公开(公告)号：US09885018B1

公开(公告)日：2018-02-06

申请号：US15072122

申请日：2016-03-16

Applicant: The Regents of the University of Colorado, a body corporate

Inventor： Kunhua Song ,  Yuanbiao Zhao ,  Pilar Londono ,  Timothy McKinsey

IPC: C12N5/077 ,  A61K48/00 ,  A61K31/713 ,  A61K38/17 ,  A61K45/06

CPC classification number: C12N5/0657 ,  A61K31/713 ,  A61K38/1709 ,  A61K45/06 ,  A61K48/00 ,  C12N2501/15 ,  C12N2501/60 ,  C12N2501/65 ,  C12N2501/727 ,  C12N2501/999 ,  C12N2506/1307

Abstract: Pro-fibrotic signaling potently antagonizes cardiac reprogramming. Inhibition of pro-fibrotic signaling using small molecules that target the transforming growth factor-β/SMAD, or Rho kinase leads to conversion of approximately 60% of fibroblasts into beating cardiomyocytes. Conversely, over-activation of these pro-fibrotic signaling networks inhibits cardiac reprogramming. Using the disclosed methods, fibroblasts are converted to spontaneously contracting cardiomyocytes in less than two weeks.

公开(公告)号：US20200299727A1

公开(公告)日：2020-09-24

申请号：US16827454

申请日：2020-03-23

Applicant: THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE

Inventor： Kunhua Song ,  Congwu Chi ,  Yingqiong Cao

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

Abstract: The disclosed technology includes methods of treating Danon disease, for example correcting genetic mutations in the LAMP-2 gene and ameliorating at least one Danon disease phenotype, for example defective LAMP-2B-mediated autophagy. In some implementations, the disclosed methods include editing a mutated form of the LAMP-2 gene in a patient in need thereof. In some implementations, editing the mutated form of the LAMP-2 gene may include use of a CRISPR editing technique targeted to the mutated form of the LAMP-2 gene. As a result, mutated LAMP-2 proteins in mammalian subjects may be restored in at least some of the affected cells, for example cardiomyocytes.

公开(公告)号：US10519423B1

公开(公告)日：2019-12-31

申请号：US15853095

申请日：2017-12-22

Applicant: The Regents of the University of Colorado, a body corporate

Inventor： Kunhua Song ,  Yuanbiao Zhao ,  Pilar Londono ,  Timothy McKinsey

IPC: A61K48/00 ,  A61K38/17 ,  C12N5/077 ,  A61K45/06 ,  A61K31/713

Abstract: Pro-fibrotic signaling potently antagonizes cardiac reprogramming. Inhibition of pro-fibrotic signaling using small molecules that target the transforming growth factor-β/SMAD, or Rho kinase leads to conversion of approximately 60% of fibroblasts into beating cardiomyocytes. Conversely, over-activation of these pro-fibrotic signaling networks inhibits cardiac reprogramming. Using the disclosed methods, fibroblasts are converted to spontaneously contracting cardiomyocytes in less than two weeks.