Angelman syndrome: Bellwether for genetic therapy in autism
It is not a matter of whether there will be clinical trials of genetic therapy for Angelman syndrome, but when.
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Spectrum: Autism Research News
Benjamin Philpot
October 2018
July 2011
Insights for autism from Angelman syndrome
Deletions or duplications of the UBE3A gene lead to both Angelman syndrome and some cases of autism, respectively. Studying the effects of altered gene dosage in this region will provide insights into brain defects and suggest targets for therapies for both disorders, says expert Benjamin Philpot.
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