sicklecelldisease
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Stanford develops CRISPR-based therapy for sickle cell disease
A team of Stanford scientists are making great progress in their search for a cure for sickle cell disease, and they want to start human trials as soon as 2018. They used CRISPR to fix the mutated gene that causes the illness in human stem cells taken from actual patients. The CRISPR technique gave them a way to carve out the faulty part of the gene and replace it with the normal DNA sequence that was supposed to be there in the first place. That prevents previously affected red blood cells from transforming into tiny sickles that have the tendency to clog blood vessels and cause organ damage.
