Barb Bailus, PhD
Our next new scientific advisory board (SAB) member could be considered the grandfather of Angelman syndrome (AS), Dr. Art Beaudet. Dr. Beaudet received his MD from Yale and did his pediatric residency at Johns Hopkins before joining Baylor College of Medicine, where he has resided since 1971.
Dr.Beaudet first became interested in AS after the discovery of uniparental disomy in 1988, when it became clear that AS and PWS were most likely associated with genetic imprinting. His interest in imprinting disorders resulted in his lab identifying UBE3A as the causative gene for Angelman syndrome in 1997. He was elected to the National Academy of Science in 2011. He has always believed that Angelman syndrome is an excellent candidate for gene therapy interventions, and is currently very excited about all of the genetic engineering approaches entering clinical trials, including anti-sense oligonucleotides and CRISPR. His recent work identified that paternal UBE3A could be reactivated if the UBE3A-AS transcript was silenced, pointing to a promising therapeutic approach. He has mentored dozens of graduate students, many of whom have gone on to become leaders in their respective fields. One of his former students, Dr. Scott Dindot, the Chief Scientific Officer of GeneTx Biotherapeutics, LLC, is currently working on an extremely promising approach of silencing the UBE3A-ATS, thus activating the paternal UBE3A gene.
Many of us have met Dr. Beaudet at one of the FAST Galas that he has attended, which are among his favorite memories in his research career. When not in the clinic or mentoring the next generation of scientists, Dr. Beaudet enjoys sailing and playing the stock market. His newest endeavor includes founding Libra Genetics, a company hoping to revolutionize prenatal diagnoses, which could drastically change the way genetic diseases are both diagnosed and treated.