Spotlight on VOC Researcher - Dr. Alejandra Young
Dr. Alejandra Young’s passion for the cure is evident the moment one walks into her lab. With a Ph.D. in Molecular Biology, Dr. Young is an extremely dedicated researcher who has made finding the solution to ocular albinism (OA) a big part of her career. Her studies will contribute to a better understanding of the pathology of the disease and open new possibilities for OA therapy.
Dr. Young is a scientist with the Jules Stein Eye Institute at UCLA and has been a Vision of Children researcher since 2008. Most notably, she was selected as our Fellow and served in that capacity from 2011 – 2013. Her current research strives to combine stem cell therapy and gene therapy to treat ocular albinism.
Gene therapy is a powerful tool that has been tested for the prevention of blindness in a variety of ophthalmic disorders. Conventional methods for transferring functional genes have included the use of viruses, synthetic liposomes, and nanoparticles. However, these methods of gene transfer not only use infectious agents but are often inefficient, requiring multiple applications. Recently, microvesicles (MVs) released by stem cells have been identified as possible delivery vehicles for gene transfer. MVs are used by cells under normal conditions as part of a cell-to-cell communication system, since they can transfer their contents (such as nucleic acids and proteins) to other cells.
Dr. Young is investigating whether OA1 protein-containing MVs derived from engineered stem cells can transfer their contents to the abnormal retinal pigment epithelium of mice that serve as a mouse model for human ocular albinism. Successful repair of the retinal defects found in these mice might lead to new treatments for OA patients in the future.