公开(公告)号：US11999964B2

公开(公告)日：2024-06-04

申请号：US17460120

申请日：2021-08-27

Applicant: California Institute of Technology

Inventor： Yitong Ma ,  Mark W. Budde ,  Michaelle N. Mayalu ,  Michael B. Elowitz

IPC: C12N15/85 ,  C07K14/415 ,  C12N9/02 ,  C12N9/86 ,  C12N15/11

CPC classification number: C12N15/85 ,  C07K14/415 ,  C12N9/0069 ,  C12N9/86 ,  C12N15/11 ,  C12Y113/12003 ,  C07K2319/00 ,  C12N2310/20 ,  C12N2800/107 ,  C12N2810/10

Abstract: Disclosed herein include circuits, compositions, nucleic acids, populations, systems, and methods enabling cells to sense, control, and/or respond to their own population size. In some embodiments, an orthogonal communication channel allows specific communication between engineered cells. Also described herein, in some embodiments, is an evolutionarily robust ‘paradoxical’ regulatory circuit architecture in which orthogonal signals both stimulate and inhibit net cell growth at different signal concentrations. In some embodiments, engineered cells autonomously reach designed densities and/or activate therapeutic or safety programs at specific density thresholds. Methods of treatment are also provided in some embodiments.

公开(公告)号：US11898270B2

公开(公告)日：2024-02-13

申请号：US16628481

申请日：2017-08-21

Applicant: HUAZHONG AGRICULTURAL UNIVERSITY

Inventor： Shuhong Zhao ,  Shengsong Xie ,  Changzhi Zhao ,  Xinyun Li ,  Xiangdong Liu ,  Xiaosong Han ,  Gaojuan Yang ,  Yang Gao ,  Yilong Chen ,  Xiaoyong Du ,  Yiliang Miao ,  Yunlong Ma ,  Xiaolei Liu

IPC: C12N15/90 ,  C40B40/02 ,  C12N9/22 ,  C12N15/113 ,  C12N15/85 ,  C12Q1/6869 ,  C40B40/06 ,  C40B50/06

CPC classification number: C40B40/02 ,  C12N9/22 ,  C12N15/113 ,  C12N15/85 ,  C12N15/907 ,  C12Q1/6869 ,  C40B40/06 ,  C40B50/06 ,  C12N2810/10

Abstract: Provided is a pig genome-wide specific sgRNA library, a preparation method therefor, and an application thereof. The sgRNA is targeted at a pig genome-wide protein-coding gene, lincRNA and/or miRNA. Specifically, an sgRNA construct has the following structure: AL-N20-AR, wherein AL is the left homology arm sequence located at the upstream of the coding sequence of a pig specific SgRNA, N20 is the coding sequence of the pig specific SgRNA, and AR is a right homology arm sequence located at the downstream of the coding sequence of the pig specific sgRNA. The sgRNA library can be used for screening functional genes of a pig or for preparing a kit.

公开(公告)号：US20180251792A1

公开(公告)日：2018-09-06

申请号：US15980450

申请日：2018-05-15

Applicant: EDITAS MEDICINE, INC.

Inventor： Ari E. Friedland ,  Hariharan Jayaram ,  Barrett Ethan Steinberg

IPC: C12N15/90 ,  C12N9/96 ,  C12N9/22 ,  C12N15/11 ,  C12N15/10

CPC classification number: C12N15/907 ,  C12N9/22 ,  C12N9/96 ,  C12N15/102 ,  C12N15/11 ,  C12N15/113 ,  C12N15/63 ,  C12N2310/20 ,  C12N2810/10

Abstract: Engineered nucleic acids encoding genome editing system components are provided, as are engineered RNA-guided nucleases that include inserts encoded in part by cellular genomic or other sequences recognized by guide RNAs.

公开(公告)号：US09963719B1

公开(公告)日：2018-05-08

申请号：US15832567

申请日：2017-12-05

Applicant: EDITAS MEDICINE, INC.

