World's First Gene Editing Treatment Will Try To Cure Hemophilia

Posted: Dec 4 2015, 4:25am CST | by , Updated: Dec 4 2015, 9:53pm CST, in Latest Science News


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World's First Gene Editing Treatment will Try to Cure Hemophilia
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  • Gene Editing Method could Cure Hemophilia

A novel gene editing method could prove to be a cure for hemophilia.

Hemophilia B is a state of sickness that we would not wish upon our worst enemy. The problem in this disease is that the livers of those who have this genetic malady fail to produce Factor IX.

This factor is responsible for the clotting of blood. Thus the sufferers of this illness often die to to internal or external bleeding. It is indeed a tragedy of the highest order. Genetic engineering has however come to the rescue.

A novel form of gene therapy could possibly cure this ailment. Two genetic engineers, Michael Holmes and Thomas Wechsler from Richmond, California's Sangamo biopharmaceuticals, have announced at the International Summit on Human Gene Editing that the world's first in-human gene-editing therapy which pinpoints these disordered genes will begin next week.

It revolves around the CRISPR-9 gene-editing techniques. It is all about cutting out a part of the DNA strand and inserting something else in its place. Recent advancements along these lines include the saving of the life of a baby afflicted with leukemia.

But a majority of the scientists are wary of any such interventionist procedures. The NIH has even put a kibosh on the research funding for gene-editing.

The modification of the suffering person’s cells and the reintroduction of the material back into his or her body is what this procedure is all about. By snipping the Factor IX and shifting the genes towards a promoter, it is hoped a cure will be possible.

The promoter will increase the production of albumin via gene expression. The liver is especially responsible for producing albumin. Both in rats and apes, this procedure works with remarkable efficiency. However, how it will fare in man remains to be seen. After testing the scheme on adults, children and babies will be next on the list.

It comes as no surprise that many in the scientific community are not very happy about this state of affairs. They point out that this sort of interventionist response may lead to malformations and mutations in the DNA which in itself could have devastating consequences.

The FDA has however given its stamp of approval to the process. It is our only hope against such deadly diseases as hemophilia which have no cure.

If the experts are worried, it shows that they are actually expecting the results to be of the make or break variety. And since there is no change without conflict, this sort of experimentation ought to be endorsed despite some of its obviously frightening aspects.

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