公开(公告)号：WO2013003475A1

公开(公告)日：2013-01-03

申请号：PCT/US2012/044418

申请日：2012-06-27

Applicant: CELLSCRIPT, INC. ,  CHIN, Cynthia ,  PERSON, Anthony D. ,  DAHL, Gary A.

Inventor： CHIN, Cynthia ,  PERSON, Anthony D. ,  DAHL, Gary A.

IPC: C07K14/07 ,  C07K14/715 ,  C07K14/705 ,  A61K38/16 ,  A61K31/7088

CPC classification number: C12N5/0634 ,  A61K31/7105 ,  A61K31/711 ,  A61K38/162 ,  A61K38/1774 ,  A61K38/21 ,  C07K14/005 ,  C07K14/70503 ,  C07K14/7156 ,  C12N5/0658 ,  C12N5/0696 ,  C12N2710/20022 ,  C12N2710/22022 ,  C12N2710/24122 ,  C12N2760/16122 ,  A61K2300/00

Abstract: The present invention provides methods, kits, and compositions for reducing an innate immune system response in a human or animal cell, tissue or organism. One embodiment comprises: introducing an Agent mRNA comprising in vitro -synthesized mRNA encoding one or more proteins that affect the induction, activity or response of an innate immune response pathway; whereby, the innate immune response in the cell, tissue or organism is reduced compared to the innate immune response in the absence of the Agent mRNA. Other embodiments are methods, compositions and kits for using an Agent mRNA for treating a disease or medical condition in a human or animal that exhibits symptoms of an elevated innate immune system, or for reducing an innate immune response that is induced in a human or animal cell, tissue or organism by a Foreign Substance that is administered to the cell, tissue or organism.

Abstract translation: 本发明提供用于减少人或动物细胞，组织或生物体内的先天免疫系统应答的方法，试剂盒和组合物。 一个实施方案包括：引入包含影响先天免疫应答途径的诱导，活性或应答的一种或多种蛋白质的体外合成mRNA的试剂mRNA; 由此，在不存在Agent mRNA的情况下，与先天免疫应答相比，细胞，组织或生物体中的先天免疫应答降低。 其它实施方案是使用试剂mRNA用于治疗显示出先天免疫系统升高的症状的人或动物中的疾病或医学病症的方法，组合物和试剂盒，或用于减少在人或动物中诱导的先天免疫应答 细胞，组织或生物体通过给予细胞，组织或生物体的外源物质。

公开(公告)号：WO2013102203A1

公开(公告)日：2013-07-04

申请号：PCT/US2012/072301

申请日：2012-12-31

Applicant: CELLSCRIPT, INC. ,  MEIS, Judith ,  PERSON, Anthony ,  CHIN, Cynthia ,  JENDRISAK, Jerome ,  DAHL, Gary

Inventor： MEIS, Judith ,  PERSON, Anthony ,  CHIN, Cynthia ,  JENDRISAK, Jerome ,  DAHL, Gary

IPC: C12N15/10 ,  C12N15/08 ,  C12P19/34 ,  A61K31/7105

CPC classification number: A61K48/0066 ,  A61K31/7105 ,  A61K33/06 ,  A61K38/005 ,  A61K48/0041 ,  C12N5/0619 ,  C12N5/0658 ,  C12N5/0696 ,  C12N15/1003 ,  C12N15/1096 ,  C12N15/87 ,  C12N2501/60 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/605 ,  C12N2501/606 ,  C12N2501/608 ,  C12N2501/73 ,  C12N2506/1307 ,  C12N2510/00 ,  C12P19/34 ,  C12P19/38 ,  C12P21/02 ,  C12Y301/26003 ,  A61K2300/00

Abstract: The present invention relates to compositions, kits and methods for making and using RNA compositions comprising in vitro-synthesized ssRNA inducing a biological or biochemical effect in a mammalian cell or organism into which the RNA composition is repeatedly or continuously introduced. In certain embodiments, the invention provides compositions and methods for changing the state of differentiation or phenotype of a human or other vertebrate cell. For example, the present invention provides mRNA and methods for reprogramming cells that exhibit a first differentiated state or phenotype to cells that exhibit a second differentiated state or phenotype, such as to reprogram human somatic cells to pluripotent stem cells.

Abstract translation: 本发明涉及用于制备和使用RNA组合物的组合物，试剂盒和方法，所述RNA组合物包含在哺乳动物细胞或生物体中诱导生物或生物化学作用的体外合成的ssRNA，RNA组合物重复或连续引入。 在某些实施方案中，本发明提供用于改变人或其他脊椎动物细胞的分化或表型状态的组合物和方法。 例如，本发明提供mRNA和方法，用于对表现出第二分化状态或表型的细胞，例如将人体细胞重编程为多能干细胞，对第一分化状态或表型展现细胞进行重编程。