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1. 发明申请

WO2018071405A1 VECTORS AND VACCINE CELLS FOR IMMUNITY AGAINST ZIKA VIRUS 审中-公开
标题翻译：矢量和疫苗细胞免疫ZIKA病毒
公开(公告)号：WO2018071405A1
公开(公告)日：2018-04-19
申请号：PCT/US2017/055912
申请日：2017-10-10
申请人： UNIVERSITY OF MIAMI
发明人： STRBO, Natasa , ROMERO, Laura
IPC分类号： A61K39/12 , C07K14/18 , C12N7/04
CPC分类号： A61K39/12 , A61K2039/53 , A61P31/14 , C07K14/005 , C07K2319/30 , C12N2770/24134 , C12N2830/20 , Y02A50/392
摘要： The present invention provides an expression vector, host cells, methods and kits for the treatment or prevention of a flavivirus infection in a subject.
摘要翻译：本发明提供了用于治疗或预防受试者中黄病毒感染的表达载体，宿主细胞，方法和试剂盒。

2. 发明申请

WO2015042308A3 RNA-BASED HIV INHIBITORS 审中-公开
标题翻译：基于RNA的HIV抑制剂
公开(公告)号：WO2015042308A3
公开(公告)日：2015-11-12
申请号：PCT/US2014056384
申请日：2014-09-18
申请人： HOPE CITY
发明人： ROSSI JOHN , DIGIUSTO JOHN , CHUNG JANET , SCHERER LISA
IPC分类号： A61K48/00
CPC分类号： C12N15/1132 , A61K48/005 , C12N7/00 , C12N15/1131 , C12N15/1138 , C12N15/86 , C12N2310/13 , C12N2310/14 , C12N2310/51 , C12N2310/531 , C12N2320/31 , C12N2330/51 , C12N2740/15043 , C12N2740/16043 , C12N2830/20 , C12N2830/205 , C12N2840/20
摘要： Provided herein are, inter alia, antiviral recombinant nucleic acid compositions and methods of using the same. The recombinant nucleic acid compositions include nucleic acids encoding antiviral polycistronic RNAs, which are capable of inhibiting viral replication. The antiviral recombinant nucleic acid compositions provided herein are therefore particularly useful for therapeutic applications such as combinational HIV-1 gene therapy.
摘要翻译：此处提供的是抗病毒重组核酸组合物和使用其的方法。重组核酸组合物包括编码抗病毒多顺反子RNA的核酸，其能够抑制病毒复制。因此本文提供的抗病毒重组核酸组合物因此特别用于治疗应用，例如组合HIV-1基因疗法。

3. 发明申请

WO2013049493A1 INDUCIBLE ADENO -ASSOCIATED VIRUS VECTOR MEDIATED TRANSGENE ABLATION SYSTEM 审中-公开
标题翻译：可诱导的腺病毒载体介导的转基因系统
公开(公告)号：WO2013049493A1
公开(公告)日：2013-04-04
申请号：PCT/US2012/057803
申请日：2012-09-28
申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
发明人： WILSON, James, M. , TRETIAKOVA, Anna, P. , GREIG, Jenny, Agnes
IPC分类号： C12N15/86 , A61K48/00
CPC分类号： A61K48/0066 , C07K7/08 , C07K14/00 , C07K14/4702 , C07K2319/09 , C07K2319/715 , C07K2319/80 , C07K2319/81 , C07K2319/92 , C12N7/00 , C12N9/22 , C12N15/86 , C12N2750/14143 , C12N2750/14151 , C12N2800/30 , C12N2800/40 , C12N2800/80 , C12N2830/002 , C12N2830/003 , C12N2830/005 , C12N2830/006 , C12N2830/20 , C12N2840/203 , C12Y301/21004
摘要： The present invention relates to gene therapy systems designed for the delivery of a therapeutic product to a subject using replication-defective virus composition(s) engineered with a built-in safety mechanism for ablating the therapeutic gene product, either permanently or temporarily, in response to a pharmacological agent - preferably an oral formulation, e.g., a pill. The invention is based, in part, on the applicants' development of an integrated approach, referred to herein as "PITA" (Pharmacologically Induced Transgene Ablation), for ablating a transgene or negatively regulating transgene expression. In this approach, replication-deficient viruses are used to deliver a transgene encoding a therapeutic product (an RNA or a protein) so that it is expressed in the subject, but can be reversibly or irreversibly turned off by administering the pharmacological agent; e.g., by administration of a small molecule that induces expression of an ablator specific for the transgene or its RNA transcript.
摘要翻译：本发明涉及基因治疗系统，其被设计用于使用使用内置安全机制工程改造的复制缺陷型病毒组合物将治疗产品递送给受试者，所述安全机制永久地或临时地用于消融治疗性基因产物药物剂 - 优选口服制剂，例如丸剂。本发明部分地基于申请人开发一种综合方法，在本文中称为“PITA”（药理诱导的转基因消融），用于消融转基因或负调控转基因表达。在这种方法中，使用复制缺陷病毒递送编码治疗产物（RNA或蛋白质）的转基因，使其在受试者中表达，但是可以通过施用药理学试剂来逆转或不可逆地关闭; 例如通过施用诱导特异于转基因或其RNA转录物的消融体表达的小分子。

