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1. 发明申请

WO2019126578A1 COMPOSITIONS AND METHODS FOR DIRECTING PROTEINS TO SPECIFIC LOCI IN THE GENOME 审中-公开
公开(公告)号：WO2019126578A1
公开(公告)日：2019-06-27
申请号：PCT/US2018/066941
申请日：2018-12-20
申请人： POSEIDA THERAPEUTICS, INC. , LI, Xianghong , YESHI, Tseten , OSTERTAG, Eric
发明人： LI, Xianghong , YESHI, Tseten , OSTERTAG, Eric
IPC分类号： C12N9/16 , C12N9/22 , C12N15/11 , C12N15/62 , C12N5/04 , C12N5/07 , A61K48/00
CPC分类号： C12N9/16 , C07K2319/80 , C12N9/22 , C12N15/62 , C12N2310/20 , C12Y301/21004
摘要： Disclosed are compositions and methods for directing proteins to specific loci in the genome and uses thereof. In one aspect, the disclosed methods allow for directing proteins to specific loci in the genome of an organism, including the steps of providing a fusion protein comprising a DNA localization component and an effector molecule. Preferred embodiments of the disclosure include, but are not limited to, the following fusion proteins: dSaCas9-Clo051, dCas9-Clo051, Xanthomonas-TALE-Clo051, and Ralstonia-TALE-Clo051.

2. 发明申请

WO2018212341A1 表皮肥厚性疾患及び／又は表皮肥厚性症状に対する予防、改善、又は治療剤审中-公开
公开(公告)号：WO2018212341A1
公开(公告)日：2018-11-22
申请号：PCT/JP2018/019345
申请日：2018-05-18
申请人：国立大学法人徳島大学 , 公益財団法人東京都医学総合研究所
发明人：山本　圭 , 村上　誠 , 酒瀬川　信一
IPC分类号： A61K38/46 , A61K31/7088 , A61K35/74 , A61K48/00 , A61P17/00 , A61P17/02 , A61P17/06 , A61P35/00 , A61P37/08 , C12N9/16 , C12N15/55 , C12N15/70
CPC分类号： A61K31/7088 , A61K35/74 , A61K38/46 , A61K48/00 , A61P17/00 , A61P17/02 , A61P17/06 , A61P35/00 , A61P37/08 , C12N9/16 , C12N15/70
摘要：表皮肥厚性疾患及び／又は表皮肥厚性症状に対する予防、改善、又は治療効果を有する有効成分を提供することを課題として、ホスホリパーゼD及びその発現カセットからなる群より選択される少なくとも1種を含有する、表皮肥厚性疾患及び／又は表皮肥厚性症状に対する予防、改善、又は治療剤により解決する。

3. 发明申请

WO2018200323A1 SMALL MOLECULE REGULATORS OF MITOCHONDRIAL FUSION AND METHODS OF USE THEREOF 审中-公开
公开(公告)号：WO2018200323A1
公开(公告)日：2018-11-01
申请号：PCT/US2018/028514
申请日：2018-04-20
申请人： WASHINGTON UNIVERSITY
发明人： DORN, II, Gerald W.
IPC分类号： A61K31/16 , C07K14/47 , C12N15/12
CPC分类号： C07D249/12 , A61K31/4196 , A61K31/454 , A61P25/28 , C07D405/12 , C12N9/16
摘要： Compositions comprising small molecule mitofusin agonists are described. The mitofusin modulating agents are useful for treating diseases or disorders associated with a mitochondria-associated disease, disorder, or condition such as diseases or disorders associated with mitofusin 1 (Mfn1) and/or mitofusin 2 (Mfn2), or mitochondrial dysfunction. Methods of treatment, pharmaceutical formulations, and screening methods for identifying compounds that regulate mitochondrial function are also described.

