Systemic subcutaneous injection of hydrophilic bile acid (TUDCA) preserves cone photoreceptors in experimental model of LCA

A research team based at the Department of Ophthalmology & Visual Sciences, University of Utah Health Sciences Center, Salt Lake City, has reported that systemic injection of the bile acid TUDCA (tauroursodeoxycholic acid) may enhance the endoplasmic-reticulum-associated protein degradation pathway (ERAD) in a model of Leber congenital amaurosis (LCA). The capability to systemically deliver the endoplasmic reticulum (ER) associated chemical chaperone may present clinical opportunities to support current gene therapy and retinoid analog therapeutic strategies for the treatment of the severe inherited retinopathy. If transferable to the clinic, the authors of the research suggest the approach may lead to the development of a new class of therapeutic drugs for treating LCA.