rAAV-CRISPRa therapy corrects Rai1 haploinsufficiency and rescues selective disease features in Smith-Magenis syndrome mice.Chang H.C., Lee Y.J., Javed S., Haque M., Chang Y.T., Lin Y.C., Oram C., Huang W.H.View abstractCategoriesFunction, ExpressionSourceMGI: 103291PubMedEurope PMCJ Biol Chem 299:102728-102728 (2023)Mapped to5
Temporal dissection of Rai1 function reveals brain-derived neurotrophic factor as a potential therapeutic target for Smith-Magenis syndrome.Javed S., Lee Y.J., Xu J., Huang W.H.View abstractCategoriesFunction, Expression, Phenotypes & VariantsSourceMGI: 103291PubMedEurope PMCHum Mol Genet 31:275-288 (2021)Mapped to16