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Background on Danon Disease Danon disease is a rare X-linked dominant genetic disorder with an incidence of fewer than 1 in 1 million individuals worldwide. It is caused by mutations in the LAMP2 gene, which plays a crucial role in lysosome function. Loss of LAMP2 gene function causes impaired cellular waste recycling – ultimately manifesting...

Published: 9/27/2023 | Inventor(s): Kunhua Song, Congwu Chi, Yingqiong Cao

Disease Areas Regenerative Medicine

Category(s): Therapeutics