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Hypertrophic cardiomyopathy (HCM) is a genetic disease of the heart muscle and the most common cause of sudden death in young people and athletes, caused by heterozygotic missense mutations in genes encoding proteins of the cardiac sarcomere. Researchers hypothesized that delivering oligonucleotide reagents with allele-specific silencing capabilities might abrogate the negative effects of the disease, and generated cell models to test the efficacy and specificity of various siRNAs and shRNAs. They identified siRNAs and shRNAs with moderate efficacy and high specificity for human N47K and R403Q mutations, and are using human transgenic mouse models and induced pluripotent cells to generate cardiomyocytes for experimental studies, aiming to develop novel therapeutics for cardiovascular diseases.

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Hypertrophic cardiomyopathy (HCM) is a genetic disease of the heart muscle and the most common cause of sudden death in young people and athletes, caused by...

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  • File Format: PDF
  • File Size: 263 KB
  • Pages: 1
  • Language: EN
  • Total Downloads: 84
  • Last Updated: 4 weeks ago

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