Inventor： Ari E. Friedland ,  Hariharan Jayaram ,  Barrett Ethan Steinberg

IPC: C12N15/90 ,  C12N9/96 ,  C12N9/22 ,  C12N15/10 ,  C12N15/11

CPC classification number: C12N15/907 ,  C12N9/22 ,  C12N9/96 ,  C12N15/102 ,  C12N15/11 ,  C12N15/113 ,  C12N15/63 ,  C12N2310/20 ,  C12N2810/10

Abstract: Engineered nucleic acids encoding genome editing system components are provided, as are engineered RNA-guided nucleases that include inserts encoded in part by cellular genomic or other sequences recognized by guide RNAs.

公开(公告)号：US20170260544A1

公开(公告)日：2017-09-14

申请号：US15603014

申请日：2017-05-23

Applicant: Regeneron Pharmaceuticals, Inc.

Inventor： Aris N. Economides ,  Andrew J. Murphy ,  David M. Valenzuela ,  David Frendewey ,  George D. Yancopoulos

IPC: C12N15/85 ,  C12N5/0735 ,  C12Q1/68 ,  C12N15/90

CPC classification number: C12N15/85 ,  A01K67/0275 ,  A01K2217/05 ,  A01K2227/105 ,  C12N5/0606 ,  C12N15/79 ,  C12N15/907 ,  C12N2510/00 ,  C12N2810/10 ,  C12Q1/6827

Abstract: A method for engineering and utilizing large DNA vectors to target, via homologous recombination, and modify, in any desirable fashion, endogenous genes and chromosomal loci in eukaryotic cells. These large DNA targeting vectors for eukaryotic cells, termed LTVECs, are derived from fragments of cloned genomic DNA larger than those typically used by other approaches intended to perform homologous targeting in eukaryotic cells. Also provided is a rapid and convenient method of detecting eukaryotic cells in which the LTVEC has correctly targeted and modified the desired endogenous gene(s) or chromosomal locus (loci) as well as the use of these cells to generate organisms bearing the genetic modification.

公开(公告)号：US20170198269A1

公开(公告)日：2017-07-13

申请号：US15330876

申请日：2016-11-07

Applicant: THE BROAD INSTITUTE INC. ,  MASSACHUSETTS INSTITUTE OF TECHNOLOGY ,  PRESIDENT AND FELLOWS OF HARVARD COLLEGE

Inventor： Feng Zhang ,  Fei Ran ,  Ophir Shalem

IPC: C12N9/22 ,  C12N15/90 ,  C12N15/82 ,  C12N9/96 ,  C12N15/86

CPC classification number: C12N9/22 ,  A61K48/00 ,  C12N9/96 ,  C12N15/1082 ,  C12N15/63 ,  C12N15/8213 ,  C12N15/85 ,  C12N15/86 ,  C12N15/907 ,  C12N2750/14143 ,  C12N2800/22 ,  C12N2810/10

Abstract: The invention provides for engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are compositions and methods related to components of a CRISPR complex particularly comprising a Cas ortholog enzyme.

公开(公告)号：US09655933B2

公开(公告)日：2017-05-23

申请号：US14384473

申请日：2013-03-15

Applicant: RAMOT AT TEL-AVIV UNIVERSITY LTD.

Inventor： Beka Solomon ,  Eyal Dor-On

IPC: A61K35/76 ,  C12N7/00

CPC classification number: A61K35/76 ,  C12N2795/14132 ,  C12N2795/14171 ,  C12N2810/10 ,  C12N2810/40 ,  Y02A50/473

Abstract: The invention relates to a filamentous bacteriophage, carrying bacterial Lipopolysaccharide (LPS) endotoxin on its surface, for use in treating cancer and in inhibiting angiogenesis, and in a different context, for use in promoting angiogenesis in diseases or conditions in which there is insufficient angiogenesis.

