CRISPR interference can achieve selective knockdown of any of the 3 CALM genes, representing a generalizable therapeutic strategy to potentially attenuate LQTS-triggered cardiac events.
Does CRISPR interference rescue function in calmodulinopathic Long-QT Syndrome?
CRISPR interference offers a generalizable precision medicine strategy to selectively knockdown CALM genes and potentially treat calmodulinopathic Long-QT Syndrome.
RATIONALE: channels. OBJECTIVE: To gain insight into the mechanistic underpinnings of calmodulinopathies and devise new therapeutic strategies for the treatment of this form of LQTS. METHODS AND RESULTS: /CaM-dependent inactivation after treatment. Moreover, CRISPR interference can be designed to achieve selective knockdown of any of the 3 CALM genes, making it a generalizable therapeutic strategy for any calmodulinopathy. CONCLUSIONS: Overall, this therapeutic strategy holds great promise for calmodulinopathy patients as it represents a generalizable intervention capable of specifically altering CaM expression and potentially attenuating LQTS-triggered cardiac events, thus initiating a path toward precision medicine.
Limpitikul et al. (Fri,) conducted a other in Malignant Calmodulinopathic Long-QT Syndrome. CRISPR interference was evaluated on Selective knockdown of CALM genes and CaM-dependent inactivation. CRISPR interference can achieve selective knockdown of any of the 3 CALM genes, representing a generalizable therapeutic strategy to potentially attenuate LQTS-triggered cardiac events.