4. 发明申请

WO2013043196A1 IMPROVED GENE THERAPY METHODS 审中-公开
标题翻译：改进的基因治疗方法
公开(公告)号：WO2013043196A1
公开(公告)日：2013-03-28
申请号：PCT/US2011/053096
申请日：2011-09-23
申请人： BLUEBIRD BIO, INC. , NEGRE, Olivier , PAYEN, Emmanuel , LEBOULCH, Philippe , BEUZARD, Yves
发明人： NEGRE, Olivier , PAYEN, Emmanuel , LEBOULCH, Philippe , BEUZARD, Yves
IPC分类号： C12N15/867 , C12N15/12 , A61K48/00 , A61P7/00
CPC分类号： C12N15/85 , A61K35/18 , A61K35/28 , A61K38/1816 , A61K48/00 , A61K2035/124 , C07K14/71 , C07K14/805 , C12N15/86 , C12N2740/16043 , C12N2800/22 , C12N2830/008 , C12N2830/15 , C12N2830/20 , C12N2830/40
摘要： The present invention generally provides improved gene therapy vectors, cell-based compositions, and methods of using the same in methods of gene therapy. The present invention further provides improved gene therapy compositions for expanding hematopoietic cells and related methods for treatment of diseases, disorders, and conditions of the hematopoietic system such as thalassemias and anemias.
摘要翻译：本发明通常提供改进的基因治疗载体，基于细胞的组合物，以及在基因治疗方法中使用它们的方法。本发明进一步提供用于扩增造血细胞的改进的基因治疗组合物和用于治疗诸如地中海贫血和贫血的造血系统的疾病，病症和状况的相关方法。

5. 发明申请

WO2012165270A1 遺伝子ターゲティングベクター、その作製方法及び利用方法审中-公开
标题翻译：基因定向矢量，其制造方法及其使用方法
公开(公告)号：WO2012165270A1
公开(公告)日：2012-12-06
申请号：PCT/JP2012/063248
申请日：2012-05-24
申请人：公立大学法人横浜市立大学 , 足立典隆
发明人：足立　典隆
IPC分类号： C12N15/09 , C12N1/15 , C12N1/19 , C12N1/21 , C12N5/10
CPC分类号： C12N15/907 , C12N15/64 , C12N15/85 , C12N15/90 , C12N2800/107 , C12N2830/20
摘要：　効率が高い遺伝子ターゲティングを可能とする遺伝子ターゲティングベクターを提供する。　バイシストロン性発現を可能にするDNA配列が選択用マーカーの5'上流に存在することを特徴とする遺伝子ターゲティングベクター。標的部位の5'上流領域と相同なDNA断片、バイシストロン性発現を可能にするDNA配列が5'上流に存在する選択用マーカー及び標的部位の3'下流領域と相同なDNA断片を連結することを含む、遺伝子ターゲティングベクターを作製する方法。
摘要翻译：提供能够高效基因靶向的基因靶向载体。基因靶向载体的特征在于能够双顺反子表达的DNA序列位于选择标记的5'上游。一种生产基因靶向载体的方法，包括连接与靶位点的5'上游区同源的DNA片段，其中存在5'上游存在双顺反子表达的DNA序列的选择标记，以及与靶位点的3'下游区域同源。

