December 21, 2019
$200,000 (2 years) Oligonucleotides are short, single-stranded DNA or RNA molecules. Previous studies have demonstrated that the antisense RNA of Ube3a (Ube3a-ATS) silences the paternal Ube3a gene. The investigators propose using antisense oligonucleotides (ASOs) to specifically target Ube3a-ATS, which is […]
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December 19, 2019
Ben Philpot, Ph.D. – University of North Carolina, Chapel Hill Ype Elgersma, Ph.D. – Erasmus Medical Center, Rotterdam, Netherlands $400,000 (2 years – $200,000 per institute) A previously developed mouse model for Angelman syndrome (AS) enables the maternal copy of […]
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December 14, 2019
Ben Philpot, Ph.D. – University of North Carolina-Chapel Hill Heather Hazlett, Ph.D. – Carolina Institute of Developmental Disabilities at University of North Carolina-Chapel Hill Ron Thibert, M.D. – Massachusetts General Hospital $295,970 (2 years) This unique research project is the […]
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December 14, 2019
$200,000 (2 years) At approximately two weeks of age in a proven AS mouse model, there is an increase in the expression and subsequent activity of a specific molecule prior to other molecules in the AS brain, which results in […]
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December 14, 2019
$200,000 (2 years) This research study builds upon previous ASF-funded research conducted by Dr. Art Beaudet at Baylor College of Medicine and will determine whether a drug that increases UBE3A expression can restore normal function to human AS brain cells. […]
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December 14, 2019
$181,800 Many families of individuals with Angelman syndrome express concern to their doctors about anxiety in their loved one. However, very little is understood about anxiety in individuals with AS at the clinical level. Many clinicians feel that once physical […]
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December 14, 2019
Dr. Geeske Van Woerden and her lab will research the best time to correct certain symptoms of AS when Ube3a is restored in a mouse model. Overall, the research team attempts to answer the question: can certain behaviors or symptoms […]
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December 14, 2019
$200,000 Based on previous research conducted at Dr. Mark Zylka’s lab and Dr. Ben Philpot’s lab, Dr. Zylka’s team will continue this work with the goal of unsilencing the paternal copy of Ube3a using new methods. Past research has shown […]
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