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1. (WO2017151717) COMPOSITIONS AND METHODS FOR ADENO-ASSOCIATED VIRUS MEDIATED GENE EXPRESSION IN MYOFIBROBLAST-LIKE CELLS

Pub. No.:    WO/2017/151717    International Application No.:    PCT/US2017/020113
Publication Date: Sat Sep 09 01:59:59 CEST 2017 International Filing Date: Thu Mar 02 00:59:59 CET 2017
IPC: A61K 35/34
A61K 48/00
A61P 9/10
C12N 15/63
Applicants: FRENCH, Brent A.
Inventors: FRENCH, Brent A.
Title: COMPOSITIONS AND METHODS FOR ADENO-ASSOCIATED VIRUS MEDIATED GENE EXPRESSION IN MYOFIBROBLAST-LIKE CELLS
Abstract:
The present application discloses a recombinant AAV vector comprising a promoter that targets gene expression to myofibroblast-like lineage of cells in the heart. The present application, also discloses the preparation and use of AAV expression cassettes using a modified periostin promoter that successfully drives gene expression in cardiac myofibroblast-like cells. The present invention encompasses compositions and methods useful for treating myocardial infarction. Further comprising compositions and methods for preparing and using AAV vectors for targeting cells and inducing gene expression. The compositions and methods of the invention are useful for efficiently targeting cardiac myofibroblasts following a cardiac injury, disease, or disorder. Further comprising a kit for effecting alleviation of the various diseases or disorders recited herein.