Tech Comm Available Technologies :: The Research Institute at Nationwide Children's Hospital, Columbus, Ohio

Details

Project TitleCrispRCas9 Therapeutic Strategies for Titin Based Cardiomyopathies
Track Code2015-074
Short Description

Researchers at Nationwide Children's Hospital have developed a method for correcting mutations in titin by CRISPR/Cas9 genome editing. This method can be delivered directly via intracoronary delivery. 

Abstract

Titin is a large protein that is important for muscle elasticity and mutations in titin result in muscle impairment, including cardiomyopathies. Researchers at Nationwide Children's Hospital have developed a method for correcting mutations in titin. Using the CRISPR/Cas9 system of genome editing, they are able to modify the titin gene and resulting protein. This method can be delivered directly via intracoronary delivery to correct titin mutations and rescue protein function.

 
TagsCRISPR/Cas9, titin, rAAV.rh74, therapeutic, titin-based cardiomyopathy, titinopathies
 
Posted DateSep 25, 2017 2:25 PM

Researcher

Name
Louise Rodino-Klapac
Rachael Potter

Manager

Name
Margaret Barkett

Inquiry

Intellectual Property

Patent Number Issue Date Type Country of Filing
62/255,887 None Provisional United States
Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000