4. 发明申请

WO2018055211A1 NOVEL COMPOUNDS AND METHODS FOR MODULATING UBIQUITINATION 审中-公开
标题翻译：新型化合物和调节泛滥的方法
公开(公告)号：WO2018055211A1
公开(公告)日：2018-03-29
申请号：PCT/EP2017/074410
申请日：2017-09-26
申请人： JOHANN WOLFGANG GOETHE-UNIVERSITÄT FRANKFURT AM MAIN
发明人： BHOGARAJU, Sagar , DIKIC, Ivan
IPC分类号： C12N15/09 , A61K45/00 , C12N9/00
CPC分类号： C12N9/16 , A61K45/00 , C12N9/1077
摘要： The present invention pertains to the modification of the eukaryotic ubiquitin system by using the bacterial virulence factor SdeA and variants and mutants thereof. The invention is based on the phosphodiesterase activity of SdeA, which catalyzes a phospho-ribosylation (or simply "ribosylation") event of ubiquitin and ubiquitin-like proteins. The invention provides SdeA proteins and mutants without said phosphodiesterase activity, as well as medical applications of these recombinant proteins, their encoding nucleic acids, ribosylated ubiquitin proteins, and compounds which may act as selective or non-selective phosphodiesterase inhibitors.
摘要翻译：本发明涉及通过使用细菌毒力因子SdeA及其变体和突变体来修饰真核生物泛素系统。本发明基于SdeA的磷酸二酯酶活性，其催化遍在蛋白和遍在蛋白样蛋白的磷酸核糖基化（或简称“核糖基化”）事件。本发明提供了不具有所述磷酸二酯酶活性的SdeA蛋白质和突变体，以及这些重组蛋白质，它们的编码核酸，核糖基化泛素蛋白质以及可以用作选择性或非选择性磷酸二酯酶抑制剂的化合物的医学应用。

5. 发明申请

WO2018031636A1 METHODS OF DETECTING MLH1 METHYLATION 审中-公开
标题翻译：检测MLH1甲基化的方法
公开(公告)号：WO2018031636A1
公开(公告)日：2018-02-15
申请号：PCT/US2017/046070
申请日：2017-08-09
申请人： QUEST DIAGNOSTICS INVESTMENTS LLC
发明人： QU, Kevin , HANTASH, Feras , DONAHUE, Amber
IPC分类号： C12Q1/68 , C12N9/16
CPC分类号： C12Q1/6886 , C07K14/82 , C12N9/16 , C12Q1/6816 , C12Q1/6827 , C12Q2600/112 , C12Q2600/154 , C12Q2600/156 , C12Q2600/16
摘要： The present technology relates to methods for excluding Lynch syndrome as a possible diagnosis in patients suffering from colorectal cancers or endometrial cancers. These methods are based on detecting the methylation status of the MLH1 promoter 'C , region in colorectal and endometrial cancer patients using an improved and highly sensitive methylation-specific multiplex ligation-dependent probe amplification (MS-MLPA) assay.
摘要翻译：本技术涉及在患有结直肠癌或子宫内膜癌的患者中排除Lynch综合征作为可能诊断的方法。这些方法基于使用改进的和高度敏感的甲基化特异性多重连接依赖性检测结直肠癌和子宫内膜癌患者中MLH1启动子C ^{区的甲基化状态探针扩增（MS-MLPA）测定。}

6. 发明申请

WO2017215218A1 一种植酸酶突变体及其制备方法和应用审中-公开
公开(公告)号：WO2017215218A1
公开(公告)日：2017-12-21
申请号：PCT/CN2016/110231
申请日：2016-12-16
申请人：昆明爱科特生物科技有限公司
发明人：黄遵锡 , 苗华彪 , 韩楠玉
IPC分类号： C12N9/16 , C12N15/55 , C12N15/81 , A23K20/189
CPC分类号： A23K20/189 , C12N9/16 , C12N15/81
摘要：一种植酸酶突变体及其制备方法和应用，植酸酶突变体的序列SEQ ID No：1，该突变体由编码无花果曲霉植酸酶氨基酸序列发生改变而获得；使用烟曲霉植酸酶的部分氨基酸序列来替代无花果曲霉植酸酶氨基酸序列，使其成为耐热性高的植酸酶；以无花果曲霉植酸酶基因序列作为模版，通过基因点突变技术使无花果植酸酶基因发生突变，获得了新的植酸酶基因的突变体SEQ ID NO：2，该植酸酶基因的突变体与质粒PPIC9K等相连构建重复质粒，转化宿主GS115等获得基因工程菌株，通过基因工程菌株发酵，获得新的植酸酶突变体；能有较宽的pH作用范围和较理想耐热特性，适合耐高温制粒，较好地满足饲料工业的要求。

