Patricia Inacio, PhD,  science writer—

Deleting the Ube3a gene alters the social behavior of mice in a way that may mimic that of humans with Angelman syndrome, a study suggests. The research, “Hypersociability in the Angelman syndrome mouse model,” appeared in the journal Experimental Neurology. Humans inherit two copies of the Ube3a gene,…

Researchers have developed a method to study the enzymes involved in the disposal of proteins that are faulty or no longer needed by cells. The research will help scientists investigate the mechanism of diseases where the disposal system is functioning badly, such as it does in Angelman syndrome, which may…

University of California researchers have developed a touchscreen test for mice with Angelman syndrome that they believe will shed additional light on learning disabilities stemming from the disease. It is one of a batteries of tests they are giving the animals to obtain a greater understanding of Angelman’s effect on…

The U.S. Food and Drug Administration (FDA) awarded a fast track designation to Ovid Therapeutics’ OV101 for the treatment of Angelman syndrome. The designation allows for expedited development and review of therapeutics for serious diseases with high unmet medical need. “This designation is an important milestone for both Ovid…

Ovid Therapeutics presented data from several studies that support the clinical development of the investigational therapy TAK-935/OV935 as a potential treatment for developmental and epileptic encephalopathies. These results are particularly significant for Angelman syndrome patients, 85 percent of whom suffer from some form of epilepsy. The findings from…

Agilis Biotherapeutics, which develops innovative DNA therapeutics for rare diseases of the central nervous system, presented the latest results of its gene therapy program for Angelman syndrome at two recent international conferences. During the annual meeting of the European Society of Gene and Cell Therapy (ESGCT), Dr. Kevin…

Throughout evolution, genomic recycling of protein-coding genes gave rise to part of non-coding genes within our genome, known as long non-coding RNAs, or lncRNAs, a Weizmann Institute of Science study suggests. The findings will help scientists understand the role of lncRNAs in healthy and diseased conditions, such as Angelman…

A new, revised protocol of the non-invasive prenatal test (NIPT) Panorama identifies genetic abnormalities underlying Angelman and four other syndromes with higher accuracy and efficiency than the original test, a study found. The study, “Clinical Experience with a Single-Nucleotide Polymorphism-Based Noninvasive Prenatal Test for Five Clinically Significant…