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1. 发明申请

WO2012138453A1 EFFICIENT PROTEIN EXPRESSION IN VIVO USING MODIFIED RNA (MOD-RNA) 审中-公开
标题翻译：使用修饰型RNA（MOD-RNA）的VIVO中有效的蛋白表达
公开(公告)号：WO2012138453A1
公开(公告)日：2012-10-11
申请号：PCT/US2012/028802
申请日：2012-03-12
申请人： THE GENERAL HOSPITAL CORPORATION , IMMUNE DISEASE INSTITUTE, INC. , CHIEN, Kenneth, R. , PTASZEK, Leon, M. , LUI, Kathy, Oi-Lan , ZANGI, Lior , EBINA, Wataru , ROSSI, Derrick J.
发明人： CHIEN, Kenneth, R. , PTASZEK, Leon, M. , LUI, Kathy, Oi-Lan , ZANGI, Lior , EBINA, Wataru , ROSSI, Derrick J.
IPC分类号： C12N15/87 , C12N15/11 , A61K48/00 , A61P43/00 , A61P35/00
CPC分类号： A61K38/1866 , A01K67/0276 , A01K2207/05 , A01K2217/075 , A01K2227/105 , A01K2267/03 , A01K2267/0393 , A61K31/4745 , A61K31/513 , A61K31/517 , A61K31/519 , A61K31/555 , A61K31/7068 , A61K31/7115 , A61K33/24 , A61K48/005 , A61L31/10 , A61L31/16 , A61L2300/258 , C12N15/111 , C12N2310/11 , C12N2310/14 , C12N2800/30 , A61K2300/00
摘要： Aspects of the invention described herein relate to synthetic, modified RNAs and their use in vivo to modulate gene expression. Aspects of the invention further relate to the use of these synthetic, modified RNAs in myocytes, cardiomyoctes, and tumors.
摘要翻译：本文描述的本发明的方面涉及合成，修饰的RNA及其在体内用于调节基因表达的用途。本发明的方面还涉及这些合成的，修饰的RNA在肌细胞，心肌细胞和肿瘤中的用途。

2. 发明申请

WO2010144678A3 GENERATION OF VASCULARIZED HUMAN HEART TISSUE AND USES THEREOF 审中-公开
标题翻译：血管化人心脏组织的产生及其用途
公开(公告)号：WO2010144678A3
公开(公告)日：2011-04-21
申请号：PCT/US2010038134
申请日：2010-06-10
申请人： GEN HOSPITAL CORP , CHIEN KENNETH R , BU LEI , JIANG XIN , LUI KATHY OI LAN
发明人： CHIEN KENNETH R , BU LEI , JIANG XIN , LUI KATHY OI LAN
IPC分类号： C12N5/071 , A61P9/00 , C07K14/475 , C12N5/02
CPC分类号： C12N5/0662 , A61K35/12 , C12N5/0657 , C12N2501/15 , C12N2501/165 , C12N2501/415 , C12N2506/02
摘要： The present invention relates to the generation of vascularized human heart tissue from human primordial Islet1-positive (ISL1+) progenitors, and more particularly the generation of vascularized human heart tissue from human primordial Islet1+ cardiovascular stem cells which are positive for markers ISL1+/NKX2.5-/KDR-. One aspect of the invention relates to isolation of human ISL1+ primordial cells from human pluripotent cells, such as human ES cells or other human pluripotent stem cell sources, wherein the human ISL1+ primordial cells can differentiate into three different lineages; cardiomyocyte lineages, endothelial lineages and smooth muscle lineages. Another aspect relates to use and implantation of the human primordial ISL1+ progenitors into an animal model to generate human vascularized heart tissue, and more particularly, the production of an in vivo humanized model of vascular disease. One embodiment relates to the use of an in vivo humanized model of vascular disease as an assay, for example to assess drug toxicity and/or identify agents which increase and decrease coronary blood flow to the human vascularized heart tissue. Another embodiment relates to the therapeutic use of human primordial ISL1+ progenitors, for example, in one embodiment the invention provides methods for the treatment cardiovascular disorders and/or congenital heart disease in a subject comprising transplanting into subjects vascularized human heart tissue generated from human ISL1+ progenitors.
摘要翻译：本发明涉及从人原始Islet1阳性（ISL1 +）祖细胞产生血管化人心脏组织，更具体地说，从人原代Islet1 +心血管干细胞产生血管化人心脏组织，其对于标志物ISL1 + / NKX2.5是阳性的 - / KDR-。本发明的一个方面涉及从人多能细胞例如人ES细胞或其他人多能干细胞来源分离人ISL1 +原始细胞，其中人ISL1 +原始细胞可分化成三种不同谱系; 心肌细胞谱系，内皮谱系和平滑肌谱系。另一方面涉及将人原始ISL1 +祖细胞用于和植入动物模型中以产生人血管化的心脏组织，更具体地，涉及血管疾病的体内人源化模型的产生。一个实施方案涉及血管疾病的体内人源化模型的用途作为测定，例如用于评估药物毒性和/或鉴定增加和减少冠心病血液流向人血管化心脏组织的药剂。另一个实施方案涉及人原始ISL1 +祖细胞的治疗用途，例如，在一个实施方案中，本发明提供了治疗受试者心血管疾病和/或先天性心脏病的方法，所述方法包括移植入受试者从人ISL1 +祖细胞产生的血管化人心脏组织。

