Dr. Yong-Hui Jiang, MD, PhD Baylor College of Medicine, Houston, Texas
2007
Explore the therapeutic potential of levodopa to treat Angelman syndrome in mouse model
$50,000
There is a small amount of anecdotal experience and some neuroscience studies suggesting that levodopa might have therapeutic effect on the symptoms of AS. The Angelman mouse model is an ideal way to study this before developing any human clinical trials and that was the reason for ASF funding this project. The investigator was able to use Angelman mice to investigate effects of levodopa supplementation and in some areas no effect was demonstrated (such as on a type of synaptic activity called long-term potentiation) but in other areas, such as assessment of mouse EEG patterns, an effect was noted. The results did not reveal dramatic effects from levodopa supplementation but did identify areas worthy of further study.