Crossing the finish line towards a disease-modifying treatment for Angelman syndrome.
Judson MC., de Almeida LP., Burdine RD., Chamberlain SJ., Deverman BE., Distel B., Ehlers MD., Jalazo E., Kushner SA., Nespeca M., Sanders SJ., Scheffner M., Yi JJ., Zylka MJ., Elgersma Y., Philpot BD.
Recent progress in the development of genetic therapies promises that impactful treatments for single-gene neurodevelopmental disorders are imminent. But can derailed neurodevelopmental processes be mended after broken genes are replaced or otherwise restored? The results of ongoing clinical trials for Angelman syndrome will soon yield answers to this pressing question, yet the trials face significant obstacles. Here we identify insights needed to aid the quest for a disease-modifying Angelman syndrome therapy, which could serve as a roadmap for the expeditious development of genetic therapies for other single-gene neurodevelopmental disorders.