CRISPR Therapeutics, based in Switzerland, said it used CRISPR to try to cure one patient of beta thalassemia, which leaves patients dependent on transfusions.
Patients who have beta thalassemia inherit defective copies of the gene that makes hemoglobin, which red blood cells use to transport oxygen. However, everyone’s body carries a second copy of the gene called fetal hemoglobin that is shut off after we’re born. In the clinical trial, scientists are using CRISPR editing to try and turn the fetal gene back on.
The treatment involves gathering bone marrow stem cells from a patient and fixing their DNA in the lab using CRISPR. The cells are then reinfused into the bloodstream. The one-time treatment could theoretically cure the disease, but researchers say it is too soon to know the outcome.
CRISPR Therapeutics said it will wait to see how the first patient responds before treating more patients. The company plans to treat patients with sickle-cell anemia using the same method.
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