Targeted gene integration in skeletal muscle – Muscle represents a critical tissue for gene replacement. Over 70 neuromuscular diseases are cause by genetic mutations in muscle. Designing a versatile gene therapy to address these diseases requires new approaches. We are investigating hijacking of endogenous genes for sustained gene therapy. Moreover, this approach could be used to turn muscle into a protein “factory”.
Relevant work:
Biorxiv link
News (Harumi)
Delivery of genome editing technologies – As therapeutics, genome editing technologies hold great promise. The challenge is safe and efficient delivery to diseased tissues in the patient. We are adapting viral and non-viral delivery vehicles and assessing safety and efficiency in preclinical models of disease.
Relevant work:
Review article: Nelson & Gersbach. Engineering Delivery Vehicles for Genome Editing. ARCBE. 2016
Gene regulation in regenerative medicine and wound healing – Changes to gene expression in chronic disease delaying or preventing tissue regeneration or restoration. Targeted control of gene expression can illuminate new drug targets (Review, 2014).
Relevant work:
