The treatment, called Casgevy, is developed by Vertex Pharmaceuticals and CRISPR Therapeutics.
It affects approximately 100,000 people in the United States, the majority of whom are Black.
The first signs of the disease usually show up within the first year of life.
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This is a cure.
How Does the Treatment Work?
The patient must then undergo chemotherapy before the edited stem cells can be inserted back into the patient.
CRISPR proteins and their guides are not perfect, Glassberg said.
Sometimes the guides can match up with DNA in unintended locations, causing unwanted cuts.
Most of the time, these off-target hits are harmless, but very rarely they can be dangerous.
This could be a cure that would not cause infertility or death, and could be accessed worldwide.
What This Means For You
There’s now an FDA-approved gene therapy for sickle cell disease.
An FDA panel said the treatment is safe enough for clinical use.
However, this treatment might come with a hefty price tag.
Centers for Disease Control and Prevention.Complications of sickle cell disease.
National Library of Medicine: MedlinePlus.What are genome editing and CRISPR-Cas9?.
