BioSpace5dOpinion
Opinion: Angelman Syndrome Race Heats Up Among Ionis, Ultragenyx and Neuren
Ionis and Ultragenyx are competing to develop oligonucleotide treatments for Angelman syndrome, but will Neuren’s peptide ...
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What Is Angelman Syndrome - A Rare Genetic Disorder Colin Farrell's Son Has Been Diagnosed With
According to experts, it is caused by a loss of function in the UBE3A gene - usually only active from the mother. When the maternal copy of the gene is missing or damaged, the brain is not able to ...
Morningstar2d
Quiver Bioscience Announces Collaboration with the Dup15q Alliance to Advance Antisense Oligonucleotide Therapeutic Program
Dup15q is associated with genomic copy number variations that result in increased levels of the UBE3A gene product. Dup15q syndrome is clinically related to Angelman syndrome (AS), a distinct ...
Nature4y
Genetic mechanisms leading to Angelman syndrome
Angelman syndrome is caused by a large deletion of the region of the maternal chromosome that contains UBE3A, or by a DNA sequence change (mutation) in the UBE3A gene inherited from the mother.
Live Science on MSN3mon
Angelman syndrome: A disorder that stops people walking and speaking
The disorder principally affects the central nervous system, meaning the brain and spinal cord, and it's caused by mutations ...