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articles and announcements

Categories: Antisense oligonucleotides

December 21, 2019

The role of antisense RNA Ube3a-ATS in Ube3a imprinting and Angelman syndrome

$100,000 Angelman syndrome is caused by deficiency of UBE3A. Unlike ordinary autosomal genes, it is subject to genomic imprinting with expression only from maternal chromosome. It is unknown how such imprinted status is established, since no differential DNA methylation was […]

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Dr. Arthur Beaudet
December 21, 2019

Ube3a-ATS Targeted Antisense Oligonucleotides As Therapies for Angelman Syndrome

$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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Stormy Chamberlain, PhD
December 14, 2019

Testing the efficacy of antisense oligonucleotides (ASOs) against UBE3A-ATS in human neurons

$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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