3. 发明申请

WO2010144678A2 GENERATION OF VASCULARIZED HUMAN HEART TISSUE AND USES THEREOF 审中-公开
标题翻译：血管化人心脏组织的产生及其用途
公开(公告)号：WO2010144678A2
公开(公告)日：2010-12-16
申请号：PCT/US2010/038134
申请日：2010-06-10
申请人： THE GENERAL HOSPITAL CORPORATION , CHIEN, Kenneth R , BU, Lei , JIANG, Xin , LUI, Kathy Oi Lan
发明人： CHIEN, Kenneth R , BU, Lei , JIANG, Xin , LUI, Kathy Oi Lan
IPC分类号： C12N5/071 , C12N5/02 , C07K14/475 , A61P9/00
CPC分类号： C12N5/0662 , A61K35/12 , C12N5/0657 , C12N2501/15 , C12N2501/165 , C12N2501/415 , C12N2506/02
摘要： The present invention relates to the generation of vascularized human heart tissue from human primordial Islet1-positive (ISL1+) progenitors, and more particularly the generation of vascularized human heart tissue from human primordial Islet1+ cardiovascular stem cells which are positive for markers ISL1+/NKX2.5-/KDR-. One aspect of the invention relates to isolation of human ISL1+ primordial cells from human pluripotent cells, such as human ES cells or other human pluripotent stem cell sources, wherein the human ISL1+ primordial cells can differentiate into three different lineages; cardiomyocyte lineages, endothelial lineages and smooth muscle lineages. Another aspect relates to use and implantation of the human primordial ISL1+ progenitors into an animal model to generate human vascularized heart tissue, and more particularly, the production of an in vivo humanized model of vascular disease. One embodiment relates to the use of an in vivo humanized model of vascular disease as an assay, for example to assess drug toxicity and/or identify agents which increase and decrease coronary blood flow to the human vascularized heart tissue. Another embodiment relates to the therapeutic use of human primordial ISL1+ progenitors, for example, in one embodiment the invention provides methods for the treatment cardiovascular disorders and/or congenital heart disease in a subject comprising transplanting into subjects vascularized human heart tissue generated from human ISL1+ progenitors.
摘要翻译：本发明涉及从人原始Islet1阳性（ISL1 +）祖细胞产生血管化人心脏组织，更具体地说，从人原代ISL1 + / NKX2.5阳性的人原始Islet1 +心血管干细胞产生血管化人心脏组织 - / KDR-。本发明的一个方面涉及从人多能细胞例如人ES细胞或其他人多能干细胞来源分离人ISL1 +原始细胞，其中人ISL1 +原始细胞可分化成三种不同谱系; 心肌细胞谱系，内皮谱系和平滑肌谱系。另一方面涉及将人原始ISL1 +祖细胞用于和植入动物模型中以产生人血管化的心脏组织，更具体地，涉及血管疾病的体内人源化模型的产生。一个实施方案涉及血管疾病的体内人源化模型的用途作为测定，例如用于评估药物毒性和/或鉴定增加和减少冠心病血液流向人血管化心脏组织的药剂。另一个实施方案涉及人原始ISL1 +祖细胞的治疗用途，例如，在一个实施方案中，本发明提供了治疗受试者心血管疾病和/或先天性心脏病的方法，所述方法包括移植入受试者从人ISL1 +祖细胞产生的血管化人心脏组织。

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