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1. 发明申请

WO2017091630A4 TRACKING AND MANIPULATING CELLULAR RNA VIA NUCLEAR DELIVERY OF CRISPR/CAS9 审中-公开
标题翻译：通过核交付CRISPR / CAS9追踪和操纵细胞RNA
公开(公告)号：WO2017091630A4
公开(公告)日：2017-08-24
申请号：PCT/US2016063429
申请日：2016-11-22
申请人： UNIV CALIFORNIA , FANG MARK , BATRA RANJAN
发明人： FANG MARK , BATRA RANJAN , YEO GENE , NELLES DAVID
IPC分类号： C12N15/11 , C07K19/00 , C12N15/113 , C12N15/62
CPC分类号： C12N9/22 , A61K38/465 , A61K48/0058 , C07K2319/09 , C07K2319/80 , C12N15/111 , C12N15/113 , C12N2310/10 , C12N2310/20
摘要： Cas9 polypeptides which target RNA and methods of using them are provided.
摘要翻译：提供靶向RNA的Cas9多肽及其使用方法。

2. 发明申请

WO2016095934A3 A NOVEL GENETIC DEVICE TO ENGINEER CELL BEHAVIOR 审中-公开
标题翻译：一种新颖的遗传装置，用于工程师细胞行为
公开(公告)号：WO2016095934A3
公开(公告)日：2017-07-13
申请号：PCT/EG2014000038
申请日：2014-12-14
申请人： EL ABD HISHAM MOHAMED MAGDY
发明人： EL ABD HISHAM MOHAMED MAGDY
IPC分类号： A01K67/027 , C12N15/09 , C12N15/10 , C12N15/11 , C12N15/63 , C12N15/85
CPC分类号： C12N15/85 , A01K67/0275 , C12N15/111 , C12N15/635 , C12N2310/10 , C12N2310/20 , C12N2330/51
摘要： This invention is related to the design and production of novel genetic device to control cell behavior. This device can be used to engineer eukaryotic and prokaryotic cells, so it has a wide variety of application from biotechnological to biomedical science and biomedical engendering. Also this invention is related to the production of novel genetic circuit and recombined DNA to control cell behavior. These genetic circuits and recombined DNA can be used to engineer eukaryotic and prokaryotic cells, so it has a wide variety of application from biotechnological to biomedical science and biomedical engendering. Also this invention is contains novel methods to solve some pharmaceutical, biopharmaceutical, and medical obstacles as miRNA delivery system and develop cancer therapeutics. Also this invention contains novel methods related to the production of synthetic promoters.
摘要翻译：本发明涉及控制细胞行为的新型遗传装置的设计和生产。该设备可用于设计真核生物和原核生物细胞，因此它具有从生物技术到生物医学科学和生物医学产生的广泛应用。本发明还涉及新型遗传电路和重组DNA的生产以控制细胞行为。这些遗传电路和重组DNA可以用于设计真核和原核细胞，因此它具有从生物技术到生物医学科学和生物医学产生的广泛应用。本发明还包含解决一些制药，生物制药和医疗障碍的新方法，如miRNA递送系统和开发癌症治疗剂。本发明还包含涉及合成启动子生产的新方法。

3. 发明申请

WO2017031483A1 NUCLEASE WITH ENHANCED EFFICIENCY OF GENOME EDITING 审中-公开
标题翻译：具有增强编码效率的核苷酸
公开(公告)号：WO2017031483A1
公开(公告)日：2017-02-23
申请号：PCT/US2016/047937
申请日：2016-08-22
申请人： APPLIED STEMCELL, INC.
发明人： TSAI, Ruby Yanru , KONG, Ling-Jie , CAO, Jingyuan
IPC分类号： C12N15/63 , C12N15/113 , C12N9/22
CPC分类号： C12N9/22 , C07K2319/00 , C07K2319/80 , C07K2319/81 , C12N15/111 , C12N15/907 , C12N2310/10 , C12N2310/20
摘要： The present disclosure provides a composition comprising a site-specific nuclease domain capable of cleaving a target DNA sequence; and a sequence-specific DNA binding domain capable of specifically binding to a recognition DNA sequence, wherein the site-specific nuclease domain operably links to the sequence-specific DNA binding domain.
摘要翻译：本公开提供了包含能够切割靶DNA序列的位点特异性核酸酶结构域的组合物; 以及能够特异性结合识别DNA序列的序列特异性DNA结合结构域，其中位点特异性核酸酶结构域可操作地连接到序列特异性DNA结合结构域。

