公开(公告)号：US20160151491A1

公开(公告)日：2016-06-02

申请号：US15008376

申请日：2016-01-27

申请人： Yale University

发明人： Peter M. Rabinovich ,  Sherman M. Weissman ,  Marina E. Komarovskaya ,  Erkut Bahceci ,  Samuel Katz ,  Efim Golub

IPC分类号： A61K39/395 ,  A61K35/12 ,  A61K45/06 ,  C12N15/85

CPC分类号： A61K39/39558 ,  A61K35/15 ,  A61K35/17 ,  A61K39/0011 ,  A61K45/06 ,  A61K48/005 ,  A61K2039/5156 ,  A61K2039/5158 ,  C12N5/0635 ,  C12N5/0638 ,  C12N5/0646 ,  C12N5/0696 ,  C12N15/85 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/606 ,  C12N2506/094 ,  C12N2506/1307 ,  C12N2510/00 ,  C12N2830/008

摘要： Compositions and methods of making cells using RNA, and cells made using the disclosed compositions and methods are also provided. In exemplary embodiments, RNA is transfected into cells to effect a molecular, biological, physiological, or histological change in the cells. In preferred embodiments, the RNA is prepared in vitro, more preferably using a DNA template according to the provided compositions and methods. Methods for treating or inhibiting a disorder or disease such cancer are also provided. The methods can include, for example, locally or systemically administering to the host an effective amount of one or more RNAs; or an effective amount of population of cells isolated from the subject or a syngeneic or histocompatible subject, contacted ex vivo with one or RNAs, and optionally expanded. The cells can be, for example, immune cells or stem cells.

摘要翻译： 还提供了使用RNA制备细胞的组合物和方法，以及使用所公开的组合物和方法制备的细胞。 在示例性实施方案中，将RNA转染到细胞中以实现细胞中的分子，生物学，生理学或组织学变化。 在优选的实施方案中，RNA在体外制备，更优选根据所提供的组合物和方法使用DNA模板。 还提供了治疗或抑制疾病或疾病的方法。 所述方法可以包括例如局部或全身给予宿主有效量的一种或多种RNA; 或从受试者或同基因或组织相容性受试者分离的有效量的细胞群体，用一种或多种RNA离体接触，并任选地扩增。 细胞可以是例如免疫细胞或干细胞。

公开(公告)号：US10155038B2

公开(公告)日：2018-12-18

申请号：US15008376

申请日：2016-01-27

申请人： Yale University

发明人： Peter M. Rabinovich ,  Sherman M. Weissman ,  Marina E. Komarovskaya ,  Erkut Bahceci ,  Samuel Katz ,  Efim Golub

IPC分类号： A61K9/66 ,  A61K39/395 ,  C12N15/85 ,  A61K45/06 ,  A61K48/00 ,  C12N5/074 ,  A61K39/00 ,  C12N5/0781 ,  C12N5/0783 ,  A61K35/15 ,  A61K35/17

摘要： Compositions and methods of making cells using RNA, and cells made using the disclosed compositions and methods are also provided. In exemplary embodiments, RNA is transfected into cells to effect a molecular, biological, physiological, or histological change in the cells. In preferred embodiments, the RNA is prepared in vitro, more preferably using a DNA template according to the provided compositions and methods. Methods for treating or inhibiting a disorder or disease such cancer are also provided. The methods can include, for example, locally or systemically administering to the host an effective amount of one or more RNAs; or an effective amount of population of cells isolated from the subject or a syngeneic or histocompatible subject, contacted ex vivo with one or RNAs, and optionally expanded. The cells can be, for example, immune cells or stem cells.

公开(公告)号：US10017782B2

公开(公告)日：2018-07-10

申请号：US15012285

申请日：2016-02-01

申请人： Yale University

发明人： Peter M. Rabinovich ,  Sherman M. Weissman ,  Erkut Bahceci ,  Marina E. Komarovskaya

IPC分类号： A61K9/66 ,  C12N15/85 ,  A61K48/00 ,  C12N5/074 ,  A61K35/17 ,  A61K35/33 ,  A61K35/36 ,  A61K35/12

CPC分类号： C12N15/85 ,  A61K35/17 ,  A61K35/33 ,  A61K35/36 ,  A61K48/005 ,  A61K2035/124 ,  C12N5/0696 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/606 ,  C12N2506/094 ,  C12N2506/1307 ,  C12N2510/00

摘要： RNA prepared by in vitro transcription using a polymerase chain reaction (PCR)-generated template can be introduced into a cell to modulate cell activity. This method is useful in de-differentiating somatic cells to pluripotent, multipotent, or unipotent cells; re-differentiating stem cells into differentiated cells; or reprogramming of somatic cells to modulate cell activities such as metabolism. Cells can also be transfected with inhibitory RNAs, such as small interfering RNA (siRNA) or micro RNA (miRNA), or combinations thereof to induce reprogramming of somatic cells. For example, target cells are isolated from a donor, contacted with one or more RNA's causing the cells to be de-differentiated, re-differentiated, or reprogrammed in vitro, and administered to a patient in need thereof. The resulting cells are useful for treating one or more symptoms of a variety of diseases and disorders, for organ regeneration, and for restoration of the immune system.