公开(公告)号：WO2017120089A1

公开(公告)日：2017-07-13

申请号：PCT/US2016/069079

申请日：2016-12-29

Applicant: WARREN, Luigi

Inventor： WARREN, Luigi

IPC: A61K48/00 ,  A01N63/00 ,  C12N15/00 ,  C12N5/02 ,  C07H21/04

CPC classification number: C12N5/0696 ,  C07K14/4702 ,  C12N15/00 ,  C12N2501/60 ,  C12N2501/602 ,  C12N2506/1307 ,  C12N2510/00

Abstract: Methods for accelerated cell lineage conversion and the treatment of patients with the lineage converted cells are provided. The methods include the steps of transfecting a cell with a composition that includes at least one synthetic mRNA encoding a chimeric protein that corresponds to an engineered fusion of a transcription factor and an heterologous peptide sequence derived from the C- terminal transactivation domain (TAD) of Yes-associated protein (YAP). The YAP domain enhances the epigenetic remodeling activity of the chimeric protein increasing the speed of lineage conversion. The converted cells may be used for research or administered to a human or animal patient as a therapy. The reprogramming of a somatic cell to pluripotency is accelerated by using a cocktail of mRNAs expressing a combination of reprogramming factors expressed as YAP TAD chimeras.

Abstract translation: 提供加速细胞谱系转化和用谱系转化细胞治疗患者的方法。 所述方法包括用包含至少一种合成mRNA的组合物转染细胞的步骤，所述合成mRNA编码嵌合蛋白，所述嵌合蛋白对应于转录因子和源自C末端反式激活结构域（TAD）的异源肽序列的工程融合 是相关蛋白（YAP）。 YAP结构域增强了嵌合蛋白的表观遗传重构活性，增加了谱系转换的速度。 转化的细胞可用于研究或作为治疗施用给人或动物患者。 通过使用表达重编程因子组合的mRNA混合物表达为YAP TAD嵌合体，可加速体细胞重编程为多能性。

公开(公告)号：WO2019241569A1

公开(公告)日：2019-12-19

申请号：PCT/US2019/037069

申请日：2019-06-13

Applicant: WARREN, Luigi

Inventor： WARREN, Luigi

IPC: A61K35/545 ,  C07K14/47 ,  C12N5/074

Abstract: Methods for accelerated cell lineage conversion and the treatment of patients with the lineage converted cells are provided. The methods include the steps of transfecting a cell with a composition that includes at least one synthetic mRNA encoding a chimeric protein that corresponds to an engineered fusion of a transcription factor and an heterologous peptide sequence derived from the C-terminal TAD of Gal4. The TAD domain enhances the epigenetic remodeling activity of the chimeric protein increasing the speed of lineage conversion. The converted cells may be used for research or administered to a human or animal patient as a therapy. In one preferred embodiment, the reprogramming of a somatic cell to pluripotency is accelerated by using a cocktail of mRNAs expressing a combination of wild-type or engineered reprogramming factors where Oct4 and/or Sox2 and/or Nanog are expressed as Gal4 TAD chimeras.

公开(公告)号：WO2011130624A2

公开(公告)日：2011-10-20

申请号：PCT/US2011/032679

申请日：2011-04-15

Applicant: IMMUNE DISEASE INSTITUTE, INC. ,  ROSSI, Derrick ,  WARREN, Luigi

Inventor： ROSSI, Derrick ,  WARREN, Luigi

IPC: C12N5/10 ,  C12N15/113 ,  C12N5/074 ,  C07H21/00

CPC classification number: C12N5/0696 ,  C07K14/435 ,  C12N15/11 ,  C12N15/111 ,  C12N2310/14 ,  C12N2320/30

Abstract: Described herein are synthetic, modified RNAs for changing the phenotype of a cell, such as expressing a polypeptide or altering the developmental potential. Accordingly, provided herein are compositions, methods, and kits comprising synthetic, modified RNAs for changing the phenotype of a cell or cells. These methods, compositions, and kits comprising synthetic, modified RNAs can be used either to express a desired protein in a cell or tissue, or to change the differentiated phenotype of a cell to that of another, desired cell type.

Abstract translation: 本文描述了用于改变细胞表型的合成的，修饰的RNA，例如表达多肽或改变发育潜能。 因此，本文提供的是包含用于改变细胞或细胞的表型的合成的，修饰的RNA的组合物，方法和试剂盒。 包含合成的修饰的RNA的这些方法，组合物和试剂盒可用于在细胞或组织中表达所需的蛋白质，或将细胞的分化表型改变为另一种所需细胞类型的分化表型。

公开(公告)号：WO2011130624A3

公开(公告)日：2012-02-02

申请号：PCT/US2011032679

申请日：2011-04-15

Applicant: IMMUNE DISEASE INST INC ,  ROSSI DERRICK ,  WARREN LUIGI

Inventor： ROSSI DERRICK ,  WARREN LUIGI

IPC: C12N5/10 ,  C07H21/00 ,  C12N5/074 ,  C12N15/113

CPC classification number: C12N5/0696 ,  C07K14/435 ,  C12N15/11 ,  C12N15/111 ,  C12N2310/14 ,  C12N2320/30

Abstract: Described herein are synthetic, modified RNAs for changing the phenotype of a cell, such as expressing a polypeptide or altering the developmental potential. Accordingly, provided herein are compositions, methods, and kits comprising synthetic, modified RNAs for changing the phenotype of a cell or cells. These methods, compositions, and kits comprising synthetic, modified RNAs can be used either to express a desired protein in a cell or tissue, or to change the differentiated phenotype of a cell to that of another, desired cell type.

Abstract translation: 本文描述了用于改变细胞表型的合成的，修饰的RNA，例如表达多肽或改变发育潜能。 因此，本文提供的是包含用于改变细胞或细胞的表型的合成的，修饰的RNA的组合物，方法和试剂盒。 包含合成的修饰的RNA的这些方法，组合物和试剂盒可用于在细胞或组织中表达所需的蛋白质，或将细胞的分化表型改变为另一种所需细胞类型的分化表型。