How does CRISPR inhibition work?
CRISPR technology provides an avenue for effectively targeting and manipulating specific regions of the human genome, and the potential to permanently cure the root cause of a disease. RENOGENYX is developing a CRISPR-based approach to treat the underlying problem that gives rise to FSHD.
CRISPR technology consists of two components working together: 1) the Cas9 enzyme, which cuts DNA and 2) a guide RNA, which directs Cas9 to a specific site in the genome for targeted editing. Rather than using Cas9 to cut and edit DNA, we use a non-cutting version of Cas9 to target repressor proteins to the DUX4 gene and return the disease locus to a healthy state of repression.
This approach, called CRISPR inhibition, is potentially much safer, more effective, and ideally suited to treat FSHD.
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