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Regina Bailey

Gene Therapy for Mitochondrial Diseases

By October 31, 2012

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Oregon Health & Science University researchers have developed a method for preventing the spread of inherited diseases from mother to child. The method focuses on diseases caused by gene mutations in cell mitochondria. The process involves removing the nucleus from the mother's egg cell or sex cell, and inserting it into a donor egg that has had its nucleus removed. The egg cell is then fertilized and implanted into the mother where it develops normally.

According to researcher Shoukhrat Mitalipov, "When certain mutations in mitochondrial DNA are present, a child can be born with severe conditions, including diabetes, deafness, eye disorders, gastrointestinal disorders, heart disease, dementia and several other neurological diseases. Because mitochondrial-based genetic diseases are passed from one generation to the next, the risk of disease is often quite clear. The goal of this research is to develop a therapy to prevent transmission of these disease-causing gene mutations." The researchers state that the procedure outlined in this study was first used in primates and resulted in the birth of a healthy monkey.

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