4. 发明申请

WO2016200263A1 NEW CRISPR ASSAYS 审中-公开
标题翻译：新CRISPR测试
公开(公告)号：WO2016200263A1
公开(公告)日：2016-12-15
申请号：PCT/NL2016/050422
申请日：2016-06-10
申请人： ERASMUS UNIVERSITY MEDICAL CENTER ROTTERDAM
发明人： LOUWEN, Rogier Petrus Leonardus
IPC分类号： C12N15/11 , C12N15/09 , C12N15/63
CPC分类号： C12N15/113 , C12N15/111 , C12N15/63 , C12N15/85 , C12N2310/10 , C12N2310/14 , C12N2310/20 , G01N33/5023
摘要： The present invention relates to expression vectors harboring eukaryotic CRISPR sequences and their use in assays to study the intervention of eukaryotic CRISPR sequences with gene regulation and with gene editing in eukaryotes using CRISPR-Cas technology.
摘要翻译：本发明涉及携带真核CRISPR序列的表达载体及其在用于研究使用CRISPR-Cas技术的真核生物中具有基因调控和基因编辑的真核CRISPR序列的干预的测定中的用途。

5. 发明申请

WO2016197132A1 TREATING HEPATITIS B VIRUS INFECTION USING CRISPR 审中-公开
标题翻译：使用CRISPR治疗乙型肝炎病毒感染
公开(公告)号：WO2016197132A1
公开(公告)日：2016-12-08
申请号：PCT/US2016/036068
申请日：2016-06-06
申请人： PROTIVA BIOTHERAPEUTICS INC. , LEE, Amy C. H. , WEBER, Nicholas D.
发明人： LEE, Amy C. H. , WEBER, Nicholas D.
IPC分类号： C12N15/113 , A61K9/127 , A61P31/20
CPC分类号： C12N15/1131 , A61K9/127 , C12N9/22 , C12N15/1137 , C12N2310/10 , C12N2310/20 , C12N2320/32
摘要： The present invention provides compositions comprising therapeutic nucleic acids such as gRNA that target Hepatitis B virus (HBV) gene expression, lipid particles comprising one or more (e.g., a combination) of the therapeutic nucleic acids, and methods of delivering and/or administering the lipid particles ( e.g ., for treating HBV infection and/or HDV infection in humans).
摘要翻译：本发明提供包含靶向乙型肝炎病毒（HBV）基因表达的治疗性核酸例如gRNA，包含治疗性核酸的一种或多种（例如组合）的脂质颗粒的组合物，以及递送和/或施用脂质颗粒（例如，用于治疗人类的HBV感染和/或HDV感染）。

6. 发明申请

WO2016183236A1 CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR TREATING HIV INFECTION AND AIDS 审中-公开
标题翻译： CRISPR / CAS相关方法和治疗艾滋病毒感染和艾滋病的组合物
公开(公告)号：WO2016183236A1
公开(公告)日：2016-11-17
申请号：PCT/US2016/031922
申请日：2016-05-11
申请人： EDITAS MEDICINE, INC.
发明人： GORI, Jennifer, Leah , WELSTEAD, G., Grant , ODONNELL, Penrose
IPC分类号： C12N15/113 , A61K31/713 , C12N15/63
CPC分类号： C12N9/22 , A61K31/713 , A61K38/465 , A61K48/00 , C12N15/1138 , C12N2310/10 , C12N2310/20 , C12N2310/317 , C12N2320/34
摘要： CRISPR/CAS-related systems, compositions and methods for editing CCR5 and/or CXCR4 genes in human cells are described, as are cells and compositions including cells edited according to the same.
摘要翻译：描述用于编辑人细胞中CCR5和/或CXCR4基因的CRISPR / CAS相关系统，组合物和方法，细胞和组合物包括根据其编辑的细胞。

7. 发明申请

WO2016135558A3 MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES 审中-公开
公开(公告)号：WO2016135558A3
公开(公告)日：2016-10-20
申请号：PCT/IB2016000282
申请日：2016-02-23
申请人： CRISPR THERAPEUTICS AG , GEORGIA TECH RES INST , UNIV RICE WILLIAM M
发明人： PORTEUS MATTHEW HEBDEN , CRADICK THOMAS JAMES , BAO GANG , LEE CIARAN M
IPC分类号： C12N15/113 , C12N15/09 , C12N15/63
CPC分类号： A61K38/42 , A61K35/28 , A61K35/545 , C12N5/0647 , C12N5/0696 , C12N15/102 , C12N15/113 , C12N15/63 , C12N2310/10 , C12N2310/20 , C12N2320/11 , C12N2501/73 , C12N2510/00
摘要： The present application provides materials and methods for treating hemoglobinopathies. More specifically, the application provides methods for producing progenitor cells that are genetically modified via genome editing to increase the production of fetal hemoglobin (HbF), as well as modified progenitor cells (including, for example, CD34+ human hematopoietic stem cells) producing increased levels of HbF, and methods of using such cells for treating hemoglobinopathies such as sickle cell anemia and β-thalassemia.

