Targeting Angelman Syndrome Therapeutics to the Brain Utilizing Novel Cell Penetrating Peptides
Targeting Angelman Syndrome Therapeutics to the Brain Utilizing Novel Cell Penetrating Peptides
In the last fifteen years much progress has been made in both understanding the basic genetics of AS and developing tools for treating AS. However, one area that still needs further exploration is finding the best delivery method for all of the therapies being developed. The brain is an extremely challenging organ to target for treatments. Multiple promising technologies have been successful in animal models of AS, but achieving widespread delivery of these therapeutics in a human brain remains challenging and could be a limiting factor in the effectiveness of these therapeutic approaches. The main goal of this grant is to increase delivery options for AS therapeutics by developing novel cell penetrating peptides (CPPs) from viruses that target neuronal cells. These cell penetrating peptides will act as “access codes” through the blood brain barrier allowing for delivery of potential AS therapeutics. Development of neuronal CPPs could bring the AS field closer to a mode of delivery that is minimally invasive and can potentially be repeated. This has the potential to benefit all therapeutic modalities in AS and have a broad impact in the entire neurodevelopmental field.
Principle Investigator
Barbara Bailus, PhD
Dr. Barbara Bailus is an Assistant Professor of Genetics at The Keck Graduate Institute, a Member of The Claremont Colleges. She did her doctoral research at University of California, Davis in the Segal Laboratory developing a gene therapy based on artificial transcription factors for the treatment of Angelman syndrome. Her postdoctoral research was in the Ellerby Lab at The Buck Institute for Research on Aging, focused on developing therapies for neurodegenerative disorders. In 2020 Dr. Bailus established her own laboratory at The Keck Graduate Institute which has focused on therapeutic delivery mechanisms for neurological disorders including Angelman syndrome, SETBP1 disorder, Huntington’s disease, and Alzheimer’s disease. In 2021 Barbara was honored to take on the role of Chair of the Scientific Advisory Board for the Foundation for Angelman Syndrome Therapeutics (FAST). Dr. Bailus has a passion for teaching the next generation of scientists and communicating exciting scientific advances to the public. When not in the lab or classroom she enjoys being at the beach, traveling, riding horses, painting and playing with her cat.
