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1. 发明申请

WO2011133890A1 CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF 审中-公开
标题翻译： CNS目标AAV矢量及其使用方法
公开(公告)号：WO2011133890A1
公开(公告)日：2011-10-27
申请号：PCT/US2011/033616
申请日：2011-04-22
申请人： UNIVERSITY OF MASSACHUSETTS , GAO, Guangping , ZHANG, Gongwei , WANG, Hongyan , XU, Zuoshang
发明人： GAO, Guangping , ZHANG, Gongwei , WANG, Hongyan , XU, Zuoshang
IPC分类号： C12N15/00 , 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.
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。

2. 发明申请

WO2005007877A2 REGULATABLE PROMOTERS FOR SYNTHESIS OF SMALL HAIRPIN RNA 审中-公开
标题翻译：用于合成小毛发RNA的可调节促进剂
公开(公告)号：WO2005007877A2
公开(公告)日：2005-01-27
申请号：PCT/US2004/023789
申请日：2004-07-19
申请人： UNIVERSITY OF MASSACHUSETTS , XU, Zuoshang , XIA, Xugang
发明人： XU, Zuoshang , XIA, Xugang
IPC分类号： C12Q
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。

3. 发明申请

WO2012058220A2 ANTI-SOD1 ANTIBODIES AND USES THEREOF 审中-公开
标题翻译：抗SOD1抗体及其用途
公开(公告)号：WO2012058220A2
公开(公告)日：2012-05-03
申请号：PCT/US2011/057699
申请日：2011-10-25
申请人： UNIVERSITY OF MASSACHUSETTS , AMBROSINO, Donna , BABCOCK, Gregory , BROERING, Teresa , XU, Zuoshang
发明人： AMBROSINO, Donna , BABCOCK, Gregory , BROERING, Teresa , XU, Zuoshang
IPC分类号： C07K16/18 , C07K14/435 , C12N15/13 , A61K39/395 , A61K38/17 , A61P21/00
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-SODl 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ALS相关症状的方法。

4. 发明申请

WO2004042027A2 ALLELE-SPECIFIC RNA INTERFERENCE 审中-公开
标题翻译： ALLELE-SPECIFIC RNA干扰
公开(公告)号：WO2004042027A2
公开(公告)日：2004-05-21
申请号：PCT/US2003/035009
申请日：2003-11-04
申请人： UNIVERSITY OF MASSACHUSETTS , XU, Zuoshang , ZAMORE, Phillip, D.
发明人： XU, Zuoshang , ZAMORE, Phillip, D.
IPC分类号： C12N
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).
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。

5. 发明申请

WO2007044362A2 ALLELE-SPECIFIC RNA INTERFERENCE 审中-公开
标题翻译： ALLELE特异性RNA干扰
公开(公告)号：WO2007044362A2
公开(公告)日：2007-04-19
申请号：PCT/US2006/038704
申请日：2006-10-02
申请人： UNIVERSITY OF MASSACHUSETTS , XU, Zuoshang
发明人： XU, Zuoshang
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 (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产生显性，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asysmmetric siRNA和shRNA。

6. 发明申请

WO2005096781A3 METHODS AND COMPOSITIONS FOR TREATING GAIN-OF-FUNCTION DISORDERS USING RNA INTERFERENCE 审中-公开
公开(公告)号：WO2005096781A3
公开(公告)日：2005-10-20
申请号：PCT/US2005/011983
申请日：2005-04-06
申请人： UNIVERSITY OF MASSACHUSETTS , XU, Zuoshang , XIA, Xugang
发明人： XU, Zuoshang , XIA, Xugang
IPC分类号： C07H21/04 , A61K31/70
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.

7. 发明申请

WO2005007875A2 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：WO2005007875A2
公开(公告)日：2005-01-27
申请号：PCT/US2004/023215
申请日：2004-07-19
申请人： UNIVERSITY OF MASSACHUSETTS , XU, Zuoshang , XIA, Xugang
发明人： XU, Zuoshang , XIA, Xugang
IPC分类号： C12Q
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。

8. 发明申请

WO2017040813A2 DETECTION OF GENE LOCI WITH CRISPR ARRAYED REPEATS AND/OR POLYCHROMATIC SINGLE GUIDE RIBONUCLEIC ACIDS 审中-公开
标题翻译：用CRISPR ARRAYED REPEATS和/或POLYCHROMATIC单指导RIBONUCLEICID检测基因位点
公开(公告)号：WO2017040813A2
公开(公告)日：2017-03-09
申请号：PCT/US2016/049945
申请日：2016-09-01
申请人： UNIVERSITY OF 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
CPC分类号： C07H21/02 , C12N15/85 , C12N2830/003 , C12Q1/6816 , C12Q2521/507 , C12Q2563/107 , C12Q2563/119
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.
摘要翻译：可以使用CRISPR阵列重复检测系统（CARDS）检测C9orf72 DNA重复扩增。基于支持该平台的组合物和方法，可以在常规临床诊断实验室条件下测试原代细胞培养物和/或血细胞涂片，以诊断具有DNA重复扩增的遗传性疾病，包括但不限于ALS。也考虑到dCas9构建体具有与任何或所有茎环序列结合的荧光蛋白，其中检测多个具有不同着色荧光蛋白的dCas9构建体可同时检测至少六（6）个不同基因靶基因座。

9. 发明申请

WO2017040813A3 DETECTION OF GENE LOCI WITH CRISPR ARRAYED REPEATS AND/OR POLYCHROMATIC SINGLE GUIDE RIBONUCLEIC ACIDS 审中-公开
公开(公告)号：WO2017040813A3
公开(公告)日：2017-03-09
申请号：PCT/US2016/049945
申请日：2016-09-01
申请人： UNIVERSITY OF 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： C12N5/071 , C12N5/074 , C12N15/86 , C12N15/63 , 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.

10. 发明申请

WO2022192687A1 SYNTHETIC PROMOTERS FOR GENE THERAPY AND PROTEIN EXPRESSION 审中-公开
公开(公告)号：WO2022192687A1
公开(公告)日：2022-09-15
申请号：PCT/US2022/019976
申请日：2022-03-11
申请人： UNIVERSITY OF MASSACHUSETTS
发明人： WANG, Yang , KLEMPNER, Mark S. , EJEMEL, Monir , LI, Qi , XU, Zuoshang
IPC分类号： C12N15/09 , C12N15/11 , C12N15/113 , C12N15/63 , C12Q1/68
摘要： The invention provides synthetic promoters and methods of using the same.

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