公开(公告)号：US20170088819A1

公开(公告)日：2017-03-30

申请号：US15311579

申请日：2015-05-18

Applicant: VRIJE UNIVERSITEIT BRUSSEL ,  THE GENERAL HOSPITAL CORPORATION

Inventor： Thierry VANDENDRIESSCHE ,  Marinee CHUAH ,  J. Keith JOUNG ,  Yanfang FU ,  Deepak REYON

IPC: C12N5/074 ,  C12N9/12 ,  A61K35/34 ,  C12N15/90

CPC classification number: C12N5/0696 ,  A61K35/34 ,  A61K48/00 ,  A61K2039/515 ,  A61K2039/53 ,  C12N5/0658 ,  C12N9/1205 ,  C12N15/113 ,  C12N15/907 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2800/80 ,  C12N2810/10

Abstract: The invention relates to polynucleotides suitable for reducing or eliminating the expression of expanded repeat RNA (CUGexp) of the dystrophy myotonic-protein kinase (DMPK) gene in a cell of a DM-1 patient. The polynucleotides are a combination of a polynucleotide for a site specific nuclease targeting the dystrophy myotonic-protein kinase (DMPK) gene locus, and a donor polynucleotide having 5′ and 3′ regions which are homologous with the sequence of DMPK gene which flank the target site of the nuclease. The invention further relate to in vivo and in vitro methods to reduce or eliminate CTG repeats in the DMPK gene. The invention further relates to the medical use of polynucleotides and cells for treating DM-1 patient.

公开(公告)号：US20170067078A1

公开(公告)日：2017-03-09

申请号：US15354270

申请日：2016-11-17

Applicant: Regeneron Pharmaceuticals, Inc.

Inventor： David Frendewey ,  Wojtek Auerbach ,  Ka-Man Venus Lai ,  David M. Valenzuela ,  George D. Yancopoulos

IPC: C12N15/85 ,  A01K67/027 ,  C12N15/90

CPC classification number: C12N15/8509 ,  A01K67/0276 ,  A01K67/0278 ,  A01K2217/072 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/0362 ,  A01K2267/0387 ,  C12N9/22 ,  C12N15/1024 ,  C12N15/85 ,  C12N15/907 ,  C12N2015/8527 ,  C12N2800/40 ,  C12N2810/00 ,  C12N2810/10 ,  C12N2810/40 ,  C12N2999/007

Abstract: Compositions and methods are provided for modifying a genomic locus of interest in a eukaryotic cell, a mammalian cell, a human cell or a non-human mammalian cell using a large targeting vector (LTVEC) comprising various endogenous or exogenous nucleic acid sequences as described herein. Further methods combine the use of the LTVEC with a CRISPR/Cas system. Compositions and methods for generating a genetically modified non-human animal comprising one or more targeted genetic modifications in their germline are also provided.

Abstract translation: 提供组合物和方法用于使用包含本文所述的各种内源或外源核酸序列的大靶向载体（LTVEC）修饰真核细胞，哺乳动物细胞，人细胞或非人哺乳动物细胞中的感兴趣的基因组座位。 。 其他方法将LTVEC的使用与CRISPR / Cas系统结合使用。 还提供了用于产生遗传修饰的非人动物的组合物和方法，其包含其种系中的一种或多种靶向遗传修饰。

公开(公告)号：US09526693B2

公开(公告)日：2016-12-27

申请号：US13049825

申请日：2011-03-16

Applicant: Tariq M. Rana

Inventor： Tariq M. Rana

IPC: A61K9/19 ,  A61K9/00 ,  A61K47/48 ,  C12N15/87

CPC classification number: A61K47/645 ,  A61K9/0019 ,  A61K47/542 ,  A61K47/543 ,  A61K47/549 ,  A61K47/6455 ,  C12N15/113 ,  C12N15/87 ,  C12N2310/113 ,  C12N2310/321 ,  C12N2310/3515 ,  C12N2810/10

Abstract: Provided are compositions and methods for delivery of therapeutic agents, such as chemically stabilized antisense oligonucleotides useful in RNA silencing. The compositions include interfering nanoparticles (iNOPs) associated with one or more agents. Several functional iNOP derivatives are provided which allow for targeted delivery of agents to specific cell types as well as exhibiting reduced cellular toxicity.

Abstract translation: 提供用于递送治疗剂的组合物和方法，例如可用于RNA沉默的化学稳定的反义寡核苷酸。 组合物包括与一种或多种试剂相关的干扰性纳米颗粒（iNOP）。 提供了几种功能性的iNOP衍生物，其允许将药物靶向递送至特定细胞类型以及显示降低的细胞毒性。