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Targeted delivery of gene therapy via exosomes for cardiomyocyte reprogramming

Targeted delivery of gene therapy via exosomes for cardiomyocyte reprogramming

Unmet Need

Every year, close to 1 million Americans experience a heart attack. A heart attack results in the loss of specialized cells called cardiomyocytes, which are crucial for the heart’s contraction and rhythmic beating. Cardiomyocyte depletion increases the risk of heart failure. Consequently, researchers have prioritized ways to replenish the cardiomyocyte population. Despite significant efforts, no method for replenishing the cardiomyocyte population has reached the clinic. Thus, there is a clear need for interventions that can boost the population of cardiomyocyte cells in patients following cardiac arrest.

Technology

Duke inventors have developed a novel method to convert fibroblasts within the heart attack scar into cardiomyocytes. They uniquely achieve this via a combination of RNA molecules called miR combo. The critical issue is one of delivery. Up until this point, researchers and commercial entities have focused solely on viruses as the delivery vehicle. Viruses are problematic because they cannot hold much material and they generally lack cell specificity. This lack of specificity is particularly concerning in clinical settings, as it increases the risk of harmful off-target effects. Motivated by this concern, the Duke inventors developed a unique delivery approach involving exosomes. Specifically, they identified that exosomes derived from C166 cells targeted scar fibroblasts to the exclusion of all other cell-types. When compared to viral delivery approaches, C166-derived exosome delivery of miR combo resulted in significant improvements in reprogramming efficacy as well as functional recovery in a mouse myocardial infarction injury model.

Other Applications

Fibroblasts can differentiate into many different cell types including adipocytes (fat cells), hepatocytes (liver cells), osteocytes (bone cells), endothelial cells, and epithelial cells. As such, this technology could be applied to many other diseases that are defined by fibrosis/tissue injury. Examples of such diseases include end stage liver disease, kidney disease, lung scarring from infections, as well as fibrosis from autoimmune diseases like rheumatoid arthritis, ulcerative colitis, lupus, and Crohn’s disease.

Advantages

  • C166-derived exosomes specifically target scar fibroblasts.
  • More effective than current strategies: viral approaches typically increase ejection fraction, a measure of cardiac function, by 5-10%. In contrast, the C166-derived exosome approach improves ejection fraction by 25%.
  • Unlike viral vectors, exosomes not limited by packaging size constraints.

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