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Lab-Grown Miniature Hearts Revolutionize Childhood Heart Disease Research

Scientists have made a significant breakthrough in understanding childhood heart diseases by utilizing lab-grown miniature hearts, known as cardiac organoids. These tiny hearts, developed by researchers at Australia’s QIMR Berghofer’s Cardiac Bioengineering Lab, closely mimic the structure and function of real adult human heart muscle. Grown from human pluripotent stem cells, these organoids address a longstanding scientific challenge in studying heart conditions.

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One major obstacle in using lab-grown heart cells has been their tendency to remain in an immature state, akin to fetal cells. To overcome this limitation, scientists activated specific biological pathways that simulate the effects of exercise on these cells. By pushing the cells to mature and function more like adult heart tissue, this innovative approach bridges the gap between lab-grown and real heart tissue, enhancing the accuracy of disease modeling and drug testing.

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As a result of this breakthrough, researchers now have a more precise model to test drugs and explore genetic heart conditions, particularly those affecting children. Professor James Hudson from QIMR Berghofer emphasizes the benefits of studying heart diseases using human cardiac organoids, highlighting the accelerated drug development process enabled by screening numerous compounds efficiently in the lab.

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Moreover, these cardiac organoids have successfully replicated key features of heart diseases associated with gene mutations, such as ryanodine, calsequestrin, and desmoplakin. Notably, the challenging condition of desmoplakin cardiomyopathy, known for its complexity in study, was effectively recreated in the lab. The organoids exhibited scarring and diminished pumping ability, mirroring patient experiences with heart disease.

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Following treatment with a novel drug class called a ‘bromodomain and extra-terminal protein inhibitor,’ the lab-grown tissues displayed significant improvement, offering hope for potential therapies. The collaborative efforts of QIMR Berghofer, the Murdoch Children’s Research Institute (MCRI), and The Royal Children’s Hospital, supported by genetic and protein analysis, are propelling advancements in precision treatments for childhood heart conditions.

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Associate Professor Richard Mills from MCRI stresses the importance of accurately modeling childhood heart diseases to enhance treatment outcomes for affected individuals. The unified approach adopted by the research consortium is streamlining progress towards improved therapies, with the potential to impact a wide spectrum of childhood heart conditions. The study, featured in the journal Nature Cardiovascular Research, signifies a significant stride in decoding childhood heart diseases and developing tailored treatment solutions.

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