公开(公告)号：US20210380650A1

公开(公告)日：2021-12-09

申请号：US17392137

申请日：2021-08-02

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

Inventor： Eric N. OLSON ,  Rhonda S. BASSEL-DUBY ,  Catherine A. MAKAREWICH ,  Benjamin R. NELSON

IPC: C07K14/47 ,  A61K38/17 ,  A61K45/06 ,  A61K48/00

Abstract: The present disclosure describes a new native peptide designated herein as Dwarf Open Reading Frame, or DWORF. This peptide enhances the apparent activity of the SERCA pump, is positively inotropic and lusitropic, and therefore is provided as a therapeutic agent for disorders characterized by cytosolic calcium overload.

公开(公告)号：US20230241249A1

公开(公告)日：2023-08-03

申请号：US18004522

申请日：2021-07-06

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

Inventor： Eric N. OLSON ,  Rhonda S. BASSEL-DUBY ,  Benjamin R. NELSON ,  Catherine A. MAKAREWICH

IPC: A61K48/00 ,  A61P9/04

CPC classification number: A61K48/0058 ,  A61K48/0016 ,  A61P9/04

Abstract: Disclosed are methods of treating a subject, such as those having or at risk of cardiomyopathies, with an effective amount of a recombinant adeno-associated virus (rAAV) virion, the rAAV virion comprising an AAV capsid and an expression cassette comprising a polynucleotide encoding a DWORF polypeptide operatively linked to a promoter. Compositions and kits relating to the same are also disclosed.