7. 发明申请

WO2017201425A1 ANABOLIC ENHANCERS FOR AMELIORATING NEURODEGENERATION 审中-公开
标题翻译：用于改善神经退化的抗疲劳增强剂
公开(公告)号：WO2017201425A1
公开(公告)日：2017-11-23
申请号：PCT/US2017/033575
申请日：2017-05-19
申请人： THE TRUSTEES COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK
发明人： TSANG, Stephen
IPC分类号： A61K48/00 , C12N15/63 , C12N15/85
CPC分类号： C12N15/113 , A61K9/0048 , A61K31/7088 , A61K31/713 , A61K38/465 , A61K45/06 , A61K48/00 , A61P25/28 , A61P27/02 , C07K14/47 , C12N7/00 , C12N9/1077 , C12N9/16 , C12N9/22 , C12N15/11 , C12N15/1137 , C12N15/86 , C12N2310/111 , C12N2310/122 , C12N2310/14 , C12N2310/20 , C12N2310/531 , C12N2320/32 , C12N2750/14143 , C12N2750/14171 , C12N2800/80
摘要： The present disclosure relates to methods and compounds for promoting anabolic pathways in neuronal cells leading to improved neuronal survival. In particular, the present disclosure relates to inhibiting TSCI and or SIRT6 to promote glycolysis and neuronal survival in a variety of neurodegenerative conditions, and specifically in retinitis pigmentosa.
摘要翻译：本公开涉及用于促进神经元细胞中的合成代谢途径以提高神经元存活的方法和化合物。具体而言，本公开涉及抑制TSCI和/或SIRT6以促进多种神经变性病症中的糖酵解和神经元存活，并且特别是在视网膜色素变性中。

8. 发明申请

WO2017192925A1 IMPROVED MICROBIAL PRODUCTION OF FATS 审中-公开
标题翻译：改进的FATS微生物生产
公开(公告)号：WO2017192925A1
公开(公告)日：2017-11-09
申请号：PCT/US2017/031174
申请日：2017-05-05
申请人： WILLIAM MARSH RICE UNIVERSITY
发明人： SAN, Ka-Yiu , LI, Zhilin , ZHANG, Xian
IPC分类号： C12P7/64 , C12P7/40 , C12N15/74 , C12N1/21
CPC分类号： C12P7/6409 , C12N1/20 , C12N9/0006 , C12N9/0008 , C12N9/16 , C12N9/88 , C12N15/52 , C12N15/74 , C12Y102/01051 , C12Y102/07001
摘要： This invention describes a method of using microbial to produce fats, such as fatty acids and their derivatives, or products derived from the fatty acid synthesis cycle, such as hydroxyfatty acids, methyl ketones, and the like.
摘要翻译：本发明描述了使用微生物来生产脂肪（例如脂肪酸及其衍生物）或衍生自脂肪酸合成循环的产品（例如羟基脂肪酸，甲基酮等）的方法。

