New Research Raises Hopes For Treatment of Mitochondrial Genetic Disease

In a number of past research projects, scientists have tried to treat mitochondrial disease, a life-threatening genetic disorder that affected thousands of children in the U.S every year.

But now, a team of researchers at Oregon Health and Science University in Portland, Oregon, has used a unique cloning and cell reprogramming technique to create a fresh piece of tissue or stem cells that perfectly match and is compatible with the skin of the affected patient.

Shoukhrat Mitalipov, the lead study author, says that in the near future, the research team will replace the diseased tissue with the healthy tissue developed with the help of a technique that used somatic-cell nuclear transfer. During the study, the researchers used the technique to develop patient-specific stem cells that could potentially be used to treat a variety of genetic diseases.

Mitochondria are a type of cell organelle, also known as the “powerhouse” of the cell. The tiny structures convert fats and carbohydrates into energy. In case of a genetic mutation, the normal functioning of the mitochondria is disrupted and it results in a variety of problems, including diabetes, muscular dystrophy, deafness, dementia and heart disease.

Many researchers in the past used the traditional gene therapy technique to treat mitochondrial disorders. However, they recorded no successes. In February 2015, the U.S. government allowed IVF clinics to use the DNA from another “donor” mother to treat the defective eggs of a mother who was at risk of passing on the genetic mitochondrial disease to her child.

Based on the latest study findings, the researchers claim that now there is no need for a three-parent IVF to treat the mitochondrial genetic disorders.