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Will Curing a Rare Disease Help Bring Genome Surgery Into Everyday Practice?

Genome surgery uses the powerful, and still controversial, CRISPR/Cas9 gene-editing tool to remove specific genes from a person’s DNA. UCSF researchers, in collaboration with UC Berkeley and the Gladstone Institute, are piloting a genome surgery trial focused on successfully curing Best disease in a volunteer by harvesting blood cells from the patient, coaxing them into stem cells, and then directing them towards becoming retinal cells. After this conversion, the CRISPR systems are used to “edit out” the BEST1 gene which is the cause of the disease and, subsequently, to replace affected cells in the patient.

The team responsible for this research hopes that by curing a disease with a well-understood genetic causation, they may open the door to wider acceptance of the ability to modify our genomes for better health.

Click here to read more about this effort at the UCSF News Center.