Novel AAV systems
We leverage our background in structural biology, biophysics and computational biology to develop novel AAV systems. These systems address limitations of AAVs, such as packaging capacity, production cost and efficiency, transduction efficiency and tropsim, to enable safe and efficient gene therapies that are also accessible and cost-effective.
Protein homeostasis in photoreceptor disease
Proteopathies, where mutations in proteins prevent their proper folding, are one of the main causes behind many retinal degenerative diseases including autosomal dominant Retinitis Pigmentosa. In proteopathies, the increased amount of misfolded protein poses a tremendous burden on retinal cells, which must now allocate a large part of their proteostatic machinery to recognizing, attempting to fold, and degrading misfolded proteins. This severely limits the proteostatic capacity available to the rest of proteins for normal cell function and greatly reduces the cell’s ability to overcome additional stress and aging, resulting in premature cell death and leading to impaired vision and blindness.
Our research is centered on identifying and characterizing proteostatic mechanisms that can be therapeutically delivered to diseased photoreceptors to increase their proteostatic capacity, preserving cellular health and delaying disease progression.