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A method of allele-specific targeting which allows for direct treatment of underlying disease mechanisms in patients with inherited cardiomyopathies.
About
Summary
Stanford researchers have developed a method of allele-specific targeting which allows for direct treatment of underlying disease mechanisms in patients with inherited cardiomyopathies. Reduction in expression of mutated alleles in a site-specific manner promotes normal allele expression ratio and will facilitate normalization of protein and myocyte function.
Applications
Treatment of myocardial disease, including dilated cardiomyopathy, hypertrophic cardiomyopathy, Arrhythmogenic right ventricular cardiomyopathy, left ventricular noncompaction cardiomyopathy.
Advantages
There are no currently available treatments that directly target gene expression in human cardiomyopathy. Allele-specific constructs can target the mutated allele for silencing while sparing the normal allele. Use of the modified AAV9 vector allows for cardiac-directed gene targeting at physiologically tractable titers with reduced systemic reaction and minimal risk of systemic infection.