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The eukaryotic genome can be cut at any desired position by introducing plasmids containing Cas genes and specifically constructed CRISPRs into eukaryotic cells. CRISPR nucleases serve as an important genome editing tool. However, not all innovations are equal and nor do they follow a constant upward trend.
EDIT-301 is an experimental, ex vivogeneediting cell medicine in development for the treatment of sickle cell disease. Previously, the Company received Rare Pediatric Disease designation from the FDA for EDIT-301. Enrolled patients will receive a single administration of EDIT-301.
Assuming these findings are replicated in clinical trials, this vaccine, with its more-appealing route of administration and convenient (refrigerator) storage requirements could make an important contribution to containing the disease.
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