UC Davis Leads Breakthrough Research: Gene Therapy for Angelman Syndrome & Bio-Pacemaker for Heart Rhythm
A UC Davis professor, David Segal, PhD, is spearheading a groundbreaking research project, backed by a $1.36 million grant, to develop a gene therapy for Angelman syndrome. This disorder affects around 2,500 children and families in California and many more worldwide. The therapy aims to restore normal function to deficient nerve cells.
The research project, led by Segal, focuses on Angelman syndrome, a genetic disorder caused by a deficiency in the maternal copy of the UBE3A gene. The therapy's goal is to increase UBE3A enzyme levels in affected nerve cells. It targets and destroys the UBE3A antisense transcript, which silences the paternal gene copy. The therapy will use an adeno-associated virus (AAV) to deliver an RNA nuclease (Cas13) to the brain.
In parallel, another UC Davis researcher, Deborah Lieu, PhD, received a second DISC2 grant to develop a bio-pacemaker to restore heart rhythm. Her team aims to create this bio-pacemaker by injecting cardiomyocytes derived from induced pluripotent stem cells (iPSCs) into the heart.
Both projects, funded by the California Institute for Regenerative Medicine (CIRM) under the Discovery Stage Research Projects (DISC2) program, hold promise for improving the lives of those affected by these conditions. The gene therapy for Angelman syndrome could provide a much-needed treatment option, while the bio-pacemaker could revolutionize heart rhythm management.
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