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Powerhouses for the New Frontier
A fundamental fact of biology is that mitochondria are the powerhouses for the cell. Mitochondria are small, kidney bean shaped organelles that convert our glucoseinto energy for our cells to use. Some people may not know that some of your genetic information doesn’ t come from the DNA in the nucleus; your mitochondria also contain small bits of genetic informationas well, completely separate from the nucleus. The mitochondrial DNA don’t really affect traits such as eye color and dimples. Rather, mitochondrial DNA plays roles in maintaining energy and protein production within the human body.
While your mitochondria only have 37 genes (a fraction of the 21,000 total genes in a human being), that doesn‘t mean that those genes aren’t important. Mutationsof the mitochondrial DNA can lead to devastating effects such as degradation in the central nervous system (Leigh syndrome),muscles (mitochondrial encephalomyopathy),and possible formation of cancers.
However, with the advent of a new form of gene therapy, there may be possible cures for mitochondrial DNA (“mtDNA”) in store for us.
A technique that has been dubbed as the “three parent baby” therapy has reached the point where it may soon becomeavailable on a broad scale. The therapyinvolves replacing faulty mitochondria in the mother’ s egg with healthy versions from a donor, leading to a child who has no faulty mitochondria and thus no faulty mtDNA. The child with the rectified mtDNAhas three genetic parents: the mother, father, and the egg donor who contributed her mitochondria, hence the name “three parent baby.”This technique is currently pending for approval for testing with expectant mothersin Britain in early 2017, and if allowed, could start this potentially life-saving therapy as early as April 2017.
Only the egg’s mitochondria would have to be replaced as a child‘s mtDNA is always inherited from the mother. The mitochondriain the father’s sperm die after making it to the egg. The possible benefits with this technique are obvious: women with mitochondrial defects would be able to have children without fear of their child having any lifelong damaging disabilities. However, this technique is still relatively experimental, and as such, trial in the UK has been very limited. Because this techniquedeals with mtDNA, whatever genetic material is replaced there will be passed from mother to child even throughout future generations. There will be, and of course should, more testing of such promisingnew cures in the UK. However, with mitochondrial mutations making up 1 in every 4,000 births in the U.S.A. alone, new techniques such as mtDNA gene therapy may pave the way to higher quality of life for all.