Science & Nature
Promising gene therapy, gene editing and antisense oligonucleotide approaches herald a new era of cardiac medicine for patients with certain so-far intractable heart conditions.
In addition, a trio of agenda-setting papers from the labs of Eric Olson at the University of Texas Southwestern Medical Center and a complementary study from Christine Seidman’s lab at Harvard Medical School have shown that CRISPR–Cas9 editing, base editing and prime editing can all be harnessed to correct genetic models of cardiac disease in mice. Technical proof of concept has now been established for tackling cardiomyopathies caused by mutations in the MYH7 and the RBM20 genes, as well as for disrupting a pathological signaling mechanism caused by chronic overactivation of calcium/calmodulin-dependent protein kinase IIδ that is present in many patients with heart failure.
This is a preview of subscription content, access via your institution
Access options
Access Nature and 54 other Nature Portfolio journals
Get Nature+, our best-value online-access subscription
$29.99 per month
cancel any time
Subscribe to this journal
Receive 12 print issues and online access
$209.00 per year
only $17.42 per issue
Rent or buy this article
Get just this article for as long as you need it
$39.95
Prices may be subject to local taxes which are calculated during checkout
Rights and permissions
About this article
Cite this article
Sheridan, C. Genetic medicines aim straight for the heart.
Nat Biotechnol (2023). https://doi.org/10.1038/s41587-023-01745-4
Published:
DOI: https://doi.org/10.1038/s41587-023-01745-4
Read More
Cormac Sheridan