This pioneering mouse model replicates the key characteristics of hypoplastic left heart syndrome by exhibiting an underdeveloped aorta and left ventricle, along with defective aortic and mitral valves. It demonstrates a unique cardiac arrangement where the right ventricle compensates for the impaired left, channeling blood through a patent ductus arteriosus with an accompanying atrial septal defect that permits mixing of oxygenated and deoxygenated blood. The methodology integrates traditional forward genetics with CRISPR-Cas9 validation, ensuring the model confidently mimics the human condition through rigorous in vivo testing.
Description
What sets this technology apart is its exclusive focus on hypoplastic left heart syndrome—the first of its kind—unlike previous models for other cardiovascular defects. Its methodical combination of classic genetic screening and modern genome editing not only achieves precise replication of congenital heart anomalies but also offers reproducibility and specificity absent in related systems. Extensive patent searches reinforce its novelty, confirming that no similar approach has been previously documented, which positions this model as an invaluable asset for advancing research and therapeutic innovation.
Applications
- HLHS therapeutic drug screening
- Preclinical cardiac device testing
- Congenital heart defect research
- Genetic therapy validation
Advantages
- Introduces the first animal model specifically tailored to study hypoplastic left heart syndrome (HLHS).
- Accurately replicates key cardiac malformations found in HLHS for high translational relevance.
- Utilizes a combination of traditional forward genetics with CRISPR-Cas9 for precise genetic editing and verification.
- Provides a reproducible, in vivo validated platform to study disease mechanisms and develop potential therapies.
- Fills a critical research gap by offering a dedicated model for a previously unmodeled severe congenital heart defect.
IP Status
Research Tool
