPC分类号： C07H21/04 , A61K38/00 , A61K48/00 , C12N5/02 , C12N15/11 , C12N15/113
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 (SODl), which causes inherited amyotrophic lateral sclerosis (ALS). The present invention further provides asysmmetric siRNAs and shRNAs with enhanced efficacy and specificity and mediating RNAi.

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