Dr. Allisandra Rha is a molecular biologist at the Children’s Hospital of Orange County, dedicated to advancing
research and therapeutic development for lysosomal storage diseases. Dr. Rha received her PhD from Emory
University, where she defined pivotal interactions underlying the neuropathology of protein misfolding disorders.
As a postdoctoral fellow at Auburn University, Dr. Rha joined one of the research teams responsible for the
clinical translation of gene therapies for GM1 and GM2 gangliosidosis. In her current role, Dr. Rha has
spearheaded projects focused on variant-specific editing in GM1 gangliosidosis and Pompe disease. Her present
work builds upon recent studies evaluating the efficacy of base editing as a therapeutic candidate for GM1
gangliosidosis, extending strategies to sophisticated cell culture models of neurodegeneration including cerebral
organoids. Through her work, she intends to deliver on the hope and promises of therapeutics for patients with
rare lysosomal storage diseases.
Dr. Allisandra Rha
