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1. 发明申请

WO2018002812A1 MATERIALS AND METHODS FOR TREATMENT OF MYOTONIC DYSTROPHY TYPE 1 (DM1) AND OTHER RELATED DISORDERS 审中-公开
标题翻译：治疗1型（DM1）和其他相关疾病的材料和方法
公开(公告)号：WO2018002812A1
公开(公告)日：2018-01-04
申请号：PCT/IB2017/053816
申请日：2017-06-27
申请人： CRISPR THERAPEUTICS AG
发明人： LUNDBERG, Ante Sven , KULKARNI, Samarth , KLEIN, Lawrence , PADMANABHAN, Hari Kumar
IPC分类号： C12N9/22 , A61K48/00 , C12N15/90
CPC分类号： C12N15/907 , A61K35/34 , A61K38/465 , C12N9/22 , C12N15/102 , C12N15/11 , C12N15/113 , C12N2310/20 , C12N2750/14143 , C12N2800/80
摘要： The present application provides materials and methods for treating a patient with one or more conditions associated with DMPK whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of DMPK gene in a cell by genome editing.
摘要翻译：本申请提供了用于治疗患有一种或多种与DMPK相关的病症的材料和方法，无论是离体还是体内。另外，本申请提供了通过基因组编辑来编辑和/或调节细胞中DMPK基因表达的材料和方法。

2. 发明申请

WO2017172645A2 BACTERIOPHAGE ENGINEERING METHODS 审中-公开
标题翻译：细菌学工程方法
公开(公告)号：WO2017172645A2
公开(公告)日：2017-10-05
申请号：PCT/US2017/024369
申请日：2017-03-27
申请人： THE CHARLES STARK DRAPER LABORATORY, INC.
发明人： HOLDER, Jason W. , MCBRINE, Connor , GRUSZKA, Sarah , ROGERS, Miles , BILLINGS, Nicole
IPC分类号： C12N15/86 , C12N15/90 , C12N15/09
CPC分类号： C12Q1/70 , B01L3/502753 , B01L3/502761 , B01L2200/0652 , B01L2200/10 , B01L2300/0627 , B01L2300/12 , C12N7/00 , C12N15/11 , C12N15/66 , C12N15/86 , C12N15/902 , C12N2310/20 , C12N2795/10221 , C12N2795/10243 , C12N2795/10251 , C12N2800/80 , C12Y301/00
摘要： The present disclosure provides methods and kits for generating recombinant bacteriophage genomes.
摘要翻译：本发明提供了用于产生重组噬菌体基因组的方法和试剂盒。

3. 发明申请

WO2017070404A2 ENHANCED PRODUCTIVITY BY ATTENUATION OF CHLOROPHYLL BINDING PROTEIN GENES 审中-公开
标题翻译：通过抑制叶绿酸结合蛋白基因提高生产力
公开(公告)号：WO2017070404A2
公开(公告)日：2017-04-27
申请号：PCT/US2016/058000
申请日：2016-10-20
申请人： SYNTHETIC GENOMICS, INC.
发明人： MEUSER, Jonathan, E. , DIPETRILLO, Christen, G. , MCCARREN, Jay , BAILEY, Shaun
IPC分类号： C07K14/405 , C12N1/12
CPC分类号： C07K14/405 , C12N1/12 , C12N15/113 , C12N15/902 , C12N2800/80
摘要： Mutant photosynthetic algae having increased biomass productivity are provided. The mutants have attenuated expression of violaxanthin chlorophyll a binding proteins (VCP) or fucoxanthin chlorophyll a/c binding proteins (FCP), reduced chlorophyll, higher apparent ETR(II), little to no reduction in P max per cell, and decreased NPQ over a wide range of light intensities. Provided herein are constructs for attenuating or disrupting VCP or FCP genes. Also provided are methods of culturing VCP or FCP mutants for the production of biomass or other products.
摘要翻译：提供了具有提高的生物质生产力的突变光合藻类。突变体已经减弱了紫黄质叶绿素a结合蛋白（VCP）或岩藻黄质叶绿素a / c结合蛋白（FCP）的表达，减少了叶绿素，表观ETR（II）更高，P max降低很少或没有减少，每个细胞，并在广泛的光强度下NPQ下降。本文提供了用于减毒或破坏VCP或FCP基因的构建体。还提供了培养用于生产生物质或其他产品的VCP或FCP突变体的方法。

