Long-term transcriptome-wide therapeutic effects of AAV-hpCas13d

To fully understand the enduring therapeutic effects of hpCas13d and its potential for clinical translation, comprehensive investigation is needed. In this study, we employed RNA-sequencing to analyze transcriptome alterations in Myh6RH/+ hearts, Myh6RH/RQ hearts, and hpCas13d[RQ]-treated Myh6RH/RQ hearts, aiming to elucidate the molecular mechanisms underlying the regression of cardiac hypertrophy induced by hpCas13d[RQ] treatment. To evaluate the transcriptome-wide therapeutic impact, hearts were harvested from Myh6RH/+, Myh6RH/RQ, and Myh6RH/RQ+AAV-hpCas13d mice 12 months after neonatal administration. Total RNA was extracted and subjected to RNA sequencing analysis.