Gene Therapy Mouse Models

Gene therapy models include knockout models (loss-of-function diseases), point mutation knockin models (specific disease variants), conditional knockout models (tissue specific disease), and humanized models (testing human gene replacement). Selection depends on disease mechanism and therapeutic strategy (gene replacement, correction, or augmentation).

Gene therapy efficacy is validated through functional rescue (behavioral, biochemical, or physiological endpoints), protein expression (Western blot, immunohistochemistry), vector biodistribution (PCR, imaging), safety assessment (toxicity, immune response), and long-term persistence (expression duration, stability).

Yes. Reporter knockins can track gene expression, vector transduction efficiency, and therapeutic transgene expression. GFP or luciferase reporters enable real-time monitoring of gene therapy delivery and expression through imaging or flow cytometry. This is particularly valuable for AAV-based therapies. (/request-quote)