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articles and announcements

Categories: Antisense oligonucleotides

February 9, 2023

Contribution of hemizygous HERC2 deletion to Angelman syndrome pathophysiology

$200,000 2 years Individuals with a deletion of chromosome 15q11-q13 suffer from Angelman syndrome (AS), a neurogenetic developmental disorder characterized by intellectual disability, motor ataxia, absent speech, and seizures. The specific gene that is responsible for AS encodes the ubiquitin […]

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Dr. Ype Elgersma
February 3, 2023

Individuals with ICD/UPD Mutations Participating in ASO Trials

$99,000 2 years Angelman syndrome (AS) is caused by loss of maternal UBE3A expression. In neurons, paternal UBE3A is silenced by the UBE3A-ATS transcript, resulting in a total loss of UBE3A in AS neurons. Promising preclinical data by the Beaudet […]

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Dr. Ype Elgersma
October 10, 2022

ASO Treatment for a Better Understanding of AS Pathophysiology and Optimizing Therapeutic Efficacy

$199,100 Researchers will investigate in more detail the requirements of ASO-mediated rescue of behavioral phenotypes of an AS mouse model. The most important questions they will attempt to address are: How much ASO and how much UBE3A protein is really […]

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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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