Will gene therapy be able to cure hemophilia?

Hemophilia is a rare disorder in which the blood does not clot normally. It is usually inherited. Hemophilia usually occurs in males. If you have hemophilia, you have little or no clotting factor. Clotting factor is a protein needed for normal blood clotting.

So what might be the answer?

Gene therapy.

“The dream of gene therapy is to fix your DNA so you’re not sick anymore—a “cure.” During 2016, Italian scientists at Milan’s San Raffaele Telethon Institute for Gene Therapy reported that they had cured 18 children of a rare but terrible immune deficiency disease, ADA-SCID. They removed the children’s bone marrow, added a gene to make the ADA enzyme their bodies lack, and replaced it. Technology Review explained how the treatment, now called Strimvelis and owned by Glaxo, took 14 years to develop and test. It was approved in Europe in May of this year.”

Using gene therapy, mice have recently been cured of hemophilia:

Hemophilia is a devastating genetic condition—without the ability to form blood clots, those who have it risk bleeding to death from even the slightest cut.

But scientists at the University of Pennsylvania have their sights set on a potential cure. Working in mice, they used the gene editing technique CRISPR to repair defective genes in a clotting protein called factor IX, the source of the mutations that cause hemophilia B. And it worked.

It appears that it could be just a question of (a short period of) time until hemophilia is easily curable.

See also: First real gene therapy “cure”?


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