9. 发明申请

WO2017132420A1 COMPOSITIONS AND METHODS FOR SUPPRESSION AND DIAGNOSIS OF CANCER METASTASIS 审中-公开
标题翻译：癌症转移的抑制和诊断的组合物和方法
公开(公告)号：WO2017132420A1
公开(公告)日：2017-08-03
申请号：PCT/US2017/015198
申请日：2017-01-26
申请人： THE METHODIST HOSPITAL
发明人： LI, Xian Chang , FAN, Yihui
IPC分类号： A61K38/17 , A61K48/00 , A61K9/127 , A61K9/51
CPC分类号： A61K38/465 , A01K2207/12 , A01K2227/105 , A01K2267/0331 , C12N9/16 , C12N9/22
摘要： Disclosed are methods for determining the metastatic potential of mammalian tumors and other cancers. Also disclosed are methods for altering the metastatic potential of mammalian cancers, by a) directly or indirectly increasing the level of Regnase peptide or polypeptide; b) selectively increasing the activity or specificity of Regnase peptide or polypeptide; c) selectively increasing the amount or stability of Regnase-specific mRNA; or d) selectively inhibiting one or more negative effectors of Regnase within a cancer cell in an amount and for a time effective to reduce, delay, retard, or prevent subsequent metastasis of the cancer within the body of a mammal. Also disclosed are methods for predicting cancer cell metastasis, and methods for determining the likelihood of patient outcomes by quantitating Regnase-specific expression or activity thereby providing a biomarker for determining the metastatic potential of a given cancer.
摘要翻译：公开了用于确定哺乳动物肿瘤和其他癌症的转移潜能的方法。还公开了通过以下方法来改变哺乳动物癌症的转移潜能的方法：a）直接或间接增加雷珠朊酶肽或多肽的水平; b）选择性地增加Regnase肽或多肽的活性或特异性; c）选择性增加Regnase特异性mRNA的量或稳定性; 或d）选择性抑制癌细胞内Regnase的一种或多种负效应物，其量和时间有效减少，延迟，延迟或预防哺乳动物体内癌症的后续转移。还公开了用于预测癌细胞转移的方法，以及通过定量Regnase特异性表达或活性来确定患者结果的可能性的方法，从而提供用于确定给定癌症的转移潜能的生物标志物。

10. 发明申请

WO2017106528A3 TARGETED DISRUPTION OF THE T CELL RECEPTOR 审中-公开
标题翻译：靶向破坏T细胞受体
公开(公告)号：WO2017106528A3
公开(公告)日：2017-07-27
申请号：PCT/US2016066975
申请日：2016-12-15
申请人： SANGAMO BIOSCIENCES INC
发明人： LEE GARY K , PASCHON DAVID , ZHANG LEI
IPC分类号： A61K35/15 , A61K35/17 , A61K35/545 , C07K14/705 , C12N5/0783 , C12N5/0789 , C12N5/10 , C12N9/22 , C12N15/62
CPC分类号： C12N15/52 , C12N5/0603 , C12N5/0606 , C12N5/0636 , C12N5/0696 , C12N9/16 , C12N15/62
摘要： Disclosed herein are methods and compositions for inactivating TCR genes, using engineered nucleases comprising at least one DNA binding domain and a cleavage domain or cleavage half-domain in conditions able to preserve cell viability. Polynucleotides encoding nucleases, vectors comprising polynucleotides encoding nucleases and cells comprising polynucleotides encoding nucleases and/or cells comprising nucleases are also provided. Disclosed herein are also methods and compositions for expressing a functional exogenous TCR in the absence of endogenous TCR expression in T lymphocytes, including lymphocytes with a central memory phenotype. Polynucleotides encoding exogenous TCR, vectors comprising polynucleotides encoding exogenous TCR and cells comprising polynucleotides encoding exogenous TCR and/or cells comprising exogenous TCR are also provided.
摘要翻译：本文公开了使用在能够保存细胞活力的条件下包含至少一个DNA结合结构域和切割结构域或切割半结构域的工程化核酸酶来灭活TCR基因的方法和组合物。还提供了编码核酸酶的多核苷酸，包含编码核酸酶的多核苷酸的载体和包含编码核酸酶的多核苷酸和/或包含核酸酶的细胞的细胞。本文还公开了用于在T淋巴细胞（包括具有中枢记忆表型的淋巴细胞）中不存在内源性TCR表达的情况下表达功能性外源性TCR的方法和组合物。还提供了编码外源TCR的多核苷酸，包含编码外源TCR的多核苷酸的载体和包含编码外源TCR的多核苷酸和/或包含外源TCR的细胞的细胞。

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