Tag Archives: Angelman syndrome

Babies born with a faulty maternal copy of the UBE3A gene will develop Angelman syndrome, a severe neurodevelopmental disorder with no cure and limited treatments. Now, for the first time...

from UT San Diego by Bradley J. Fikes Isis Pharma approach helps mouse models of genetic disease A potential therapy for Angelman syndrome, a baffling genetic disease that impairs intellectual development, has been proposed by scientists including researchers at Isis Pharmaceuticals. The treatment, tested in mice, activates a gene that restores some of the neurological activity reduced in Angelman syndrome, ...