A tiny biotech just suffered a safety scare in a gene-editing trial, raising 3 big questions for the entire CRISPR field
The first patient treated with Graphite Bio's gene-editing program suffered a serious side effect.
But in the months that have since passed, the number of red blood cells and platelets in the patient's body haven't recovered to normal levels. A key question for the gene-editing field will be understanding what part of Graphite's treatment is culpable. In a nutshell, Graphite's therapy has two components: usingto knock out a typo in the genetic code, and using a modified virus called AAV6 to deliver a correct version of that gene into the cells.
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