公开(公告)号：US20190338311A1

公开(公告)日：2019-11-07

申请号：US16476137

申请日：2018-01-05

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

Inventor： Leonela AMOASII ,  Eric OLSON

IPC: C12N15/86 ,  C12N15/113 ,  C07K14/47 ,  C12N9/22 ,  C12N15/90 ,  C12N5/077 ,  C12N5/074

Abstract: CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene. Here, using three promoters to drive expression of the same DMD guide RNA, a more robust and safe form of genome editing was achieved in a humanized mouse model for DMD with a deletion 13 in exon 50, and in a ΔEx50-MD Dog.

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.