4. 发明申请

WO2017059241A1 LENTIVIRAL PROTEIN DELIVERY SYSTEM FOR RNA-GUIDED GENOME EDITING 审中-公开
标题翻译：用于RNA指导基因组编辑的生物蛋白递送系统
公开(公告)号：WO2017059241A1
公开(公告)日：2017-04-06
申请号：PCT/US2016/054759
申请日：2016-09-30
申请人： THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES
发明人： UCHIDA, Naoya , HARO MORA, Juan J. , TISDALE, John F.
IPC分类号： C12N9/22 , C12N15/10 , C12N15/86 , C07K7/64 , C12N15/90 , C12N15/867
CPC分类号： C12N15/86 , C07K7/645 , C07K14/155 , C07K2319/00 , C12N9/22 , C12N15/102 , C12N15/907 , C12N2740/15011 , C12N2740/15023 , C12N2740/15032 , C12N2740/15041 , C12N2740/15043 , C12N2740/16011 , C12N2740/16023 , C12N2740/16031 , C12N2740/16041 , C12N2740/16043 , C12N2800/80
摘要： The invention is directed to a system comprising a lentivirus vector particle which encodes a guide RNA sequence that is complementary to a first DNA sequence in a host cell genome, a Cas9 protein, and optionally a donor nucleic acid molecule comprising a second DNA sequence. The invention also is directed to a method of altering a DNA sequence in a host cell using such a system, where the host cell can be in a human and the altered DNA can be of the human β-globin gene. The invention also is directed to a fusion protein comprising a Cas9 protein and a cyclophilin A (CypA) protein. The invention also is directed to sequences of vectors that can be used in the system and method.
摘要翻译：本发明涉及包含慢病毒载体颗粒的系统，其编码与宿主细胞基因组中的第一DNA序列互补的引导RNA序列，Cas9蛋白和任选的包含第二DNA序列的供体核酸分子。本发明还涉及使用这样的系统来改变宿主细胞中的DNA序列的方法，其中宿主细胞可以在人中，并且改变的DNA可以是人β-珠蛋白基因。本发明还涉及包含Cas9蛋白和亲环蛋白A（CypA）蛋白的融合蛋白。本发明还涉及可用于该系统和方法的载体序列。

5. 发明申请

WO2017023801A1 INTRACELLULAR GENOMIC TRANSPLANT AND METHODS OF THERAPY 审中-公开
标题翻译：细胞基因组转运和治疗方法
公开(公告)号：WO2017023801A1
公开(公告)日：2017-02-09
申请号：PCT/US2016/044856
申请日：2016-07-29
申请人： REGENTS OF THE UNIVERSITY OF MINNESOTA , INTIMA BIOSCIENCE, INC.
发明人： MORIARITY, Branden , WEBBER, Beau , MCIVOR, R., Scott , CHOUDHRY, Modassir
IPC分类号： A61K35/17 , C12N1/15 , C12N15/09 , C12N15/85 , C12Q1/68
CPC分类号： A61K35/17 , C07K14/4718 , C07K14/70503 , C07K14/7051 , C07K14/70521 , C07K14/7158 , C12N5/0636 , C12N9/22 , C12N9/96 , C12N15/113 , C12N15/87 , C12N15/907 , C12N2310/20 , C12N2510/00 , C12N2800/80 , Y02A50/463
摘要： Genetically modified compositions, such as non-viral vectors and T cells, for treating cancer are disclosed. Also disclosed are the methods of making and using the genetically modified compositions in treating cancer.
摘要翻译：公开了用于治疗癌症的转基因组合物，例如非病毒载体和T细胞。还公开了制备和使用转基因组合物治疗癌症的方法。