8. 发明申请

WO2016164303A1 METHODS FOR DIAGNOSING AND TREATING COLORECTAL CANCER USING snoRNA 审中-公开
标题翻译：使用snoRNA诊断和治疗彩色癌的方法
公开(公告)号：WO2016164303A1
公开(公告)日：2016-10-13
申请号：PCT/US2016/025857
申请日：2016-04-04
申请人： BAYLOR RESEARCH INSTITUTE
发明人： GOEL, Ajay , OKUGAWA, Yoshinaga , YOSHIDA, Kazuhiro
IPC分类号： C12N15/113 , C12Q1/68
CPC分类号： C12N15/113 , A61K31/00 , C12N2310/10 , C12Q1/68 , C12Q1/6886 , C12Q2600/118 , C12Q2600/158 , C12Q2600/178
摘要： The current disclosure relates to therapeutic treatments and diagnostic methods for colorectal cancer based on the expression level of snoRNA expression. Aspects of the current disclosure relate to a method for treating a patient for early or advanced colorectal cancer comprising: treating the patient for advanced colorectal cancer after the patient is determined to have a high elevated level of snoRNA expression in a biological sample from the patient; and treating the patient for early colorectal cancer after the patient is determined to have a low elevated level of snoRNA expression in a biological sample from the patient.
摘要翻译：目前的公开涉及基于snoRNA表达的表达水平的结肠直肠癌的治疗治疗和诊断方法。本公开的方面涉及用于治疗早期或晚期结直肠癌患者的方法，其包括：在确定患者在来自患者的生物样品中具有高水平的snoRNA表达水平之后，对所述患者进行晚期结肠直肠癌治疗; 并且在确定患者在来自患者的生物样品中具有低水平的snoRNA表达水平之后，对患者进行早期结肠直肠癌治疗。

9. 发明申请

WO2016154675A1 PLATFORM FOR NON-NATURAL AMINO ACID INCORPORATION INTO PROTEINS 审中-公开
标题翻译：非天然氨基酸合成蛋白质的平台
公开(公告)号：WO2016154675A1
公开(公告)日：2016-10-06
申请号：PCT/AU2016/050239
申请日：2016-03-29
申请人： THE UNIVERSITY OF QUEENSLAND
发明人： MUREEV, Sergey , CUI, Zhenling , ALEXANDROV, Kirill
IPC分类号： C12N15/67 , C12P21/02
CPC分类号： C12N9/93 , C07K7/64 , C12N15/52 , C12N15/67 , C12N15/70 , C12N2310/10 , C12N2310/332 , C12P21/02 , C12Y603/04019 , C40B40/10
摘要： A complement to tRNAs and a protein translation system are provided that enable the incorporation of non-natural moieties such as non-natural amino acids without compromising the ability to incorporate all of the twenty natural amino acids into the protein. This is achieved by reassigning one of the tRNA anticodons for amino acids that are normally decoded by at least two different tRNA anticodons to a non-natural moiety, wherein at least one codon can be uniquely recognized by the reassigned anticodon and at least one another codon from the same codon box cannot be recognized by the reassigned anticodon. Accordingly, an mRNA for translation is engineered to comprise one or more specific codons corresponding to the reassigned tRNA anticodons so that the non-natural moiety is incorporated into the translated protein at a selected position.
摘要翻译：提供了对tRNA和蛋白翻译系统的补充，其能够引入非天然部分如非天然氨基酸，而不损害将所有二十种天然氨基酸掺入蛋白质的能力。这通过将通常由至少两个不同tRNA反密码子解码的氨基酸的一个tRNA反密码子重新分配给非天然部分来实现，其中至少一个密码子可以被重新分配的反密码子和至少一个其他密码子唯一地识别从相同的密码子框不能被重新分配的反密码子识别。因此，用于翻译的mRNA被设计为包含对应于重新分配的tRNA反密码子的一个或多个特定密码子，使得非天然部分在选定位置并入翻译的蛋白质中。

10. 发明申请

WO2015138510A8 CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR TREATING LEBER'S CONGENITAL AMAUROSIS 10 (LCA10) 审中-公开
标题翻译： CRISPR / CAS相关方法和组合物用于治疗LEBER'S CONGENITAL AMAUROSIS 10（LCA10）
公开(公告)号：WO2015138510A8
公开(公告)日：2016-10-06
申请号：PCT/US2015019790
申请日：2015-03-10
申请人： EDITAS MEDICINE INC
发明人： MAEDER MORGAN L , BUMCROT DAVID A , SHEN SHEN
IPC分类号： C12N15/113 , A61K31/7088 , A61K48/00 , C12N9/22 , C12N15/86
CPC分类号： C12N15/1024 , C12N9/22 , C12N15/11 , C12N15/113 , C12N15/86 , C12N2310/10 , C12N2310/20 , C12N2750/14132 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2750/14151
摘要： CRISPR/CAS-related compositions and methods for treatment of Leber's Congenital Amaurosis 10 (LCAIO) are disclosed.
摘要翻译：公开了用于治疗Leber先天性黑霉素10（LCAIO）的CRISPR / CAS相关组合物和方法。

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