FSHD (facioscapulohumeral muscular dystrophy)
FSHD (facioscapulohumeral muscular dystrophy) affects more than 1 million people of all races and ethnicities. It affects both men and women equally, and a severe early onset form affects children. With clinical weakness typically appearing in the second or third decade of life and ~20% of patients ultimately becoming wheelchair-bound, the personal, social, and economic costs of this disease are enormous. There are no cures or ameliorative treatments for FSHD, so an effective therapy is critically needed.
FSHD is caused by mutations that result in a loss of repression at the disease locus and misexpression of the DUX4 gene in skeletal muscles. Over time, this leads to progressive weakness and muscle atrophy. The most fundamental path to therapy is silencing expression of DUX4 in FSHD patients by returning the disease locus to a normal state of repression.
This will require a mechanism for long-term silencing of DUX4 and efficient therapeutic delivery to skeletal muscles throughout the body.
RENOGENYX has overcome this challenge by developing a highly specific FSHD therapeutic cassette which fits into a viral vector enabling AAV-mediated therapy.
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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.
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