Year 2016 begins with a flying start in the scientific field. Already on the first day, three promising studies that show how severe the disease can be treated with new technology.
On the science side, we have written lots of new genetic tool crispr, partly developed in Umeå, and first presented to the world just three years ago.
We have speculated that the rise People Emmanuelle Charpentier, Jennifer Doudna and Feng Zhang would get the Nobel Prize this year. (At that point we were wrong, but they will certainly be considered for the Nobel Prize within a year or two.)
Just a week ago we reported that the technology crispr has been named the greatest scientific breakthrough of 2015 .
And we have talked about an international research meeting, initiated by the US Academy of Sciences, who discussed ethical dilemmas associated with the new technology.
Ethics meeting in the US culminated in a document, which described the new technology amazing opportunities. The document encouraged the world’s scientists to go ahead and use crispr to cure human diseases. However, with an important reservation. In the current situation it is not appropriate, posted the document’s signatories, to tinker with eggs, sperm and human embryos and then putting the fertilized embryos into a woman’s uterus, to thereby produce transgenic human child.
The reservation applies even if the intent is the best and the goal is to treat severe genetic diseases.
One of the genetic diseases that immediately comes to mind is Duchenne muscular dystrophy. It is a very difficult disease, which means that the muscles slowly atrophy – both skeletal muscles in the legs and arms, and heart. The first symptoms come before the age of six, and although the possibility of processing has improved die sufferers usually young.
They are almost always boys, because the disease is due to a gene on the X chromosome has been broken. (Girls have two X chromosomes, and it is enough that one of them has the gene intact.)
A healthy woman can carry the gene and pass it on to his son. Sometimes incurred a mutation, and a child affected by the disease, but it has been in the family before.
Scientists have known for thirty years which gene causes the disease. But knowing which gene is defective are one thing, to repair it is another.
So far, the genetic tools have been too blunt and uncertain. Crispr means totally new possibilities. Researchers have already demonstrated in vitro on cell cultures that they now can basically fix the gene that causes Duchenne muscular dystrophy.
But it is as I said unethical to treat the genes carrying the woman’s egg or a fertilized embryo, if the results to be a living child. Remains to try to treat the child once it is born, and the disease is a fact.
And it could well move to make, now shows three studies in Science magazine by publishing date January 1, 2016.
The three American research groups have shown that they can deal with both the newborn mouse pups and adult mice with the equivalent of Duchenne muscular dystrophy. Although scientists can not cook all the muscle cells in the mice’s bodies. But it is also not necessary. The body can function normally if only a few percent of the muscle cells has a properly functioning gene.
Note that so far is the research on mice. The step from mice to humans can be long, and it is important not to patients currently suffering from Duchenne muscular dystrophy and their families make for great hopes for himself.
But the treatment seems to work on mice are still a big step forward. A year that begins so bright on the very first day, we very much hope.
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