martes, 30 de julio de 2019

CRISPR is (gradually) moving from lab to bedside

The Readout
Damian Garde

CRISPR is (gradually) moving from lab to bedside


Yesterday, CRISPR Therapeutics revealed that it had treated its first patient with a genome-editing approach to sickle cell disease. And in beta thalassemia, a rare blood disease, the first patient treated with the same CRISPR’d cells has been free of blood transfusions for more than four months.

Each is an incremental milestone but a milestone no less. And the news comes a week after Editas Medicine, another CRISPR company, opened enrollment in a study that will test a genome editing therapy for an inherited form of blindness. Intellia Therapeutics, the third CRISPR biotech, hopes to start a first trial next year.

That’s all to say that, after years of theoretical debate and legalistic dispute, we’re finally going to get actual data on how CRISPR might help actual human beings.

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