6. 发明申请

WO2012112603A1 CONSTRUCTS AND METHODS FOR GENERATING INDUCED PLURI POTENT STEM ( IPS) CELLS 审中-公开
标题翻译：用于产生诱导性脑脊液（IPS）细胞的构建和方法
公开(公告)号：WO2012112603A1
公开(公告)日：2012-08-23
申请号：PCT/US2012/025117
申请日：2012-02-14
申请人： UNIVERSITY OF UTAH RESEARCH FOUNDATIONS , PULST, Stefan, M. , PAUL, Sharan , DANSITHONG, Warunee
发明人： PULST, Stefan, M. , PAUL, Sharan , DANSITHONG, Warunee
IPC分类号： C12N15/861 , C12N5/10 , C12N5/07
CPC分类号： C12N5/0696 , C12N15/86 , C12N2501/602 , C12N2501/603 , C12N2501/604 , C12N2501/606 , C12N2506/1307 , C12N2510/00 , C12N2710/10343 , C12N2830/20
摘要： Systems, constructs, and methods for reprogramming cells and loading viral particles are provided. In one aspect, for example, a transformation construct for generating iPS cells can include a vector backbone having a plurality of reprogramming factors, each reprogramming factor being under control of a separate promoter, wherein the vector backbone is contained in an adenovirus particle. In one aspect, the vector backbone has a size of at least 8 kb. In another aspect, the vector backbone has a size of at least 10 kb.
摘要翻译：提供了用于重编程细胞和加载病毒颗粒的系统，构建体和方法。一方面，例如，用于产生iPS细胞的转化构建体可以包括具有多个重编程因子的载体骨架，每个重编程因子都在单独的启动子的控制下，其中载体骨架包含在腺病毒颗粒中。一方面，载体骨架具有至少8kb的大小。另一方面，载体骨架具有至少10kb的大小。

7. 发明申请

WO2012085911A1 MEMBRANE BOUND REPORTER MOLECULES AND THEIR USE IN CELL SORTING 审中-公开
标题翻译：膜结合报告分子及其在细胞分选中的应用
公开(公告)号：WO2012085911A1
公开(公告)日：2012-06-28
申请号：PCT/IL2011/000956
申请日：2011-12-19
申请人： MERCK SERONO S.A , HELMAN, Daniel , TOISTER-ACHITUV, Mira , BAR-SHIMON, Meirav , SMOLARSKY, Moshe
发明人： HELMAN, Daniel , TOISTER-ACHITUV, Mira , BAR-SHIMON, Meirav , SMOLARSKY, Moshe
IPC分类号： C12Q1/68 , C12N15/10
CPC分类号： G01N33/56966 , C07K2319/035 , C07K2319/20 , C12N15/85 , C12N2830/20 , C12N2830/40 , C12Q1/6897 , C12Q2563/131
摘要： The present invention relates to nucleic acid molecules comprising a nucleic acid sequence encoding a membrane-bound biotin mimetic peptide (BMP) or biotin acceptor peptide (BAP). The invention also relates to a method for selection of high producer cells secreting a protein of interest.
摘要翻译：本发明涉及包含编码膜结合生物素模拟肽（BMP）或生物素受体肽（BAP）的核酸序列的核酸分子。本发明还涉及用于选择分泌目的蛋白质的高生产细胞的方法。

