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CrispRCas9 Therapeutic Strategies for Titin Based Cardiomyopathies

詳細技術說明: 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 largeprotein that is important for muscle elasticity and mutations in titin resultin muscle impairment, including cardiomyopathies. Researchers at NationwideChildren's Hospital have developed a method for correcting mutations in titin.Using the CRISPR/Cas9 system of genome editing, they are able to modify thetitin gene and resulting protein. This method can be delivered directly viaintracoronary delivery to correct titin mutations and rescue protein function.

*Inquiry: Margaret Barkett, PhD The Research Institute at Nationwide Children's Hospital Office of Technology Commercialization 700 Children's Drive Columbus OH 43205 T: 614 355-2957 F: 614 722-2716 Margaret.barkett@nationwidechildrens.org

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