- First established BMD cardiomyopathy model in middle-sized animals
- Highly extrapolative approximation to humans
- Anesthetic injection causes high rate of cardiac dysfunction (cyanosis, arrhythmia)
- Possible for drug validation and screening by serum myocardial injury marker, electro cardiogram, body color in this model
Background & Technology
Although BMD is said to have milder symptoms than DMD, BMD sometimes expresses cardiac dysfunction earlier than muscle weakness. Because of no gait abnormalities, it is difficult to recognize BMD and in the meantime the heart failure symptoms progress and increases risk of sudden death with exercise or anesthetic injection. A therapeutic drug for DMD was commercialized in 2020. By this medicine, it is assumed that the more cases with the DMD symptoms will be milder and similar to the BMD symptoms. Until now, as the drug for BMD has not been developed, only symptomatic treatment such as cardiotonic agent, steroid have been available, the need for therapeutic drug has been increasing.
Inventors have discovered the carrier families with genetic mutations showing BMD phenotype in the care animals in Azabu University and have established the families as a model. As the mutations are abnormality of X chromosome, mother swine with no symptoms and the half number of male piglets show the symptoms. Inventors have been obtaining frozen eggs from mother swine and frozen sperm from male piglets and establishing BMD model breeding and reproduction method.
As a BMD drug exploring/screening method via this model (male piglet), we can handle or provide the model showing cardiac dysfunction by anesthetic injection, evaluate potential drugs by quantitatively measurement and analysis of serum myocardial injury marker, electro cardiogram, body color via this model.
Figure 1 ; Pathological features of skeletal muscle(Left) and cardiac muscle(Right) of male piglet that died of heart failure by anesthetic stress. Skeletal muscle showed a denature(arrow) and regeneration (arrow head). Cardiac muscle was highly atrophic and wavy.
Figure 2 ; Immunofluorescence in cardiac muscle. Wild type male piglet (Left), mother swine (Center), died male piglet (Right). In mother swine and died male piglet heart, dystrophin expression decreased compared to wild type heart.
We are looking for partner companies that are interested in drug potential validation & screening for BMD treatment or research & development (Assessment of activity on cardiac function) from your library. Please let us know your interest for collaboration research in muscular dystrophy field.
Product No. ON-03701b