Crossing the finish line towards a disease-modifying treatment for Angelman syndrome

Abstract 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 clinica...

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Hlavní autoři:	Matthew C. Judson, Luis Pereira de Almeida, Rebecca D. Burdine, Stormy J. Chamberlain, Benjamin E. Deverman, Ben Distel, Michael D. Ehlers, Elizabeth Jalazo, Steven A. Kushner, Mark Nespeca, Stephan J. Sanders, Martin Scheffner, Jason J. Yi, Mark J. Zylka, Ype Elgersma, Benjamin D. Philpot
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	BMC 2026-03-01
Edice:	Journal of Neurodevelopmental Disorders
On-line přístup:	https://doi.org/10.1186/s11689-026-09681-5
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