In vivo gene editing of CAMKIID: out with the bad and in with the good

John E. Smith, Henk Granzier

Research output: Contribution to journalComment/debatepeer-review


The ability to change an organism’s DNA through gene editing is of great importance for the prevention and treatment of genetic and acquired diseases. Rapid progress has been made during the last decade due to the discovery and refinement of CRISPR/Cas9 as an accurate, fast, and reliable genome editing technique. In this issue of the JCI, Lebek et al. present the culmination from a line of work in the Olson laboratory focused on in vivo gene editing of CAMK2D. The paper presents a combined state-of-the-art gene therapy approach that demonstrates how gene therapy can yield cardioprotection in a mouse model and takes notable steps toward potential applicability in patients.

Original languageEnglish (US)
Article numbere176672
JournalJournal of Clinical Investigation
Issue number1
StatePublished - Jan 20 2024

ASJC Scopus subject areas

  • General Medicine


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