6. 发明申请

WO2017004261A1 MODIFIED CRISPR RNA AND MODIFIED SINGLE CRISPR RNA AND USES THEREOF 审中-公开
标题翻译：改良的CRISPR RNA和修饰的单CRISPR RNA及其用途
公开(公告)号：WO2017004261A1
公开(公告)日：2017-01-05
申请号：PCT/US2016/040191
申请日：2016-06-29
申请人： IONIS PHARMACEUTICALS, INC.
发明人： RAHDAR, Meghdad , PRAKASH, Thazha, P. , SWAYZE, Eric, E. , BENNETT, Frank, C.
IPC分类号： C12N9/22 , C12N15/63 , A61K31/7088 , C07H19/00
CPC分类号： C12N15/11 , A61K38/00 , A61K48/00 , C07H21/00 , C12N7/00 , C12N9/22 , C12N9/96 , C12N15/102 , C12N15/111 , C12N15/907 , C12N2310/20 , C12N2310/31 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/344 , C12N2310/351 , C12N2740/15043 , C12N2750/14343 , C12N2800/80
摘要： The present disclosure provides compounds comprising modified oligonucleotides for use in CRISPR. In certain embodiments, such modified oligonucleotides provide improved properties of crRNA. In cetain embodiments, such modified oligonucleotides provide improved properties of scrRNA.
摘要翻译：本公开提供了包含用于CRISPR的修饰的寡核苷酸的化合物。在某些实施方案中，这样的修饰的寡核苷酸提供了改进的crRNA的性质。在切割实施方案中，这种修饰的寡核苷酸提供了scrRNA的改进的性质。

7. 发明申请

WO2016201155A1 CRISPR-CAS COMPOSITIONS AND METHODS 审中-公开
标题翻译： CRISPR-CAS组合物和方法
公开(公告)号：WO2016201155A1
公开(公告)日：2016-12-15
申请号：PCT/US2016/036779
申请日：2016-06-10
申请人： CARIBOU BIOSCIENCES, INC.
发明人： DONOHOUE, Paul, Daniel , MAY, Andrew, Paul , STENGEL, Katharina, Friederike Sonja
IPC分类号： C12N15/11
CPC分类号： C12N9/22 , C12N15/111 , C12N15/907 , C12N2310/20 , C12N2800/80 , C12P19/34 , C12Y301/00
摘要： Class 2 CRISPR-Cas nucleoprotein complexes are disclosed comprising a Class 2 CRISPR-Cas protein, a CRISPR-Cas associated polynucleotide lacking a spacer element (casPN), and a distinct spacer element sequence polynucleotide (sesPN) comprising a target nucleic acid binding sequence. These complexes are capable of site-directed binding to a target nucleic acid complementary to the target nucleic acid binding sequence of the sesPN. The Class 2 CRISPR-Cas nucleoprotein complexes facilitate site-specific modifications, including cleavage and mutagenesis, of a target nucleic acid sequence. Polynucleotide sequences, expression cassettes, vectors, compositions, and kits for carrying out a variety of methods are also described. Furthermore, the present specification provides guidance for methods of regulating expression of a target nucleic acid sequence, production of genetically modified cells, compositions of modified cells, transgenic organisms, pharmaceutical compositions, as well as a variety of other compositions and methods involving the Class 2 CRISPR-Cas nucleoprotein complexes comprising casPNs, sesPNs, and Cas proteins.
摘要翻译：公开了2类CRISPR-Cas核蛋白复合物，其包含2类CRISPR-Cas蛋白，缺少间隔元件（casPN）的CRISPR-Cas相关多聚核苷酸和包含靶核酸结合序列的不同间隔元件序列多核苷酸（sesPN）。这些配合物能够与与sesPN的靶核酸结合序列互补的靶核酸定点结合。 2类CRISPR-Cas核蛋白复合物促进靶核酸序列的位点特异性修饰，包括切割和诱变。还描述了用于进行各种方法的多核苷酸序列，表达盒，载体，组合物和试剂盒。此外，本说明书提供了调节靶核酸序列表达，转基因细胞的产生，修饰细胞的组合物，转基因生物，药物组合物以及涉及第2类的各种其它组合物和方法的方法的指导包含casPN，sesPN和Cas蛋白的CRISPR-Cas核蛋白复合物。

