公开(公告)号：US20190134021A1

公开(公告)日：2019-05-09

申请号：US16096536

申请日：2017-04-26

Applicant: The Board of Regents of the University of Texas System

Inventor： Eric N. OLSON ,  Rhonda BASSEL-DUBY ,  Leonela AMOASII

IPC: A61K31/4706 ,  A61P1/16 ,  A61K31/437 ,  A61K38/22 ,  A61K38/28 ,  A61P3/04

Abstract: The present disclosure relates to the identification of Nurr1 as a key regulator of metabolism, and the use of Nurr1 agonist to treat metabolic disorders such as diabetes, obesity, metabolic syndrome and hepatic steatosis.

公开(公告)号：US20160256441A1

公开(公告)日：2016-09-08

申请号：US15029222

申请日：2014-10-16

Applicant: THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM

Inventor： Eric M. SMALL ,  Eric N. OLSON

IPC: A61K31/42 ,  A61K9/08 ,  A61K9/14 ,  A61K45/06 ,  C07K16/18 ,  C12N15/113 ,  A61L17/00 ,  A61L17/06 ,  A61M1/00 ,  A61F13/00 ,  A61G10/02 ,  A61N1/04 ,  A61N5/06 ,  A61N7/00 ,  A61K9/06

CPC classification number: A61K31/42 ,  A61F13/00021 ,  A61G10/026 ,  A61K9/06 ,  A61K9/08 ,  A61K9/14 ,  A61K45/06 ,  A61L15/44 ,  A61L17/005 ,  A61L17/06 ,  A61L26/0066 ,  A61L2300/204 ,  A61L2300/412 ,  A61M1/0088 ,  A61N1/0468 ,  A61N5/062 ,  A61N7/00 ,  C07K16/18 ,  C12N15/113

Abstract: The present disclosure concerns uses for isoxazole compounds or salts or analogs thereof for the treatment of wounds. The present disclosure also concerns devices for delivering a isoxazole compound or salts or an analogs thereof to a wound site.

Abstract translation: 本公开涉及异恶唑化合物或其盐或类似物用于治疗伤口的用途。 本公开还涉及将异恶唑化合物或其盐或其类似物递送至伤口部位的装置。

公开(公告)号：US20160058889A1

公开(公告)日：2016-03-03

申请号：US14823563

申请日：2015-08-11

Applicant: The Board of Regents of the University of Texas System

Inventor： Eric N. OLSON ,  Chengzu LONG ,  John R. MCANALLY ,  John M. SHELTON ,  Rhonda BASSEL-DUBY

IPC: A61K48/00 ,  C12N15/113 ,  A61K38/46

CPC classification number: A61K48/0058 ,  A61K38/465 ,  A61P21/00 ,  C12N15/113 ,  C12N2310/20 ,  C12N2750/14143 ,  C12Y301/00

Abstract: Duchenne muscular dystrophy (DMD) is an inherited X-linked disease caused by mutations in the gene encoding dystrophin, a protein required for muscle fiber integrity. The disclosure reports CRISPR/Cas9-mediated gene editing (Myo-editing) is effective at correcting the dystrophin gene mutation in the mdx mice, a model for DMD. Further, the disclosure reports optimization of germline editing of mdx mice by engineering the permanent skipping of mutant exon (exon 23) and extending exon skipping to also correct the disease by post-natal delivery of adeno-associate virus (AAV). AAV-mediated Myo-editing can efficiently rescue the reading frame of dystrophin in mdx mice in vivo. The disclosure reports means of Myo-editing-mediated exon skipping has been successfully advanced from somatic tissues in mice to human DMD patients-derived iPSCs (induced pluripotent stem cells). Custom Myo-editing was performed on iPSCs from patients with differing mutations and successfully restored dystrophin protein expression for all mutations in iPSCs-derived cardiomyocytes.

Abstract translation: 杜氏肌营养不良（DMD）是由编码肌营养不良蛋白的基因（肌纤维完整性所需的蛋白质）的突变引起的遗传性X连锁疾病。 披露报告CRISPR / Cas9介导的基因编辑（Myo-editing）在校正mdx小鼠中的肌营养不良蛋白基因突变中是有效的，DMD的模型。 此外，该公开报告通过工程化突变外显子（外显子23）的永久性跳过并延伸外显子跳过以通过产后输送腺相关病毒（AAV）来纠正疾病来报告mdx小鼠的种系编辑的优化。 AAV介导的Myo编辑可以有效地挽救体内mdx小鼠肌营养不良蛋白的阅读框架。 Myo编辑介导的外显子跳跃的披露报告手段已经从小鼠的体细胞组织中成功推进到人DMD患者来源的iPSC（诱导多能干细胞）。 在具有不同突变的患者的iPSC上进行定制Myo编辑，并成功地恢复了iPSC来源的心肌细胞中所有突变的肌营养不良蛋白表达。

公开(公告)号：US20150307847A1

公开(公告)日：2015-10-29

申请号：US14697955

申请日：2015-04-28

Applicant: The Board of Regents of the University of Texas System

Inventor： Young-Jae NAM ,  Kunhua SONG ,  Eric N. OLSON

IPC: C12N5/077 ,  A61K45/06 ,  A61K38/17 ,  A61K31/7105

CPC classification number: C12N5/0657 ,  A61K31/7105 ,  A61K38/00 ,  A61K38/17 ,  A61K45/06 ,  C07K14/4702 ,  C12N2501/40 ,  C12N2770/00043

Abstract: The present invention involves the use of transcription factors including Tbx5, Mef2C, Hand2, myocardin and Gata4 to reprogram cardiac fibroblasts into cardiomyocytes, both in vitro and in vivo. Such methods find particular use in the treatment of patients post-myocardial infarction to prevent or limit scarring and to promote myocardial repair.

Abstract translation: 本发明涉及使用包括Tbx5，Mef2C，Hand2，心肌素和Gata4在内的转录因子在体外和体内将心脏成纤维细胞重编程为心肌细胞。 这种方法特别用于治疗心肌梗塞后的患者，以预防或限制瘢痕形成并促进心肌修复。