再生医学ジャーナル

Gene Therapy for Mitochondrial Diseases: A Promising Approach to Restoring Mitochondrial Function

Philip Kwin*

Mitochondrial diseases are caused by mutations in genes involved in the production, maintenance, or function of mitochondria, leading to a wide range of symptoms. While there are currently no cures for mitochondrial diseases, gene therapy offers a promising approach to introduce healthy copies of genes into affected cells. The article discusses different gene therapy approaches, including viral vectors and mitochondrial replacement therapy, as well as the challenges that must be addressed. The development of safe and effective delivery systems, ethical considerations, and ongoing research offer hope for new and effective treatments for mitochondrial diseases using gene therapy.