Barb Bailus, PhD
It may seem odd that FAST has added an HIV expert to the SAB, but Dr. Joseph Anderson also specializes in stem cells and unique delivery methods for biological therapeutics. Dr. Anderson received his PhD from Colorado State University, Fort Collins. Dr. Anderson currently has a laboratory at University of California, Davis Medical Center in Internal Medicine. His previous training in preclinical studies and regulatory procedures allows for his team to move benchside therapies into human clinical trials. Dr. Anderson became interested in AS when one of his students was discussing his research on AS. Dr. Anderson realized that the approach he was using for Tay-Sachs might also work for AS and put together a FAST TRAC grant with Dr. David Segal and Dr. Jill Silverman. This FAST funded project involves developing a method of delivering the UBE3A protein to the brain via hematopoietic stem cells, (stem cells in the bone marrow) which would be genetically modified to secrete this UBE3A protein, allowing for UBE3A to enter the neurons in the brain where they are so desperately needed. Dr. Anderson is very excited about the lab’s current mouse preliminary results, in which several behavioral phenotypes show significant improvements. If this strategy is successful, it is possible that AS patients would receive one injection of UBE3A producing hematopoietic stem cells that could last a lifetime.
Being very new to the AS community, his favorite memory was meeting all of the families and hearing about the amazing progress toward a cure at the 2018 FAST Gala. When not doing amazing research, Dr. Anderson enjoys hiking in the great outdoors, playing with his dogs and looking forward to civilian space travel.
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