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In vitro and in vivo models available for therapeutic discovery and development.
About
About this innovation FSHD is the third most common muscular dystrophy in man with an estimated incidence of 54 per million. Patients suffer from progressive and irreversible weakness and wasting of the facial, shoulder and upper arm muscles. Approximately 20% of gene carriers become wheelchair dependent. There is no cure for FSHD. Scientists in have discovered two novel target mechanisms whereby the two forms of FSHD can arise. The mechanisms represent targets for therapeutic intervention. In addition, cell lines and mouse models of FSHD have been developed and can be used to further research the disease and/or to screen and validate potential therapeutics. Partner companies are now sought for research collaborations in this field, and licensing of key technologies available at the institutions. Key benefits Novel and valid mechanism for FSHD etiology Novel mechanism for therapeutic intervention In vitro and in vivo models available for therapeutic discovery and development World-leading expertise Applications FSHD Research FSHD drug discovery and development FSHD gene therapy Epigenetic therapy