Additional Gene Therapy Approach For Angelman Syndrome Explored

By Paula Evans

stem-cells_IRC-signage350The Foundation for Angelman Syndrome Therapeutics (FAST) is delighted to announce that research FAST has funded, through the support of our donors, has secured a $1.1 Million grant from the California Institute for Regenerative Medicine, to develop an additional gene therapy approach for the treatment of Angelman syndrome.

Dr. David Segal, a member of the FAST FIRE Initiative (FAST Integrative Research Environment), whose work focuses on activating the paternal UBE3A gene through deactivation of the UBE3A-ATS (antisense transcript), will now be able to investigate the use of mesenchymal stem cells to activate the paternal UBE3A gene.  “This award is completely based on work that has been generously funded by the Foundation for Angelman Syndrome Therapeutics over the past few years,» stated Dr. Segal, adding, «In addition to working together with the entire FAST Integrative Research Environment (FIRE) team to advance our protein-based approach towards clinical trials, the team has also pushed to investigate alternative delivery methods to ensure the greatest likelihood for success of a treatment. I am so delighted that the California Institute for Regenerative Medicine (CIRM) has allowed us to leverage FAST’s investment in one of these alternatives, using adult stem cells to deliver the protein to the brain, so that this promising approach can also be accelerated.»

«This is such a remarkable and exciting time for Angelman Syndrome research,» stated Dr. Allyson Berent, Chief Science Officer of FAST.  «We are exploring several different therapeutic approaches to cure Angelman Syndrome and it is extraordinary to see pharmaceutical and biotechnology companies, as well as other funding agencies, share in our confidence and commitment.»

Dr. David Segal, always acutely aware of exactly who it is he is working for, added, «I think this is truly something for which we all have a reason to celebrate, from the FAST leadership who make the FIRE team able to do what they do, to all the contributors and families that make FAST able to do what they do. This is a very exciting time for the  Angelman community and I am just glad to be part of it.»

To read more about Dr. Segal’s new project, click here.