公开(公告)号：WO2015153940A1

公开(公告)日：2015-10-08

申请号：PCT/US2015/024196

申请日：2015-04-03

Applicant: MASSACHUSETTS INSTITUTE OF TECHNOLOGY

Inventor： LU, Timothy, Kuan-Ta ,  NISSIM, Lior ,  PERLI, Samuel, David

IPC: C12N15/63

CPC classification number: C12N15/113 ,  C12N9/22 ,  C12N15/111 ,  C12N15/63 ,  C12N15/85 ,  C12N2310/128 ,  C12N2310/141 ,  C12N2310/20 ,  C12N2310/51 ,  C12N2330/51 ,  C12N2830/42 ,  C12N2830/60 ,  C12N2830/85 ,  C12Y301/00

Abstract: Various aspects and embodiments of the present disclosure relate to methods and compositions that combine multiple mammalian RNA regulatory strategies, including RNA triple helix structures, introns, microRNAs, and ribozymes with Cas-based CRISPR trascription factors and ribonuclease -based RNA processing in human cells. The methods and compositions of the present disclosure, in some embodiments, enable multiplexed production of proteins and multiple guide RNAs from a single compact RNA-polymerase-II-expressed transcript for efficient modulation of synthetic constructs and endogenous human promoters.

Abstract translation: 本公开的各个方面和实施方案涉及将多种哺乳动物RNA调控策略（包括RNA三螺旋结构，内含子，微RNA和核酶）与基于Cas-based CRISPR trascription因子和基于核糖核酸酶的RNA加工在人类细胞中的方法和组合物。 在一些实施方案中，本公开的方法和组合物使得能够从单个紧凑的RNA聚合酶-II表达的转录物多肽产生蛋白质和多个引导RNA，以有效调节合成构建体和内源性人启动子。

公开(公告)号：WO2018169901A8

公开(公告)日：2018-09-20

申请号：PCT/US2018/022093

申请日：2018-03-13

Applicant: MASSACHUSETTS INSTITUTE OF TECHNOLOGY

Inventor： LU, Timothy, Kuan-Ta ,  NISSIM, Lior ,  WU, Ming-Ru

IPC: C12N15/85 ,  C12N15/869 ,  A61K48/00

Abstract: Aspects of the present disclosure provide synthetic promoters that are differentially modulated between certain diseased cells (e.g., cancer cells) and normal cells (e.g., non-cancer cells). These synthetic promoters are useful, for example, for targeted expression of therapeutic molecules in diseased cells.

公开(公告)号：WO2018169901A1

公开(公告)日：2018-09-20

申请号：PCT/US2018/022093

申请日：2018-03-13

Applicant: MASSACHUSETTS INSTITUTE OF TECHNOLOGY

Inventor： LU, Timothy, Kuan-ta ,  NISSIM, Lior ,  WU, Ming-ru

IPC: C12N15/85 ,  C12N15/869 ,  A61K48/00

Abstract: Aspects of the present disclosure provide synthetic promoters that are differentially modulated between certain diseased cells (e.g., cancer cells) and normal cells (e.g., non-cancer cells). These synthetic promoters are useful, for example, for targeted expression of therapeutic molecules in diseased cells.

公开(公告)号：WO2016205737A2

公开(公告)日：2016-12-22

申请号：PCT/US2016/038222

申请日：2016-06-17

Applicant: MASSACHUSETTS INSTITUTE OF TECHNOLOGY

Inventor： LU, Timothy, Kuan-Ta ,  NISSIM, Lior ,  WU, Ming-Ru

IPC: C12Q1/68 ,  G01N33/68

CPC classification number: G01N33/6845

Abstract: Aspects of the present disclosure provide a platform that triggers potent and effective immunotherapy against tumors from within tumors themselves, thus overcoming limitations of existing cancer immunotherapies and tumor-detecting gene circuits.

Abstract translation: 本公开的方面提供了一个平台，其触发对肿瘤本身内的肿瘤的有效和有效的免疫治疗，从而克服了现有的癌症免疫疗法和肿瘤检测基因电路的限制。