🔍 Summary:
Scientists have discovered a groundbreaking molecule that could potentially treat POLG-related diseases, which are rare genetic disorders affecting mitochondrial DNA. These conditions, affecting approximately 1 in 10,000 people globally, are caused by mutations in the POLG gene responsible for DNA replication and repair in mitochondria. The mutations lead to energy-starved cells due to the accumulation of harmful mutations or deletions in mitochondrial DNA.
The study, published in the journal Nature, introduces a molecule named PZL-A, which has shown promise in laboratory experiments. This molecule can enhance the stability and function of the POLG protein, even in its mutated forms, thereby improving the ability of cells to replicate and repair mitochondrial DNA. The research initially screened 270,000 compounds, leading to the development of PZL-A, which was tested on the four most common POLG mutations, covering about 70% of the cases.
The findings are significant as they offer a potential treatment pathway that does not rely on gene editing, which is challenging due to the numerous mutations involved. The molecule is now being tested in clinical trials by Pretzel Therapeutics to assess its safety and efficacy in humans.
This discovery not only opens potential treatment options for POLG-related diseases but may also have broader implications for other conditions linked to mitochondrial DNA depletion, such as aging-related diseases and neurodegenerative disorders.
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