Antisense oligonucleotides (AO) are an emerging class of new genetic medicines that can be rationally designed to modulate the expression of any gene. The Ocular Tissue Engineering Laboratory is developing AO-based pharmaceuticals targeting a variety of inherited retinal disease-causing gene mutations present in the WARD study cohort. Our retinal disease modeling pipeline provides material for testing these AO pharmaceuticals on patient-derived retinal tissues in the laboratory, greatly facilitating the identification of potentially therapeutic candidates. Our group is currently testing potential treatments for Stargardt disease, Usher syndrome and retinitis pigmentosa.
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