Key result
CRISPR/Cas9/AAV9-based somatic mutagenesis revealed that JPH2 protects T-tubules in the failing heart, while RYR2 is a novel T-tubule maturation factor.
Population
Postnatal murine cardiac myocytes (in vivo mouse model)
Comparison
CRISPR/Cas9/AAV9-based somatic mutagenesis… vs Nontransduced cardiac myocytes or varying doses…
Design
Preclinical
Authors
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Does not alter clinical practice; leaves open T-tubule-targeted therapies pending translational studies.
CASAAV provides a novel in vivo platform for rapid dissection of cardiac myocyte gene function, identifying RYR2 as a required T-tubule maturation factor.
Guo et al. (2017) studied Cardiac myocyte maturation. CRISPR/Cas9/AAV9-based somatic mutagenesis (CASAAV) vs. Nontransduced cardiac myocytes was evaluated on T-tubule maturation and cardiac function. CRISPR/Cas9/AAV9-based somatic mutagenesis revealed that JPH2 protects T-tubules in the failing heart, while RYR2 is a novel T-tubule maturation factor.
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