Cas9 gene therapy for Angelman syndrome traps Ube3a-ATS long non-coding RNA
Justin M. Wolter, Hanqian Mao, Giulia Fragola, Jeremy M. Simon, James L. Krantz, Hannah Bazick, Baris Oztemiz, Jason L. Stein, Mark J. Zylka
Topics & Concepts
UBE3AAngelman syndromeBiologyGeneticsNeurodevelopmental disorderCas9Adeno-associated virusGenomic imprintingCRISPRAlleleRNAPhenotypeGeneVector (molecular biology)Gene expressionRecombinant DNADNA methylationUbiquitinUbiquitin ligaseCRISPR and Genetic EngineeringGenetic Syndromes and ImprintingRNA modifications and cancer