CHICAGO – Scientists at Stanford University School of Medicine have used the CRISPR gene editing tool to repair the gene that causes sickle cell disease in stem cells from diseased patients, paving the way for a potential cure for the disease, which affects up to 5 million people globally.
“What we’ve finally shown is that we can do it. It’s not just on the chalkboard,”
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said Dr. Matthew Porteus, senior author of the study published in the journal Nature.
With the study, and unpublished findings from his lab, Porteus believes his team has amassed enough proof to start planning the first human clinical trial using the powerful CRISPR-Cas9 gene editing system to correct the genetic mutation that causes sickle cell disease.
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