Scientists report first use of CRISPR activation to treat a cardiac disease in mice

An international multidisciplinary team has demonstrated for the first time that CRISPR-based gene activation (CRISPRa) can be used to treat genetic heart disease in vivo. The study, published in the European Heart Journal and presented at the European Society of Cardiology Congress in Madrid, paves the way for novel targeted therapies for patients with genetic cardiac disorders. This approach could be especially useful for patients with conditions caused by mutations in genes too large to be targeted with conventional gene therapy.

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