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Viral Gene Therapy Researchers have used a viral vector to deliver a working copy of the ALPK3 gene to heart cells in adult mice with a inherited heart muscle disease. The study, published in Nature Cardiovascular Research on August 19, found that the treatment restored heart function and structure to levels similar to those of healthy animals. The heart contracts due to sarcomeres, repeating protein structures in muscle cells, and ALPK3 helps maintain these proteins in working condition. Without two working copies of ALPK3, the heart becomes enlarged and pumps blood less efficiently. In humans, disease-causing variants of this gene lead to inherited cardiomyopathies, with ALPK3 variants accounting for around 2% of cases of hypertrophic cardiomyopathy. The researchers used an AAV viral vector to package a 5,800-base pair construct containing ALPK3, and found that it restored order to the contraction apparatus in diseased hearts. They also tested the therapy in human heart organoids with variants of the TTN gene, which codes for the massive protein titin that forms the sarcomere framework. The results showed that delivering ALPK3 restored contraction strength to levels similar to those of organoids without the variant. 🔗 Read original →