8. 发明申请

WO2011126808A2 PHARMACOLOGICALLY INDUCED TRANSGENE ABLATION SYSTEM 审中-公开
标题翻译：药理学诱导转基因系统
公开(公告)号：WO2011126808A2
公开(公告)日：2011-10-13
申请号：PCT/US2011030213
申请日：2011-03-28
申请人： UNIV PENNSYLVANIA , WILSON JAMES M , CHEN SHU-JEN , TRETIAKOVA ANNA P
发明人： WILSON JAMES M , CHEN SHU-JEN , TRETIAKOVA ANNA P
IPC分类号： C12N15/86 , A61K48/00 , C12N15/864
CPC分类号： C12N15/86 , A61K48/0066 , C07K2319/715 , C07K2319/80 , C12N9/22 , C12N2750/14143 , C12N2800/30 , C12N2800/80 , C12N2830/002 , C12N2830/003 , C12N2830/005 , C12N2830/006 , C12N2830/20 , C12N2840/203
摘要： The present invention relates to gene therapy systems designed for the delivery of a therapeutic product to a subject using replication-defective virus composition(s) engineered with a built-in safety mechanism for ablating the therapeutic gene product, either permanently or temporarily, in response to a pharmacological agent - preferably an oral formulation, e.g., a pill. The invention is based, in part, on the applicants' development of an integrated approach, referred to herein as "PITA" (Pharmacologically Induced Transgene Ablation), for ablating a transgene or negatively regulating transgene expression. In this approach, replication-deficient viruses are used to deliver a transgene encoding a therapeutic product (an RNA or a protein) so that it is expressed in the subject, but can be reversibly or irreversibly turned off by administering the pharmacological agent; e.g., by administration of a small molecule that induces expression of an ablator specific for the transgene or its RNA transcript.
摘要翻译：本发明涉及基因治疗系统，其被设计用于使用使用内置安全机制工程改造的复制缺陷型病毒组合物将治疗产品递送给受试者，所述安全机制永久地或临时地用于消融治疗性基因产物药物剂 - 优选口服制剂，例如丸剂。本发明部分地基于申请人开发一种综合方法，在本文中称为“PITA”（药理诱导的转基因消融），用于消融转基因或负调控转基因表达。在这种方法中，使用复制缺陷病毒递送编码治疗产物（RNA或蛋白质）的转基因，使其在受试者中表达，但是可以通过施用药理学试剂来逆转或不可逆地关闭; 例如通过施用诱导特异于转基因或其RNA转录物的消融体表达的小分子。

9. 发明申请

WO2017140905A1 ZIKA VIRUS VACCINE 审中-公开
标题翻译： ZIKA病毒疫苗
公开(公告)号：WO2017140905A1
公开(公告)日：2017-08-24
申请号：PCT/EP2017/053721
申请日：2017-02-17
申请人： CUREVAC AG , SANOFI PASTEUR SA
发明人： PETSCH, Benjamin , JASNY, Edith , GIRERD-CHAMBAZ, Yves
IPC分类号： A61K39/12
CPC分类号： A61K39/12 , A61K2039/53 , C12N2770/24134 , C12N2830/20 , Y02A50/392
摘要： The present invention is directed to an artificial nucleic acid and to polypeptides suitable for use in treatment or prophylaxis of an infection with Zika virus or a disorder related to such an infection. In particular, the present invention concerns a Zika virus vaccine. The present invention is directed to an artificial nucleic acid, polypeptides, compositions and vaccines comprising the artificial nucleic acid or the polypeptides. The invention further concerns a method of treating or preventing a disorder or a disease, first and second medical uses of the artificial nucleic acid, polypeptides, compositions and vaccines. Further, the invention is directed to a kit, particularly to a kit of parts, comprising the artificial nucleic acid, polypeptides, compositions and vaccines.
摘要翻译：本发明涉及人工核酸和适用于治疗或预防寨卡病毒感染或与感染有关的病症的多肽。具体而言，本发明涉及寨卡病毒疫苗。本发明涉及包含人造核酸或多肽的人造核酸，多肽，组合物和疫苗。本发明还涉及治疗或预防病症或疾病，人造核酸，多肽，组合物和疫苗的第一和第二医学用途的方法。此外，本发明涉及试剂盒，特别是成套试剂盒，其包含人造核酸，多肽，组合物和疫苗。

