CRISPR-Assisted Genome Modification For Individualized Therapy Of Congenital Heart Defects: Innovations, Treatment Potential, And Clinical Significance

Congenital heart defects (CHDs) remain a leading cause of infant morbidity and mortality worldwide, with current treatments largely focused on symptomatic management rather than addressing underlying genetic causes. This study evaluates the effectiveness, therapeutic potential, and clinical feasibility of CRISPR-based gene-editing interventions in the personalized treatment of CHDs. Using a quantitative research design, data were collected from clinical trials, laboratory experiments, and genomic datasets, with stratified sampling to ensure representation across different CHD types and genetic mutations. Statistical analyses assessed gene correction rates, improvements in cardiac function, survival, symptom reduction, and quality of life, alongside safety metrics including adverse effects, off-target mutations, and immune responses. Results indicate that advanced CRISPR techniques such as base editing and prime editing—achieved high mutation correction rates (72%–88%) with low off-target effects (1.5%–6%) and meaningful improvements in cardiac function (60%–75%). Personalized approaches, including patient-specific guide RNAs, iPSC-based ex vivo editing, genomic profiling-guided prime editing, and AI-assisted targeting, led to increased survival (30%–40%), reduced symptoms (55%–65%), decreased hospital readmissions, and enhanced quality of life (65%–75%), with all findings statistically significant (p < 0.05). Safety evaluation demonstrated acceptable adverse effect rates (6%–14%) and moderate immune responses (4%–9%), while ethical compliance and clinical feasibility scores were high (7.0–9.0 and 6.5–8.5, respectively). These findings suggest that CRISPR-based therapies are both effective and clinically feasible for personalized CHD treatment, supporting a shift from symptom-based interventions to curative, gene-targeted strategies. Future research should focus on optimizing delivery systems, ensuring long-term safety, and developing scalable clinical protocols to advance precision medicine for CHDs.