Funding Research for Treatments and a Cure

A rigorous test in the mouse of whether increased DNA methylation can activate neuronal expression of the paternal Ube3a allele
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Dr. Arthur Beaudet, MD Baylor College of Medicine, Houston, Texas
2007

A rigorous test in the mouse of whether increased DNA methylation can activate neuronal expression of the paternal Ube3a allele

$80,000

The mouse model provides an ideal way to evaluate therapeutic attempts to increase UBE3A function on the otherwise silenced paternally-derived mouse chromosome. Using novel genetic engineering methods, Dr. Beaudet and his lab were able to develop a engineered mouse with genetic tags on both the paternally and maternally expressed genes, thus enabling a way of detecting more precisely whether drugs or agents are capable of improving activity of the otherwise silenced UBE3A gene. This is part of a number of studies aimed at developing novel treatment strategies for AS.