10. 发明申请

WO2017005923A1 LENTIVIRAL VECTOR EXPRESSING MEMBRANE-ANCHORED OR SECRETED ANTIBODY 审中-公开
标题翻译：表达载体表达的膜载体或分泌的抗体
公开(公告)号：WO2017005923A1
公开(公告)日：2017-01-12
申请号：PCT/EP2016/066349
申请日：2016-07-08
申请人： INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) , UNIVERSITÉ CLAUDE BERNARD LYON 1 , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE , ECOLE NORMALE SUPERIEURE DE LYON
发明人： FUSIL, Floriane , VERHOEYEN, Els , DEFRANCE, Thierry , COSSET, François-Loïc
IPC分类号： C12N15/86 , C07K16/10 , A61K39/29
CPC分类号： C12N15/86 , A61K39/29 , A61K2039/505 , A61K2039/5256 , C07K16/00 , C07K16/109 , C07K2317/10 , C07K2317/76 , C07K2319/03 , C12N2740/15043 , C12N2810/6054 , C12N2830/008 , C12N2830/20 , C12N2830/42 , C12N2830/48 , C12N2830/50
摘要： The invention concerns a multicistronic nucleic acid, in particular an isolated multicistronic nucleic acid, comprising: A) a sequence comprising successively: A1) a sequence encoding the light chain variable domain of an antibody of interest, fused in the frame with A2) a sequence encoding the constant region of the light chain of an immunoglobulin Ig; and B) a sequence comprising successively: B1) a sequence encoding the heavy chain variable domain of said antibody of interest, fused in the frame with B2) a sequence encoding the constant regions of the heavy chain of an immunoglobulin Ig' in secretory form; B3) an intronic sequence of the gene of the heavy chain of said immunoglobulin Ig', said intronic sequence comprising an internal 5' splice site enabling the splicing of said intronic sequence B3) and a secretory-specific poly(A) (p AS) signal from the 3' terminal exon of said gene; B4) a sequence, in frame with sequence B1), encoding the transmembrane and cytoplasmic domains M1 and M2 of the immunoglobulin Ig' BCR, wherein said sequence B4) comprises, between the coding sequences of the M1 and M2 domains, an intronic sequence containing a splice site enabling the splicing of said intronic sequence between the M1 and M2 domains coding sequences; and B5) a membrane-anchored specific poly(A) signal (p AM), after the stop codon of the M2 domain, wherein the multicistronic nucleic acid enables the co-expression of the sequences A and B into separate proteins.
摘要翻译：本发明涉及多顺反子核酸，特别是分离的多顺反子核酸，其包含：A）连续包含的序列：A1）编码感兴趣抗体的轻链可变结构域的序列，其融合在框架中A2）序列编码免疫球蛋白Ig的轻链的恒定区; 和B）连续包含的序列：B1）编码所述感兴趣抗体的重链可变结构域的序列，其与所述框架融合在一起; B2）编码分泌形式的免疫球蛋白Ig'的重链恒定区的序列; B3）所述免疫球蛋白Ig'的重链基因的内含子序列，所述内含子序列包含能够剪接所述内含子序列B3）和分泌特异性聚（A）（p AS）的内部5'剪接位点，来自所述基因的3'末端外显子的信号; B4）编码免疫球蛋白Ig'BCR的跨膜和胞质结构域M1和M2的序列，其中序列B1），其中所述序列B4）在M1和M2结构域的编码序列之间包含含有所述剪接位点能够在M1和M2结构域编码序列之间剪接所述内含子序列; 和B5）M2结构域的终止密码子之后的膜锚定的特异性聚（A）信号（pAM），其中所述多顺反子核酸能够使序列A和B共同表达成分离的蛋白质。

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