Heart failure, which affects over 64 million people worldwide, is currently considered irreversible without a heart transplant. Researchers at the University of Utah have developed a gene therapy that could change this. Using minipigs as a model, scientists successfully reversed heart failure by delivering a therapeutic gene called cBIN1 directly into heart cells. The therapy, which targets a key protein often deficient in heart failure patients, resulted in significant recovery, improving heart function by 30%. This marks a substantial leap compared to current treatments' 5–10% improvement.
The treatment relies on an adeno-associated virus (AAV9) to transport the cBIN1 gene into damaged heart cells. This one-time injection restored heart function and improved the heart's structure and pumping ability. In the study, all treated pigs survived the six-month trial, while untreated animals typically succumbed within months. Dr. Robin Shaw, the lead researcher, described the results as unprecedented, highlighting the therapy’s ability to help the heart heal itself.
The next step for researchers is to adapt the therapy for human use, with clinical trials expected to begin by 2025. Key challenges, such as ensuring safety and addressing natural immunity to the viral carrier, remain, but the team is optimistic.
If successful in humans, this therapy could revolutionize how heart failure is treated, offering millions of patients the possibility of recovery. By repairing damaged heart tissue, it could provide relief to those who currently face limited options.
Created: Dec 11th, 2024
Citations:
GEN. (2024). Gene therapy reverses effects of heart failure and restores heart function in minipigs. Genetic Engineering & Biotechnology News. Retrieved from https://www.genengnews.com/topics/translational-medicine/gene-therapy-reverses-effects-of-heart-failure-and-restores-heart-function-in-minipigs/
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