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New Genetic Therapy

Pete Springer/OPB

Roughly one out of every 4,000 children in the United States develops a mitochondrial disease before the age of 10. These genetic diseases are inherited mutations, and cause symptoms as wide-ranging as loss of muscle coordination, vision and hearing problems, diabetes, learning disabilities, and dementia. There is no known cure for mitochondrial diseases.

Now, researchers at Oregon Health and Science University have developed a new kind of genetic therapy that’s meant to keep these diseases from getting passed down from mother to child.  The procedure uses techniques drawn from cloning, and a paper on the study, published in Nature, made headlines last week. Working with rhesus macaque monkeys, scientists successfully substituted healthy donor mitochondria in place of a female’s defective genes. This allowed her to pass on her DNA to her baby, without passing on the defective mitochondria.

The research brings up many ethical and legal questions: ethicists are asking if people should be able to make genetic alternations for generations to come. They point out that this modification eliminates one type of genetic disease, but worry it opens the door to the possibility of tinkering with DNA to select for genetic advantages.

Do you have a mitochondrial disease, or do you know someone who does? If it were available, would you consider this procedure when planning to have a child? Where would you draw the line when choosing the traits you want your children to have?


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