Gene therapy reduced Heart Failure mortality in minipigs
Minipigs presumably not experiencing heart failure shown above.
Image credit: PickPik
TLDR
A research effort by Jing Li et al have developed a Gene therapy that repaired hearts in minipigs that were afflicted with non-ischemic heart failure.
Paper is HERE
More:
Researchers have developed a promising gene therapy that targets a protein called cardiac bridging integrator 1 (cBIN1) to treat heart failure. In a study involving pigs with heart failure, a single intravenous injection of this therapy restored heart function and significantly improved survival rates. The therapy works by increasing cBIN1 levels in heart cells, which helps repair damaged tissue and enhances the heart's ability to pump blood. This approach offers a potential new treatment for heart failure, a condition affecting millions worldwide.
TERMS:
Ischemic heart disease
Also known as coronary heart disease, this condition occurs when the heart's arteries narrow, reducing blood flow and oxygen to the heart muscle. Symptoms include chest pain or discomfort, known as angina pectoris
Team:
Jing Li, Pia Balmaceda, Thuy Ha, Joseph R. Visker, Nicole Maalouf, Eugene Kwan, Guillaume L. Hoareau, Michel Accad, Ravi Ranjan, Craig H. Selzman, Stavros G. Drakos, Robin M. Shaw & TingTing Hong
Abstract
Heart failure (HF) is a major cause of mortality and morbidity worldwide, yet with limited therapeutic options. Cardiac bridging integrator 1 (cBIN1), a cardiomyocyte transverse-tubule (t-tubule) scaffolding protein which organizes the calcium handling machinery, is transcriptionally reduced in HF and can be recovered for functional rescue in mice. Here we report that in human patients with HF with reduced ejection fraction (HFrEF), left ventricular cBIN1 levels linearly correlate with organ-level ventricular remodeling such as diastolic diameter. Using a minipig model of right ventricular tachypacing-induced non-ischemic dilated cardiomyopathy and chronic HFrEF, we identified that a single intravenous low dose (6 × 1011 vg/kg) of adeno associated virus 9 (AAV9)-packaged cBIN1 improves ventricular remodeling and performance, reduces pulmonary and systemic fluid retention, and increases survival in HFrEF minipigs. In cardiomyocytes, AAV9-cBIN1 restores t-tubule organization and ultrastructure in failing cardiomyocytes. In conclusion, AAV9-based cBIN1 gene therapy rescues non-ischemic HFrEF with reduced mortality in minipigs.
Paper is HERE
Conclusion:
Any tool or technology has the potential to be used for both beneficial and harmful purposes. This particular innovation might offer hope as a powerful solution to address the widespread health challenges faced by billions due to complications arising from the COVID mRNA vaccine products.