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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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Such information is provided for informational purposes only and is not meant to be a substitute for advice provided by a doctor or other qualified health care professional. Patients should not use the information contained herein for diagnosing a health problem or disease. Patients should always consult with a doctor or other health care professional for medical advice or information about diagnosis and treatment.

CRISPRi Gene Therapy for FSHD, or any other therapeutic program referenced on this website, is not an approved therapeutic product. The information on this website is for illustrative purposes only.

Any suggested treatment protocol, diagnostic process, or any other information related to any medical product is entirely proposed only. No suggestion is made as to the efficacy or safety of any therapeutic product in development. Any approved therapeutic product and/or treatment regimen for any therapy being developed by RENOGENYX may be materially different in scope, nature, and practice than any suggested.

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