Angelman Syndrome is a rare genetic disorder caused by UBE3A gene mutations, leading to developmental delays, speech issues, seizures, and distinct behaviors like frequent laughter and hyperactivity.

Dr. Roger Hollis from the University of California, Los Angeles, project scientist from the laboratory of Dr. Donald Kohn, a world leader in developing gene replacement therapies using blood stem ...

This is one of 22 cutting-edge projects selected to share in a total of $8.1 million in grants raised through the Eagles ...

Dr. Eric Levine’s cutting-edge autism research at UConn School of Medicine is reprogramming the donated skin or blood cells ...

Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the drug.

California Institute for Regenerative Medicine (CIRM) has awarded a $5.8 million translational research grant to a scientist at the University of California, Los Angeles (UCLA) to further support the ...

The data to date, funded by TransformaTx Biotherapeutics, a biotechnology company founded by the Foundation for Angelman Syndrome Therapeutics (FAST), has demonstrated complete symptomatic ...

to treat Angelman syndrome (AS). "This program has consistently delivered remarkable animal data, reinforcing its potential as a groundbreaking treatment," said Dr. Donald Kohn, Distinguished ...

Prader-Willi syndrome (PWS) and Angelman syndrome (AS) are imprinting disorders caused by genetic or epigenetic aberrations of 15q11.2-q13. Their clinical testing is often multitiered; diagnostic ...

Dr. Eric Levine’s cutting-edge autism research at UConn is reprogramming the donated skin or blood cells of Angelman or Dup15q syndrome patients to develop them in the lab into brain cells that ...

Background Deregulation of the activity of the ubiquitin ligase E6AP (UBE3A) is well recognised to contribute to the development of Angelman syndrome (AS). The ubiquitin ligase HERC2, encoded by the ...