Explore epigenetic therapy of using histone deacetylase inhibitors in the Angelman Syndrome mouse model

$98,450

ASF has supported on many fronts research strategies in an attempt to increase the expression of UBE3A in the AS brain. As discussed for previous grants, it is known that in each nerve cell a UBE3A gene is repressed or silenced and is located on the paternally derived chromosome 15. One way to increase expression of UBE3A is to modify the imprinting control center (located near UBE3A) by trying to induce changes in the protein structure that surrounds or is adjacent to UBE3A. At present, the findings demonstrate that this avenue of gene treatment, while showing some promise, remains problematic since significant expression of the otherwise silenced gene is difficult to obtain.