Invention:
This innovation is based on the finding that lack of Leiomodin-2 (Lmod2) is associated with cardiovascular issues, specifically enlarged heart or cardiomyopathy and it is better to have a truncated/mutated Lmod2 than zero or little Lmod2 present. A premature stop codon associated with Lmod2 causes truncation of Lmod2. This innovation is the use of antisense for blocking the system that recognizes and prevents the premature stop codon, thereby allowing the truncated/mutant protein synthesis.
Background:
Cardiomyopathy is a disease of the heart muscle that makes it harder for the heart to pump blood to the rest of the body. Cardiomyopathy can lead to heart failure. The main types of cardiomyopathies include dilated, hypertrophic, and restrictive cardiomyopathy. Treatment — which might include medications, surgically implanted devices, heart surgery or, in severe cases, a heart transplant — depends on the type of cardiomyopathy and how serious it is. There might be no signs or symptoms in the early stages of cardiomyopathy. But as the condition advances, signs and symptoms usually appear and tend to get worse unless treated. Leiomodin-2 (Lmod2) is a regulator of the actin thin filament lengths within the heart muscle. Knockout of Lmod2 has been shown to result in dilated cardiomyopathy. The inventors here show preliminary results that suggest that expressing either truncated/mutated Lmod2 results in improved cardiac function compared to no Lmod2 expression at all. They have designed an antisense oligonucleotide that would enable the increased expression of truncated/mutated Lmod2 in cardiomyopathy patients.
Applications:
- Heart failure treatment
- Heart failure prevention
Advantages:
- Reliable
- Proven success
- Healthier life
