公开(公告)号：US20140363380A1

公开(公告)日：2014-12-11

申请号：US14212715

申请日：2014-03-14

Applicant: Wake Forest University Health Sciences

Inventor： Baisong Lu ,  Colin E. Bishop

IPC: A61K49/00 ,  G01N30/00 ,  G01N21/25 ,  A61K31/713 ,  G01N33/68

CPC classification number: A61K49/0008 ,  A61K31/713 ,  G01N21/25 ,  G01N30/00 ,  G01N33/5023 ,  G01N33/6893 ,  G01N2800/04 ,  G01N2800/042 ,  G01N2800/044

Abstract: MEX3C deficiency impairs the development of white and brown adipose tissue. Hence the present invention provides, among other things, a method of screening a candidate compound for activity in inhibiting fat deposition in a subject in need thereof and/or treating a condition in a subject in need thereof, comprising: (a) contacting a candidate compound to a cell that expresses MEX3C protein; and then (b) detecting a quantity of expression of the MEX3C protein in the cell; a depression in the expression of MEX3C protein when the candidate compound is contacted thereto as compared to that expressed when the candidate compound is not contacted thereto indicating the compound is active in inhibiting fat deposition and/or treating a condition in a subject in need thereof. Methods of treatment and screening subjects are also described.

Abstract translation: MEX3C缺乏会损害白色和棕色脂肪组织的发育。 因此，本发明尤其提供了筛选候选化合物以抑制有需要的受试者中的脂肪沉积的活性的方法和/或治疗有需要的受试者的状况，其包括：（a）使候选者 化合物表达MEX3C蛋白的细胞; 然后（b）检测细胞中MEX3C蛋白的表达量; 当候选化合物与其接触时，与候选化合物不接触时所表达的MEX3C蛋白表达的抑制相反，表明该化合物在抑制脂肪沉积和/或治疗有需要的受试者中的病症方面是有活性的。 还描述了治疗和筛选受试者的方法。

公开(公告)号：US20240158808A1

公开(公告)日：2024-05-16

申请号：US18289875

申请日：2022-05-20

Applicant: Wake Forest University Health Sciences

Inventor： Baisong Lu ,  Anthony Atala

IPC: C12N15/86 ,  A01K67/0276 ,  A61K48/00 ,  A61P13/12 ,  C07K14/47

CPC classification number: C12N15/86 ,  A01K67/0276 ,  A61K48/0058 ,  A61K48/0075 ,  A61P13/12 ,  C07K14/47 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/03 ,  C12N2740/16043 ,  C12N2800/22 ,  C12N2830/008

Abstract: The present invention includes methods and compositions useful for the treatment of Dent's disease in a subject in need thereof. The invention of the present disclosure also includes a mouse model useful for the study of Dent's disease.

公开(公告)号：US20210047375A1

公开(公告)日：2021-02-18

申请号：US17051296

申请日：2019-05-01

Applicant: Wake Forest University Health Sciences

Inventor： Baisong Lu ,  Anthony Atala

IPC: C07K14/155 ,  C12N9/22 ,  C12N15/86 ,  C12N15/113

Abstract: Provided are compositions, systems, and methods useful for effecting gene editing in eukaryotic cells. Compositions include plasmids that encode one or more viral fusion proteins in which one or more viral proteins are fused with an aptamer-binding protein. Compositions also include plasmids that encode a non-viral nucleic acid sequence, wherein the non-viral nucleic acid sequence encodes a CRISPR system component. In some instances, the non-viral nucleic acid sequence also includes an aptamer sequence. The plasmids can be used to generate viral particles, including lentivirus-like particles that contain a viral fusion protein and a non-viral RNA sequence. Systems of producing such viral particles are provided. Also provided are methods of using the viral particles of the disclosure to effect gene editing in eukaryotic cells.

公开(公告)号：US09879278B2

公开(公告)日：2018-01-30

申请号：US14382816

申请日：2013-02-27

Applicant: WAKE FOREST UNIVERSITY HEALTH SCIENCES

Inventor： Baisong Lu ,  Qingguo Zhao ,  James Yoo ,  Anthony Atala

IPC: C12N15/85

CPC classification number: C12N15/85 ,  C12N2800/40 ,  C12N2840/007

Abstract: The invention includes compositions and methods for the selective expression of a target gene in a subset of cells. In certain embodiments, the present invention includes a construct comprising a first nucleic acid sequence comprising an episomal maintenance element and a second nucleic acid sequence comprising a target gene wherein the expression of the episomal maintenance element is regulated by a constitutive promoter and the expression of the target gene is regulated by a non-constitutive promoter. The construct is able to maintain episomal state, no matter whether the target gene is expressed in the cell.

公开(公告)号：US20150079682A1

公开(公告)日：2015-03-19

申请号：US14382816

申请日：2013-02-27

Applicant: WAKE FOREST UNIVERSITY HEALTH SCIENCES

Inventor： Baisong Lu ,  Qingguo Zhao ,  James Yoo ,  Anthony Atala

IPC: C12N15/85

CPC classification number: C12N15/85 ,  C12N2800/40 ,  C12N2840/007

Abstract: The invention includes compositions and methods for the selective expression of a target gene in a subset of cells. In certain embodiments, the present invention includes a construct comprising a first nucleic acid sequence comprising an episomal maintenance element and a second nucleic acid sequence comprising a target gene wherein the expression of the episomal maintenance element is regulated by a constitutive promoter and the expression of the target gene is regulated by a non-constitutive promoter. The construct is able to maintain episomal state, no matter whether the target gene is expressed in the cell.

Abstract translation: 本发明包括用于在细胞子集中选择性表达靶基因的组合物和方法。 在某些实施方案中，本发明包括构建体，其包含第一核酸序列，其包含附加型维持元件和包含靶基因的第二核酸序列，其中所述附加型维持元件的表达由组成型启动子调节， 靶基因由非组成型启动子调控。 无论目标基因是否在细胞中表达，该构建体能够保持附加型状态。