8. 发明申请

WO2015160683A1 METHODS AND COMPOSITIONS FOR MODIFYING GENOMIC DNA 审中-公开
标题翻译：用于修饰基因DNA的方法和组合物
公开(公告)号：WO2015160683A1
公开(公告)日：2015-10-22
申请号：PCT/US2015/025523
申请日：2015-04-13
申请人： MAXCYTE, INC.
发明人： LI, Linhong , PESHWA, Madhusudan
IPC分类号： C12N5/10
CPC分类号： C12N15/102 , A61K48/00 , C12N15/907 , C12N2800/80
摘要： Compositions and methods concern the sequence modification of an endogenous genomic DNA region. Certain aspects relate to a method for site-specific sequence modification of a target genomic DNA region in cells comprising: transfecting the cells by electroporation with a composition comprising (a) a DNA oligo and (b) a DNA digesting agent wherein the donor DNA comprises: (i) a homologous region comprising nucleic acid sequence homologous to the target genomic DNA region and (ii) a sequence modification region; and wherein the genomic DNA sequence is modified specifically at the target genomic DNA region.
摘要翻译：组合物和方法涉及内源基因组DNA区域的序列修饰。某些方面涉及用于细胞中靶基因组DNA区域的位点特异性序列修饰的方法，包括：通过电穿孔转染细胞，所述组合物包含（a）DNA寡核苷酸和（b）DNA消化剂，其中供体DNA包含（i）包含与靶基因组DNA区域同源的核酸序列的同源区和（ii）序列修饰区; 并且其中所述基因组DNA序列在靶基因组DNA区域特异性地被修饰。

9. 发明申请

WO2015086798A2 NEW METHOD OF SELECTION OF ALGAL-TRANSFORMED CELLS USING NUCLEASE 审中-公开
标题翻译：用核酸酶选择藻类转化细胞的新方法
公开(公告)号：WO2015086798A2
公开(公告)日：2015-06-18
申请号：PCT/EP2014/077513
申请日：2014-12-12
申请人： CELLECTIS
发明人： DUCHATEAU, Philippe , DABOUSSI, Fayza
IPC分类号： C12N15/79
CPC分类号： C12N15/79 , C12N9/22 , C12N15/102 , C12N15/8209 , C12N15/821 , C12N15/8213 , C12N15/902 , C12N2800/80 , C12Y301/00
摘要： The invention relates to a method to select transformed cells. In particular, the present invention relates to the use of a nuclease engineered to inactivate selectable marker which confers cell resistance to a toxic compound. The present invention relates to methods of modifying genome of a cell, preferably an algal cell comprising the present selection step. The present invention also relates to specific engineered nucleases, polynucleotides, vectors encoding thereof, kits and isolated cells comprising said nuclease.
摘要翻译：本发明涉及选择转换细胞的方法。具体而言，本发明涉及经工程改造以灭活赋予细胞对毒性化合物抗性的选择标记的核酸酶的用途。本发明涉及修饰细胞的基因组的方法，优选包含本选择步骤的藻细胞。本发明还涉及特定的工程核酸酶，多核苷酸，编码它的载体，包含所述核酸酶的试剂盒和分离的细胞。

10. 发明申请

WO2015006294A2 ORTHOGONAL CAS9 PROTEINS FOR RNA-GUIDED GENE REGULATION AND EDITING 审中-公开
标题翻译：用于RNA指导基因调控和编码的正交CAS9蛋白
公开(公告)号：WO2015006294A2
公开(公告)日：2015-01-15
申请号：PCT/US2014/045700
申请日：2014-07-08
申请人： PRESIDENT AND FELLOWS OF HARVARD COLLEGE
发明人： CHURCH, George M. , ESVELT, Kevin , MALI, Prashant
IPC分类号： C12N15/85 , C12N15/82
CPC分类号： C12N15/907 , C07K2/00 , C12N9/22 , C12N15/11 , C12N15/113 , C12N15/85 , C12N2310/3519 , C12N2320/33 , C12N2320/50 , C12N2800/107 , C12N2800/40 , C12N2800/80 , C12N2810/10 , C12N2830/00 , C12N2830/34 , C12N2999/007 , C12Y301/00
摘要： Methods of modulating expression of a target nucleic acid in a cell are provided including use of multiple orthogonal Cas9 proteins to simultaneously and independently regulate corresponding genes or simultaneously and independently edit corresponding genes.
摘要翻译：提供了调节细胞中靶核酸表达的方法，包括使用多个正交的Cas9蛋白来同时且独立地调节相应的基因，或同时并且独立地编辑相应的基因。

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