A group of scientists have succeeded in editing out mitochondrial DNA in mice. The technique may just be the key to stopping the spread of incurable diseases.
Cutting out Diseases
Researchers from Salk Institute developed molecular scissors to cut out mutations in an embryo – without damaging the healthy DNA of the organism. In the journal Cell, the researchers discussed the specifics of the study.
Two strains of mitochondrial DNA were tested in the study. Using the engineered molecular scissors, the scientists identified and snipped out the disease causing DNA in mouse embryos. The end results were healthy offspring. The study also had success in the use of the technique on defective human DNA inserted into mouse eggs. The scientists want to run laboratory tests on discarded human embryos to further the research.
If the scientists prove the safety and efficacy of the procedure, the procedure could be an alternative to the UK government-approved mitochondrial transfer therapy. The researchers hope that in the future, the technique will be of use in preventing mitochondrial disease in humans.
The mitochondria are present in almost every cell of the body. The main function of these cells is to generate the right amount of energy to perform specific functions.
The said organelles contain their own DNA – something that children inherit from their mothers. This different kind of DNA does not affect the appearance of a person and other characteristics, but if defective, life-limiting conditions may arise. Blindness and muscle weakness are two examples of the possible effects of defective mitochondria.
Experts have called the method a “technical masterpiece.”
But although it is advanced, the process is not without challenges. The process still raises a number of scientific and ethical concerns. Industry experts continue to have mixed opinions on mitochondrial transfer therapy and this new technique.
Guy’s and St. Thomas’ Hospital genetics expert Prof. Frances Flinter told the BBC, “The biggest question to address will be the possibility that DNA cutting enzymes may disrupt adjacent genes that are important, leading to unintended adverse consequences.”
There are still serious risks to consider when replacing faulty genes in embryos, say some scientists. Others argue that human genetic engineering is downright unethical.
Just recently, Chinese researchers drew flak over the manipulation of human embryo genes. The world’s first study met a lot of criticism from different organizations and scientists. The US International Society for Stem Cell Research called for a moratorium on the project and said that it is important to discuss the “societal and ethical implications” of such a procedure.